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The first gene-editing treatment: 10 Breakthrough Technologies 2024

Sickle-cell disease is the first illness to be beaten by CRISPR, but the new treatment comes with an expected price tag of $2 to $3 million.

  • Antonio Regalado archive page

a scientist looks at a tall strand of DNA in a suspension of liquid. A hose sends the liquid back to an IV and into the arm of a patient seated comfortably in a domestic chair with two nice plants and a happy, observant cat.

CRISPR Therapeutics, Editas Medicine, Precision BioSciences, Vertex Pharmaceuticals

The first gene-editing cure has arrived. Grateful patients are calling it “life changing.”

It was only 11 years ago that scientists first developed the potent DNA-snipping technology called CRISPR. Now they’ve brought CRISPR out of the lab and into real medicine with a treatment that cures the symptoms of sickle-cell disease.

Sickle-cell is caused by inheriting two bad copies of one of the genes that make hemoglobin. Symptoms include bouts of intense pain, and life expectancy with the disease is just 53 years. It affects 1 in 4,000 people in the US, nearly all of them African-American. 

So how did this disease become CRISPR’s first success ? A fortuitous fact of biology is part of the answer. Our bodies harbor another way to make hemoglobin that turns off when we’re born. Researchers found that a simple DNA edit to cells from the bone marrow could turn it back on.

Many CRISPR treatments are in trials, but in 2022, Vertex Pharmaceuticals, based in Boston, was first to bring one to regulators for approval. That treatment was for sickle-cell. After their bone marrow was edited, nearly all the patients who volunteered in the trial were pain free. 

Good news. But the expected price tag of the gene-editing treatment is $2 to $3 million. And Vertex has no immediate plans to offer it in Africa—where sickle-cell disease is most common, and where it still kills children.

The company says this is because the treatment regimen is so complex. It involves a hospital stay; doctors remove the bone marrow, edit the cells, and then transplant them back. In countries that still struggle to cover basic health needs, the procedure remains too demanding. So simpler, cheaper ways to deliver CRISPR could come next. 

Biotechnology and health

Fda advisors just said no to the use of mdma as a therapy.

The studies demonstrating MDMA’s efficacy against PTSD left experts with too many questions to greenlight the treatment.

  • Cassandra Willyard archive page

Biotech companies are trying to make milk without cows

The bird flu crisis on dairy farms could boost interest in milk protein manufactured in microorganisms and plants. 

What’s next for MDMA

The FDA is poised to approve the notorious party drug as a therapy. Here’s what it means, and where similar drugs stand in the US. 

Is this the end of animal testing?

Researchers are increasingly turning to organ-on-a-chip technology for drug testing and other applications.

  • Harriet Brown archive page

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Screening of Candida spp. in wastewater in Brazil during COVID-19 pandemic: workflow for monitoring fungal pathogens

Fungal diseases are often linked to poverty, which is associated with poor hygiene and sanitation conditions that have been severely worsened by the COVID-19 pandemic. Moreover, COVID-19 patients are treated w...

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Transgenic Arabidopsis thaliana plants expressing bacterial γ-hexachlorocyclohexane dehydrochlorinase LinA

γ-Hexachlorocyclohexane (γ-HCH), an organochlorine insecticide of anthropogenic origin, is a persistent organic pollutant (POP) that causes environmental pollution concerns worldwide. Although many γ-HCH-degra...

Molecular and agro-morphological characterization of new barley genotypes in arid environments

Genetic diversity, population structure, agro-morphological traits, and molecular characteristics, are crucial for either preserving genetic resources or developing new cultivars. Due to climate change, water ...

Microvesicles-delivering Smad7 have advantages over microvesicles in suppressing fibroblast differentiation in a model of Peyronie’s disease

This study compared the differences of microvesicles (MVs) and microvesicles-delivering Smad7 (Smad7-MVs) on macrophage M1 polarization and fibroblast differentiation in a model of Peyronie’s disease (PD).

Improvement and prediction of the extraction parameters of lupeol and stigmasterol metabolites of Melia azedarach with response surface methodology

Melia azedarach is known as a medicinal plant that has wide biological activities such as analgesic, antibacterial, and antifungal effects and is used to treat a wide range of diseases such as diarrhea, malaria, ...

Dual release of daptomycin and BMP-2 from a composite of β-TCP ceramic and ADA gelatin

Antibiotic-containing carrier systems are one option that offers the advantage of releasing active ingredients over a longer period of time. In vitro sustained drug release from a carrier system consisting of ...

Minimizing IP issues associated with gene constructs encoding the Bt toxin - a case study

As part of a publicly funded initiative to develop genetically engineered Brassicas (cabbage, cauliflower, and canola) expressing Bacillus thuringiensis Crystal ( Cry )-encoded insecticidal (Bt) toxin for Indian an...

Activating the healing process: three-dimensional culture of stem cells in Matrigel for tissue repair

To establish a strategy for stem cell-related tissue regeneration therapy, human gingival mesenchymal stem cells (hGMSCs) were loaded with three-dimensional (3D) bioengineered Matrigel matrix scaffolds in high...

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Co-overexpression of chitinase and β-1,3-glucanase significantly enhanced the resistance of Iranian wheat cultivars to Fusarium

Fusarium head blight (FHB) is a devastating fungal disease affecting different cereals, particularly wheat, and poses a serious threat to global wheat production. Chitinases and β-glucanases are two important ...

A new mRNA structure prediction based approach to identifying improved signal peptides for bone morphogenetic protein 2

Signal peptide (SP) engineering has proven able to improve production of many proteins yet is a laborious process that still relies on trial and error. mRNA structure around the translational start site is imp...

Correction: Transcriptomic and targeted metabolomic analyses provide insights into the flavonoids biosynthesis in the flowers of Lonicera macranthoides

The original article was published in BMC Biotechnology 2024 24 :19

A model approach to show that monocytes can enter microporous β-TCP ceramics

β-TCP ceramics are versatile bone substitute materials and show many interactions with cells of the monocyte-macrophage-lineage. The possibility of monocytes entering microporous β-TCP ceramics has however not...

Nutritional composition, lipid profile and stability, antioxidant activities and sensory evaluation of pasta enriched by linseed flour and linseed oil

Pasta assortments fortified with high quality foods are a modern nutritional trends. This study, explored the effects of fortification with linseed flour (LF) and linseed oil (LO) on durum wheat pasta characte...

In vitro assessment of the effect of magnetic fields on efficacy of biosynthesized selenium nanoparticles by Alborzia kermanshahica

Cyanobacteria represent a rich resource of a wide array of unique bioactive compounds that are proving to be potent sources of anticancer drugs. Selenium nanoparticles (SeNPs) have shown an increasing potentia...

ECM-mimetic, NSAIDs loaded thermo-responsive, immunomodulatory hydrogel for rheumatoid arthritis treatment

Rheumatoid arthritis (RA) is a chronic inflammatory autoimmune disease, and it leads to irreversible inflammation in intra-articular joints. Current treatment approaches for RA include non-steroidal anti-infla...

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Development of a chemiluminescence assay for tissue plasminogen activator inhibitor complex and its applicability to gastric cancer

Venous thromboembolism (VTE), is a noteworthy complication in individuals with gastric cancer, but the current diagnosis and treatment methods lack accuracy. In this study, we developed a t-PAIC chemiluminesce...

High-performance internal circulation anaerobic granular sludge reactor for cattle slaughterhouse wastewater treatment and simultaneous biogas production

This research investigates the efficacy of a high-performance pilot-scale Internal Circulation Anaerobic Reactor inoculated with Granular Sludge (ICAGSR) for treating cattle slaughterhouse wastewater while con...

Hindering the biofilm of microbial pathogens and cancer cell lines development using silver nanoparticles synthesized by epidermal mucus proteins from Clarias gariepinus

Scientists know very little about the mechanisms underlying fish skin mucus, despite the fact that it is a component of the immune system. Fish skin mucus is an important component of defence against invasive ...

3D printing of Ceffe-infused scaffolds for tailored nipple-like cartilage development

The reconstruction of a stable, nipple-shaped cartilage graft that precisely matches the natural nipple in shape and size on the contralateral side is a clinical challenge. While 3D printing technology can eff...

A cleavable peptide adapter augments the activity of targeted toxins in combination with the glycosidic endosomal escape enhancer SO1861

Treatment with tumor-targeted toxins attempts to overcome the disadvantages of conventional cancer therapies by directing a drug’s cytotoxic effect specifically towards cancer cells. However, success with targ...

Multiprotein collagen/keratin hydrogel promoted myogenesis and angiogenesis of injured skeletal muscles in a mouse model

Volumetric loss is one of the challenging issues in muscle tissue structure that causes functio laesa . Tissue engineering of muscle tissue using suitable hydrogels is an alternative to restoring the physiological...

Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF

The advancement of AAV vectors into clinical testing has accelerated rapidly over the past two decades. While many of the AAV vectors being utilized in clinical trials are derived from natural serotypes, engin...

Rice yellow mottle virus is a suitable amplicon vector for an efficient production of an anti-leishmianiasis vaccine in Nicotiana benthamiana leaves

Since the 2000’s, plants have been used as bioreactors for the transient production of molecules of interest such as vaccines. To improve protein yield, “amplicon” vectors based on plant viruses are used. Thes...

Extraction and analysis of high-quality chloroplast DNA with reduced nuclear DNA for medicinal plants

Obtaining high-quality chloroplast genome sequences requires chloroplast DNA (cpDNA) samples that meet the sequencing requirements. The quality of extracted cpDNA directly impacts the efficiency and accuracy o...

Transcriptomic and targeted metabolomic analyses provide insights into the flavonoids biosynthesis in the flowers of Lonicera macranthoides

Flavonoids are one of the bioactive ingredients of Lonicera macranthoides ( L. macranthoides ), however, their biosynthesis in the flower is still unclear. In this study, combined transcriptomic and targeted metabo...

The Correction to this article has been published in BMC Biotechnology 2024 24 :33

Effects of solid lipid nanocarrier containing methyl urolithin A by coating folate-bound chitosan and evaluation of its anti-cancer activity

Nanotechnology-based drug delivery systems have received much attention over the past decade. In the present study, we synthesized Methyl Urolithin A-loaded solid lipid nanoparticles decorated with the folic a...

Neq2X7: a multi-purpose and open-source fusion DNA polymerase for advanced DNA engineering and diagnostics PCR

Thermostable DNA polymerases, such as Taq isolated from the thermophilic bacterium Thermus aquaticus , enable one-pot exponential DNA amplification known as polymerase chain reaction (PCR). However, properties oth...

A solution for highly efficient electroporation of primary cytotoxic T lymphocytes

Cytotoxic T lymphocytes (CTLs) are central players in the adaptive immune response. Their functional characterization and clinical research depend on efficient and reliable transfection. Although various metho...

Adsorption of Hg 2+ /Cr 6+ by metal-binding proteins heterologously expressed in Escherichia coli

Removal of heavy metals from water and soil is a pressing challenge in environmental engineering, and biosorption by microorganisms is considered as one of the most cost-effective methods. In this study, the m...

Derivation of a novel antimicrobial peptide from the Red Sea Brine Pools modified to enhance its anticancer activity against U2OS cells

Cancer associated drug resistance is a major cause for cancer aggravation, particularly as conventional therapies have presented limited efficiency, low specificity, resulting in long term deleterious side eff...

Polyphyllin B inhibited STAT3/NCOA4 pathway and restored gut microbiota to ameliorate lung tissue injury in cigarette smoke-induced mice

Smoking was a major risk factor for chronic obstructive pulmonary disease (COPD). This study plan to explore the mechanism of Polyphyllin B in lung injury induced by cigarette smoke (CSE) in COPD.

Quantifying carboxymethyl lysine and carboxyethyl lysine in human plasma: clinical insights into aging research using liquid chromatography-tandem mass spectrometry

The objective of this study was to establish a methodology for determining carboxymethyl lysine (CML) and carboxyethyl lysine (CEL) concentrations in human plasma using liquid chromatography-tandem mass spectr...

Iron/Copper/Phosphate nanocomposite as antimicrobial, antisnail, and wheat growth-promoting agent

One of the current challenges is to secure wheat crop production to meet the increasing global food demand and to face the increase in its purchasing power. Therefore, the current study aimed to exploit a new ...

Staphopain mediated virulence and antibiotic resistance alteration in co-infection of Staphylococcus aureus and Pseudomonas aeruginosa : an animal model

Polymicrobial communities lead to worsen the wound infections, due to mixed biofilms, increased antibiotic resistance, and altered virulence production. Promising approaches, including enzymes, may overcome th...

Strain-specific features of Pleurotus ostreatus growth in vitro and some of its biological activities

The production of Pleurotus ostreatus mycelium as a promising object for use in food and other industries is hampered by a lack of information about the strain-specificity of this fungus mycelium growth and its a...

Antibacterial, antibiofilm, and anticancer activity of silver-nanoparticles synthesized from the cell-filtrate of Streptomyces enissocaesilis

Silver nanoparticles (Ag-NPs) have a unique mode of action as antibacterial agents in addition to their anticancer and antioxidant properties. In this study, microbial nanotechnology is employed to synthesize ...

Deep orange gene editing triggers temperature-sensitive lethal phenotypes in Ceratitis capitata

The Mediterranean fruit fly, Ceratitis capitata , is a significant agricultural pest managed through area-wide integrated pest management (AW-IPM) including a sterile insect technique (SIT) component. Male-only re...

Characterization, modeling, and anticancer activity of L.arginase production from marine Bacillus licheniformis OF2

L-arginase, is a powerful anticancer that hydrolyzes L-arginine to L-ornithine and urea. This enzyme is widely distributed and expressed in organisms like plants, fungi, however very scarce from bacteria. Our ...

Green and environmentally friendly synthesis of silver nanoparticles with antibacterial properties from some medicinal plants

Recently there have been a variety of methods to synthesize silver nanoparticles, among which the biosynthesis method is more noticeable due to features like being eco-friendly, simple, and cost-efficient. The...

Reaping the benefits of liquid handlers for high-throughput gene expression profiling in a marine model invertebrate

Modern high-throughput technologies enable the processing of a large number of samples simultaneously, while also providing rapid and accurate procedures. In recent years, automated liquid handling workstation...

Induction of antimicrobial, antioxidant metabolites production by co-cultivation of two red-sea-sponge-associated Aspergillus sp. CO2 and Bacillus sp. COBZ21

The growing spread of infectious diseases has become a potential global health threat to human beings. According to WHO reports, in this study, we investigated the impact of co-cultivating the isolated endophy...

A novel starch-active lytic polysaccharide monooxygenase discovered with bioinformatics screening and its application in textile desizing

Lytic polysaccharide monooxygenases (LPMOs) catalyzing the oxidative cleavage of different types of polysaccharides have potential to be used in various industries. However, AA13 family LPMOs which specificall...

Tuning spacer length improves the functionality of the nanobody-based VEGFR2 CAR T cell

The chimeric antigen receptor-expressing T (CAR-T) cells for cancer immunotherapy have obtained considerable clinical importance. CAR T cells need an optimized intracellular signaling domain to get appropriate...

Fabrication and characterization of metformin-loaded PLGA/Collagen nanofibers for modulation of macrophage polarization for tissue engineering and regenerative medicine

In tissue engineering (TE) and regenerative medicine, the accessibility of engineered scaffolds that modulate inflammatory states is extremely necessary. The aim of the current work was to assess the efficacy ...

Production of a potential multistrain probiotic in co-culture conditions using agro-industrial by-products-based medium for fish nutrition

Probiotics are viable microorganisms that when administered in adequate amounts confer health benefits to the host. In fish, probiotic administration has improved growth, and immunological parameters. For this...

Research on the targeted improvement of the yield of a new VB 12 -producing strain, Ensifer adhaerens S305, based on genomic and transcriptomic analysis

Vitamin B 12 (VB 12 ) has a wide range of applications and high economic value. In this study, a new strain with high VB 12 production potential, Ensifer adhaerens S305, was identified in sewage. Because E. adhaerens

Validation and calibration of a novel GEM biosensor for specific detection of Cd 2+ , Zn 2+ , and Pb 2+

In this study, we designed a novel genetic circuit sensitive to Cd 2+ , Zn 2+ and Pb 2+ by mimicking the CadA/CadR operon system mediated heavy metal homeostasis mechanism of Pseudomonas aeruginosa . The regular DNA m...

Exploring the microbial diversity and characterization of cellulase and hemicellulase genes in goat rumen: a metagenomic approach

Goat rumen microbial communities are perceived as one of the most potential biochemical reservoirs of multi-functional enzymes, which are applicable to enhance wide array of bioprocesses such as the hydrolysis...

The transcriptional factor Clr-5 is involved in cellulose degradation through regulation of amino acid metabolism in Neurospora crassa

Filamentous fungi are efficient degraders of plant biomass and the primary producers of commercial cellulolytic enzymes. While the transcriptional regulation mechanisms of cellulases have been continuously exp...

An online soft sensor method for biochemical reaction process based on JS-ISSA-XGBoost

A method combining offline techniques and the just-in-time learning strategy (JITL) is proposed, because the biochemical reaction process often encounters changing features and parameters over time.

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Existing high blood pressure drugs may prevent epilepsy, Stanford Medicine-led study finds

In an analysis of more than 2 million patient records, researchers discovered that people taking angiotensin receptor blockers for high blood pressure were less likely to develop epilepsy.

June 18, 2024 - By Sarah C.P. Williams

medication

A class of blood pressure drugs appears to reduce the chance of developing epilepsy. Sophie -   stock.adobe.com

A class of drugs already on the market to lower blood pressure appears to reduce adults’ risk of developing epilepsy, Stanford Medicine researchers and their colleagues have discovered. The finding comes out of an analysis of the medical records of more than 2 million Americans taking blood pressure medications.

The  study , published June 17 in  JAMA Neurology , suggests that the drugs, called angiotensin receptor blockers, could prevent epilepsy in people at highest risk of the disease, including older adults who have had strokes. 

“This is incredibly exciting because we don’t currently have any medicines that prevent epilepsy,” said  Kimford Meador , MD, a professor of neurology and neurological sciences and the senior author of the paper. “I hope these initial findings lead to randomized clinical trials.”

Preventing seizures after stroke

While epilepsy is often diagnosed during childhood, more than 1% of people over age 65 are diagnosed with the recurring seizures that characterize the disorder. These seizures can temporarily disrupt the brain’s function and cause a range of symptoms. 

In older adults, the most common risk factor for developing epilepsy is stroke; about 10% of stroke survivors experience seizures within five years. Vascular disease and chronic high blood pressure, even in the absence of stroke, also boost epilepsy risk. 

“This can be a very debilitating disorder, and it’s much more common in older adults than people realize,” said Meador, a member of the Wu Tsai Neurosciences Institute .

Although anti-seizure medications can be used to control epilepsy after diagnosis, no drugs are approved to prevent epilepsy in people at high risk of developing the disorder. 

During the past decade, however, studies have suggested that one type of blood pressure medication might help quell seizures because of their ability to tamp down inflammation. This aspect would be particularly apt for preventing seizures that follow stroke or traumatic brain injuries, as both cause brain inflammation that can trigger epilepsy. 

In 2022, a study of more than 160,000 people in Germany found that people taking angiotensin receptor blockers — one of multiple classes of drugs prescribed to treat high blood pressure — had a diminished risk of developing epilepsy. The drugs block certain hormone receptors, leading to lower blood pressure and decreased inflammation in blood vessels and other organs — including the brain. 

Kimford Meador

Kimford Meador

“Those results out of Germany echoed what had been found in animal studies and seemed very promising, but I felt that it was important to reproduce that analysis using data on people in the U.S.,” Meador said. 

A bigger, broader data set

For the new study, Meador and colleagues at the University of Rhode Island turned to a national database that includes information on health care claims from more than 20 million Americans enrolled in either commercial health insurance plans or Medicare — a group more racially diverse than that in the German study. They focused their analysis on 2.2 million adults who had been diagnosed with high blood pressure, were prescribed at least one high blood pressure medication and did not already have epilepsy. 

Overall, people taking angiotensin receptor blockers had a 20% to 30% lower risk of developing epilepsy between 2010 and 2017 compared with people taking other blood pressure drugs. This difference held true even when patients with strokes were removed from the analysis, suggesting that the lower rates of epilepsy were not a result solely of a decreased risk of stroke. 

“What we’ve done is replicate what was found in Germany but in a larger and completely different population,” Meador said. “That really increases the strength of the signal and tells us that there’s something real going on here.”

The data also indicated that one particular angiotensin receptor blocker — losartan — had the most powerful effect on lowering epilepsy risk, but the researchers said more work is needed to confirm that. 

Toward clinical trials

All blood pressure medications likely have an impact on decreasing epilepsy risk because high blood pressure is a contributing factor to epilepsy. Keeping blood pressure under control through any combination of antihypertensive drugs and lifestyle factors can therefore lower a person’s chance of developing epilepsy, Meador said. 

However, the new research suggests that angiotensin receptor blockers might be more beneficial than other antihypertensives for patients to reduce the risk of epilepsy. In the new study, about 14% of people taking a blood pressure drug took angiotensin receptor blockers, while most took other classes of drugs to control their blood pressure, including beta blockers, calcium channel blockers and angiotensin-converting enzyme inhibitors. 

“This could be a new chapter in the story of preventive medicine,” Meador said. “There are so many people with stroke or high blood pressure; knowing that this class of drug not only lowers blood pressure but also helps lower their epilepsy risk could change how we treat them.”

However, Meador added, randomized clinical trials are needed to prove the association between angiotensin receptor blockers and reduce epilepsy risk before treatment guidelines change. 

Researchers from Brown University were also involved in the research. 

The researchers have no outside funding sources or conflicts of interest to disclose.

  • Sarah C.P. Williams Sarah C.P. Williams is a freelance science writer.

About Stanford Medicine

Stanford Medicine is an integrated academic health system comprising the Stanford School of Medicine and adult and pediatric health care delivery systems. Together, they harness the full potential of biomedicine through collaborative research, education and clinical care for patients. For more information, please visit med.stanford.edu .

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Innovative system enhances biological-artificial interactions in neurological research

by University of Tokyo

Innovative system enhances biological-artificial interactions in neurological research

Conducting biohybrid experiments between living and artificial cells is necessary to develop the next generation of neuromorphic-based neuroprostheses. Toward this goal, teams from France, Japan, and Italy have developed a new tool to study closed-loop interactions in neuroscience.

Currently, pharmacological treatments for neurological disorders remain limited, driving the exploration of promising alternative approaches, such as electroceutics. Recent research in bioelectronics and neuromorphic engineering has facilitated the development of new-generation neuroprostheses for brain repair. However, realizing their full potential requires a deeper understanding of biohybrid interactions.

In an article published in Nature Communications , the authors introduce BioemuS, a low-cost, embedded, flexible, and real-time biomimetic tool that allows biohybrid experiments to be performed to emulate living systems in real time. This new system simplifies investigating and replicating biophysically detailed neural network dynamics, while prioritizing cost efficiency , flexibility, and ease of use.

"We showcase the feasibility of conducting biohybrid experiments using standard biophysical interfaces and a variety of biological cells, as well as the real-time emulation of diverse network configurations," says the lead author of the study, Romain Beaubois.

This embedded system offers a real-time, cost-effective, and user-friendly solution for closed-loop applications, addressing the accessibility challenges prevalent in current high-performance alternatives.

Unlike server-based infrastructures or complex systems which can be expensive and difficult to integrate into experimental setups, this new solution prioritizes simplicity and accessibility. Notably, despite GPU-accelerated computing, even software alternatives frequently struggle to achieve the low latencies necessary for closed-loop applications.

"We envision our system as a crucial step toward the development of neuromorphic-based neuroprostheses for bioelectrical therapeutics, enabling seamless communication with biological networks on a comparable timescale. The embedded real-time functionality of BiœmuS enhances practicality and accessibility, amplifying its potential for real-world applications in biohybrid experiments," explains Timothee Levi, the senior and corresponding author of the study.

"The system is a landmark achievement through our interdisciplinary collaborative efforts," says Yoshiho Ikeuchi, senior author of the article.

"We have been collaborating and exchanging ideas over the past few years. We recently published another collaborative article in Nature Communications , in which we reported that the connection between two cerebral organoids enhances their activity and complexity. We hope to make better neuronal characterization and functionalization systems by sharing these advances and communicating them effectively with the scientific community."

This research highlights the importance of multidisciplinary projects and international collaboration . Dr. Beaubois and Prof. Levi from the University of Bordeaux and CNRS, through the IMS and LIMMS laboratory, developed this real-time tool for emulating biological dynamics.

The tool was subsequently used in biohybrid experiments targeting organoids in vitro in collaboration with Prof. Ikeuchi from the Institute of Industrial Science at The University of Tokyo and applied to rodent experiments in vivo in collaboration with Prof. Chiappalone from DIBRIS at the University of Genova.

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As the F.D.A. considers a new Alzheimer’s medication, we asked experts how the rollout of a similar drug has gone.

An illustration of an Alzheimer's patient receiving an infusion of a new drug. Part of the neural pathways of the brain are blurry showing the deterioration.

By Dana G. Smith

Over the last three years, a new class of Alzheimer’s drug, the first to treat a root cause of the disease, has set off a roller coaster of hope and disappointment. But while these so-called anti-amyloid antibodies had a rough start , many patients and their doctors are feeling more optimistic now that one of the medications is finally being used more widely.

Lecanemab (brand name Leqembi; pronounced le-KEM-bee) was given full approval by the Food and Drug Administration in July 2023 and is currently the only one of its class available to Alzheimer’s patients, outside clinical trials. The drug has been shown to slow the progression of the disease, but its benefits are fairly modest. It is also a burdensome therapy and has a high risk of troubling side effects.

With lecanemab having been approved for nearly a year — and with a similar drug, donanemab , recommended for approval by an F.D.A. advisory committee at a meeting on Monday — The New York Times checked in with experts at three major medical centers about who’s receiving lecanemab and how they’re responding.

Who’s being prescribed lecanemab?

There are strict requirements for patients to be eligible for lecanemab; by one estimate , fewer than 20 percent met the qualifications for the medication. Neurologists at the Mayo Clinic, Massachusetts General Hospital and the University of California, San Francisco, all described a similar review process when deciding which patients are good candidates.

First, the patient must be diagnosed with mild cognitive impairment or mild dementia, the earliest two stages of Alzheimer’s disease. Second, because lecanemab works by removing the amyloid plaques that are a hallmark of the disease, patients undergo a PET scan or a lumbar puncture to make sure plaques are actually present in the brain. Third, the patient needs an M.R.I. to screen for signs of other brain diseases.

“We want to make sure that they don’t have another explanation for their cognitive problems,” said Dr. Ronald Petersen, the director of the Mayo Clinic Alzheimer’s Disease Research Center.

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A Clinical Diagnostic Test for Calcium Release Deficiency Syndrome

  • 1 Libin Cardiovascular Institute, Department of Physiology and Pharmacology, University of Calgary, Calgary, Alberta, Canada
  • 2 Section of Cardiac Electrophysiology, Division of Cardiology, Department of Medicine, Hamilton Health Sciences and McMaster University, Hamilton, Ontario, Canada
  • 3 Jesselson Integrated Heart Center, Eisenberg R&D Authority, Shaare Zedek Medical Center, and Hebrew University Faculty of Medicine, Jerusalem, Israel
  • 4 Division of Cardiovascular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, England
  • 5 Oxford Heart Centre, John Radcliffe Hospital, Oxford, England
  • 6 Department of Cardiology, Faculty of Medicine and Health Sciences, Antwerp University Hospital, Antwerp, Belgium
  • 7 Cardiovascular Research, Departments of Genetics, Pharmacology and Physiopathology of Heart, Blood Vessels and Skeleton, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium
  • 8 Member of the European Reference Network for Rare, Low Prevalence, and Complex Diseases of the Heart (ERN GUARD-Heart)
  • 9 Department of Clinical Cardiology, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands
  • 10 Heart Failure and Arrhythmias, Amsterdam Cardiovascular Sciences, Amsterdam, the Netherlands
  • 11 Section of Cardiac Electrophysiology, Division of Cardiology, Department of Medicine, Western University, London, Ontario, Canada
  • 12 Montreal Heart Institute and Université de Montréal, Montreal, Quebec, Canada
  • 13 Department of Cardiology, Aarhus University Hospital, Aarhus N, Denmark
  • 14 Institut Universitaire de Cardiologie et de Pneumologie de Québec, Laval University, Quebec City, Quebec, Canada
  • 15 Department of Cardiac Pacing and Electrophysiology, Hopital Cardiologique du Haut-Leveque, Centre Hospitalier Universitaire de Bordeaux, Pessac, France
  • 16 Division of Cardiology and Centre for Cardiovascular Innovation, University of British Columbia, Vancouver, Canada
  • 17 Department of Molecular Cardiology, IRCCS Istituti Clinici Scientifici Maugeri, Pavia, Italy
  • 18 Department of Molecular Medicine, University of Pavia, Pavia, Italy
  • 19 Windland Smith Rice Genetic Heart Rhythm Clinic, Division of Heart Rhythm Services, Department of Cardiovascular Medicine, Mayo Clinic, Rochester, Minnesota
  • 20 Department of Cardiovascular Medicine, Mayo Clinic, Rochester, Minnesota
  • 21 Section of Cardiac Electrophysiology, Division of Cardiology, University of Washington Medical Center, Seattle
  • 22 Population Health Research Institute, Hamilton Health Sciences, Hamilton, Ontario, Canada
  • 23 Section of Cardiac Electrophysiology, Division of Cardiology, Department of Medicine, University of California, San Francisco
  • 24 Windland Smith Rice Sudden Death Genomics Laboratory, Department of Molecular Pharmacology and Experimental Therapeutics, Mayo Clinic, Rochester, Minnesota
  • 25 Division of Pediatric Cardiology, Department of Pediatric and Adolescent Medicine, Mayo Clinic, Rochester, Minnesota
  • 26 Inherited Arrhythmia and Cardiomyopathy Program, Arrhythmia Service, Division of Cardiology, Toronto General Hospital and the University of Toronto, Toronto, Ontario, Canada
  • 27 Leviev Heart Institute, Chaim Sheba Medical Center, Ramat Gan, Israel
  • 28 Tel Aviv University, Tel Aviv, Israel
  • 29 Oxford Biomedical Research Centre and Wellcome Centre for Human Genetics, University of Oxford, Oxford, England
  • 30 Department of Clinical Medicine, Aarhus University, Aarhus C, Denmark
  • 31 Heart Institute, Hadassah University Hospital, Jerusalem, Israel
  • Editor's Note Clinical Test for Calcium Release Deficiency Syndrome? Gregory M. Marcus, MD, MAS; Gregory Curfman, MD; Kirsten Bibbins-Domingo, PhD, MD, MAS JAMA

Question   Cardiac arrest frequently occurs without explanation, even after a thorough clinical evaluation. Can a simple maneuver clinically diagnose calcium release deficiency syndrome (CRDS), a newly described cause of sudden death?

Findings   In this international, multicenter, case-control study, a provoked measure of T-wave amplitude on an electrocardiogram ascertained cases of CRDS with high accuracy. The genetic mouse models recapitulated the human findings and suggested a pathologically large systolic calcium release from the sarcoplasmic reticulum was responsible.

Meaning   These preliminary results suggest that the repolarization response on an electrocardiogram to brief tachycardia followed by a pause may effectively diagnose CRDS. Given the frequency of unexplained cardiac arrest, should these findings be confirmed in larger studies, this readily available maneuver may provide clinically actionable information.

Importance   Sudden death and cardiac arrest frequently occur without explanation, even after a thorough clinical evaluation. Calcium release deficiency syndrome (CRDS), a life-threatening genetic arrhythmia syndrome, is undetectable with standard testing and leads to unexplained cardiac arrest.

Objective   To explore the cardiac repolarization response on an electrocardiogram after brief tachycardia and a pause as a clinical diagnostic test for CRDS.

Design, Setting, and Participants   An international, multicenter, case-control study including individual cases of CRDS, 3 patient control groups (individuals with suspected supraventricular tachycardia; survivors of unexplained cardiac arrest [UCA]; and individuals with genotype-positive catecholaminergic polymorphic ventricular tachycardia [CPVT]), and genetic mouse models (CRDS, wild type, and CPVT were used to define the cellular mechanism) conducted at 10 centers in 7 countries. Patient tracings were recorded between June 2005 and December 2023, and the analyses were performed from April 2023 to December 2023.

Intervention   Brief tachycardia and a subsequent pause (either spontaneous or mediated through cardiac pacing).

Main Outcomes and Measures   Change in QT interval and change in T-wave amplitude (defined as the difference between their absolute values on the postpause sinus beat and the last beat prior to tachycardia).

Results   Among 10 case patients with CRDS, 45 control patients with suspected supraventricular tachycardia, 10 control patients who experienced UCA, and 3 control patients with genotype-positive CPVT, the median change in T-wave amplitude on the postpause sinus beat (after brief ventricular tachycardia at ≥150 beats/min) was higher in patients with CRDS ( P  < .001). The smallest change in T-wave amplitude was 0.250 mV for a CRDS case patient compared with the largest change in T-wave amplitude of 0.160 mV for a control patient, indicating 100% discrimination. Although the median change in QT interval was longer in CRDS cases ( P  = .002), an overlap between the cases and controls was present. The genetic mouse models recapitulated the findings observed in humans and suggested the repolarization response was secondary to a pathologically large systolic release of calcium from the sarcoplasmic reticulum.

Conclusions and Relevance   There is a unique repolarization response on an electrocardiogram after provocation with brief tachycardia and a subsequent pause in CRDS cases and mouse models, which is absent from the controls. If these findings are confirmed in larger studies, this easy to perform maneuver may serve as an effective clinical diagnostic test for CRDS and become an important part of the evaluation of cardiac arrest.

  • Editor's Note Clinical Test for Calcium Release Deficiency Syndrome? JAMA

Read More About

Ni M , Dadon Z , Ormerod JOM, et al. A Clinical Diagnostic Test for Calcium Release Deficiency Syndrome. JAMA. Published online June 20, 2024. doi:10.1001/jama.2024.8599

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  • Published: 15 November 2022

Medicine and health of 21st Century: Not just a high biotech-driven solution

  • Mourad Assidi   ORCID: orcid.org/0000-0003-2750-1764 1 , 2 ,
  • Abdelbaset Buhmeida 1 &
  • Bruce Budowle   ORCID: orcid.org/0000-0003-4116-2930 3  

npj Genomic Medicine volume  7 , Article number:  67 ( 2022 ) Cite this article

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  • Molecular medicine
  • Quality of life

Many biotechnological innovations have shaped the contemporary healthcare system (CHS) with significant progress to treat or cure several acute conditions and diseases of known causes (particularly infectious, trauma). Some have been successful while others have created additional health care challenges. For example, a reliance on drugs has not been a panacea to meet the challenges related to multifactorial noncommunicable diseases (NCDs)—the main health burden of the 21st century. In contrast, the advent of omics-based and big data technologies has raised global hope to predict, treat, and/or cure NCDs, effectively fight even the current COVID-19 pandemic, and improve overall healthcare outcomes. Although this digital revolution has introduced extensive changes on all aspects of contemporary society, economy, firms, job market, and healthcare management, it is facing and will face several intrinsic and extrinsic challenges, impacting precision medicine implementation, costs, possible outcomes, and managing expectations. With all of biotechnology’s exciting promises, biological systems’ complexity, unfortunately, continues to be underestimated since it cannot readily be compartmentalized as an independent and segregated set of problems, and therefore is, in a number of situations, not readily mimicable by the current algorithm-building proficiency tools. Although the potential of biotechnology is motivating, we should not lose sight of approaches that may not seem as glamorous but can have large impacts on the healthcare of many and across disparate population groups. A balanced approach of “omics and big data” solution in CHS along with a large scale, simpler, and suitable strategies should be defined with expectations properly managed.

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Historical context of over-reliance on biotechnology-driven treatments.

The contemporary healthcare system (CHS) has been shaped by century-long innovations and discoveries made notably in the late 1800s and early 1900s 1 . To achieve the ultimate goal of allowing people to live longer and healthier, scientists and clinicians, among others, have made remarkable efforts to continuously enhance the CHS, which has improved the lives of nearly every person on the planet, although not necessarily equally (i.e., there are disparity issues that need to be addressed). While preventive healthcare is practiced by some, CHS is mainly a reactive approach strategy that often waits until the person becomes ill with acute symptoms to undertake a specific surgical intervention and/or a drug-based corrective action.

The “magic bullet” era

Drug therapy began centuries ago with the use of plant extracts and progressively evolved into the development of purified and targeted materials for a wide range of health-related applications, such as morphine (1803), anesthetics (1840s), antipyretics, and analgesics (1870s) in the 19th century. At the beginning of the 20th century, Ehrlich’s research laid the foundations for drug screening and discovery by bridging the gap between chemistry, biology, and medicine. His research discovered one of the first “magic bullets”—the antisyphilitic drug Arsphenamine. Other treatments for other diseases were subsequently discovered, including insulin, penicillin, and chemotherapy 2 , 3 . Pharmacological research expanded significantly to develop new cures and ameliorative approaches for various diseases with many noted successes. Consequently, the proportion of patents and newly-developed pharmaceutical products increased from 25% of all pharmacy remedies in the 1940s to nearly 90% at the end of 20th century 2 . Based on the realized health benefits of drug therapy, there has become an enormous reliance on drug prescriptions and unprecedented levels of use 4 . With the rapid economic development and enhanced living standards since the end of World War II, the use of drugs has steadily increased, boosted in part by the support of insurance and social security systems.

Through marketing and lobbying adopted by pharmaceutical companies to continuously expand their markets and benefits 5 , a major bias has taken root in the public and healthcare providers’ mindset and culture: treatment of disease primarily is achieved through prescription of drugs with a concomitant (and unfortunate) lower reliance on prevention and health promotion 6 , 7 . This reliance on drug therapies has made their use varied and commonplace 4 , although some treatments have become cost prohibitive for the majority of the population contributing to healthcare disparity. The pushing of drug therapy without an appreciation of the human factor has seen a concomitant increase in patients suffering medication (ab)use and/or iatrogenic effects. Many individuals over 65 years of age in the Western world take anywhere between 5 to more than 20 drugs per day 4 , 8 , 9 . Moreover, most of these drugs are palliative treatments; for example, in the USA, 9 out of 10 prescribed drugs are pain killers and symptom relievers 10 . This drug reliance strategy has been associated with unprecedented waste in annual global healthcare expenditures due to overspending, unnecessary prescriptions, mistakes, and corruption costing upwards of USD 300 billion (according to the European Healthcare Fraud and Corruption Network) 11 . The epidemic opioids crisis in the USA is an example of drug (ab)use due to the underestimation of the neurobiological harm and the potential addiction effects mainly on individuals/groups with particular social vulnerabilities 12 , 13 . This crisis is a heavy public health burden that begets severe health, socioeconomic and legal consequences 12 .

Other challenges of the “drug-only” solution

This “drug solution” problem, in turn, is exacerbated with new challenges related to availability, accessibility, affordability, safety and effectiveness 11 . Beyond the heavy financial burden of healthcare commodification and, more importantly, drug interactions and side effects—due to both polypharmacy and inappropriate prescriptions—have led to frailty, severe comorbidities, higher hospital admissions, and increased mortality 14 . Similar issues can be seen with other hopeful cures such as vaccine access to immunize the population suffering from the current pandemic. While rather elegant biotechnology-based solutions have been undertaken to rapidly develop SARS-CoV-2 vaccines (experiencing the fastest development of an approved vaccine(s) in history), the roll out and access to the vaccine to all population groups, as well as willingness by some to receive it, has been far more challenging 15 , 16 , 17 . One would have thought that the logistics for dissemination could have been planned better 18 , 19 . Innovation and cost of vaccine purchase were not impediments but instead, a more basic distribution strategy(ies) and information dissemination should have been implemented. A well-planned distribution strategy reduces the virus reservoir, impacts the greater population, and reduces health disparities.

Despite the considerable budget allocated to drug discovery, pharmacogenomics, and high biotechnology, these fields have substantial bottlenecks in CHS, as they have high rates of failure. These failures were in part due to instrumentation, methods, statistics, computational power, machine learning, etc. that were not able to accommodate, organize, and process the information needed to provide more precise solutions. In the USA, 90% of new drug applications to the FDA are rejected because of a lack of efficacy and/or toxicity 20 . Moreover, among the most prescribed drugs in the USA, the most successful one was reported to be effective in only 25% of patients 21 . This “imprecision medicine” is mostly due to the complexity of human biology systems, inappropriate or limited settings during, for example, the drug development process, and/or the inability of a specific drug to fix multi-level molecular perturbations. Omics solutions will help in predicting those patients in which a positive effect will occur and which patients who will have no effect or adverse reactions to the treatment. Because one can foresee drug development will be targeted to only those individuals with a positive effect, the cost will continue to rise, and likely health disparity will be further exacerbated.

Advancements in biotechnology to improve human health

The promise of omics and big data sciences.

At the beginning of the 21st century, the completion of the human genome project (HGP) provided a blueprint map towards precision medicine (PM) with a promise to improve quality of life. The first deciphered blueprint in itself had little impact. However, the HGP fostered biotechnology innovation and advances in bioinformatics, such as exquisite massively parallel sequencing technologies turning the herculean effort of sequencing an entire human genome into a reasonable cost and trivial exercise today. Boosted by digital analytics, the HGP has metamorphosed the way life science research is conceived and applied. Subsequently, several new disciplines have emerged, such as biobanking, bioinformatics, comparative genomics, pharmacogenomics, clinical genomics, and projects such as the human proteome project, the human microbiome project, the cancer genome atlas project, and the illuminating druggable genome program, to name a few. Furthermore, the emergence of the digital revolution has been progressively introducing extensive changes on all aspects of contemporary society, economy, firms, and job market 22 . This huge impact has also encompassed the way science and research are conducted in every discipline 23 . In medicine, mega sets of sequence and metagenomic data, super-libraries of medical images, and complex drug databases are generated on a massive scale. These huge data sets are clear illustrations of a new complex, automated and data-driven trend to gain insights about both the clinical profiles and molecular signatures in health and disease statuses 24 , 25 , 26 . Experts estimate that innovations, such as omics biotechnologies mainly integrative personal omics profile (iPOP) 27 , connected health systems, wireless wearable devices, blockchain technology, the Internet of Things (IoT), health tokens, artificial intelligence (AI), and machine learning (ML) are promising ways to address CHS’s challenges 28 , 29 , 30 (Table 1 ).

With the advent of these omics-based biotechnologies (e.g., genomics, transcriptomics, epigenomics, proteomics, and metabolomics) and big data science, a new wave of hope has spread over the scientific and clinical communities as well as the general public, in search of instant, individualized, and accurate theranostics 31 . There are and will be CHS improvements at both the individual and population levels in NCDs and infectious diseases. While omics undoubtedly will impact positively precision medicine 32 , it is important not to lose focus that individualized solutions that can be leveraged to affect population level challenges still will provide the greatest improvement in CHS. One of the main outcomes of deciphering cancer using omics technologies was targeted therapies. In fact, targeted anticancer therapy (TAT) is an expanding area that revolutionized cancer treatment modalities and significantly improved prognosis, treatment, and prediction of several malignancies 33 , 34 . Furthermore, TATs have played a major role in converting several cancers from fatal diseases to manageable chronic conditions 35 , 36 , 37 , 38 . However, these TATs-induced improvements were lacking enough specificity and effectiveness. The wide genomic instability and tumor heterogeneity marked by myriad possible multi-mutations precludes any hope for precision treatments 33 . Therefore, TATs were often combined with the other treatment modalities as surgery, chemo, radio, hormonal therapy, and even other targeted therapies. So far, the developed TATs were not able to overcome the toxicity, and cross-reactivity on nontarget tissues, relapse, and drug resistance 37 . Notably, only a small proportion of the population benefits from TATs at a higher cost. Therefore, it is obvious that a “magic bullet” solution for cancer treatment is still unreachable.

Noteworthy, of five health determinants (genome and biology, lifestyle choices, social circumstances, environment, and healthcare system), medical care’s contribution does not exceed 11% of each individual’s health 39 , 40 . This means that 89% of one’s health is impacted by determinants outside of the CHS realm. Thus, more emphasis on the remaining health determinants will substantially improve CHS’s performance. A tendency to marginalize prevention and health promotion—perhaps due to its unprofitability character or its lack of glamour or lack of insurance support—has impeded more focus on implementation of health quality pillars and adequate prevention strategies.

For instance, half of all deaths in the U.S. were due to behavioral causes 41 and therefore may be preventable. These health-related behaviors, which are only part of the problem, were mainly driven/influenced by social determinants as education, employment, and income 41 , 42 . Another illustration of the cost-effective and global impact of the health determinants outside the healthcare realm were the findings of McKeown who demonstrated that the sharp increases in life expectancy at the 19th century in the UK was mainly triggered by the improved living conditions, such as nutrition, sanitation, and potable water availability, decades ahead of the discovery of antibiotics, vaccines, and intensive care units 41 , 43 . Strikingly, more than 75% of healthcare spending in rich countries is dedicated to managing lifestyle-induced conditions. However, it is estimated that 80% of these NCDs are preventable by readily and cost-effective lifestyle choices improvements 44 . Taken together, these findings highlight that CHS effectiveness could not be enhanced by high-tech-driven inputs only but must consider the other health determinants as the foundation of any future reform.

Although better insights and resolution about diseases’ diagnoses and stratification, as well as healthcare management, can be observed given their descriptive character 45 , the digital revolution impact on precision therapeutics may not be realized readily, except for applications such as rapid vaccine development, robotic surgery, detection of unknown pathogens, disease monitoring and predicting adverse drug reactions to name a few. The new and unprecedented challenges are related to big data and biospecimens’ collection, storage, sharing, analysis, reliability, reproducibility, interpretation, governance, and bioethics that have emerged, with accompanying logistics requirements and considerations 46 , 47 , 48 , 49 . The PM concept at this post-genomic era—although inspirational—remains costly with limited success for population level impact at least in the short and medium term 50 . We are not advocating a reduced effort in this regard but managing expectations should become part of the strategy and more so not to lose sight of alternate not as “newsworthy” strategies that may have greater outreach to improving healthcare disparity and quality.

Biology: inconceivable complexity nevertheless user-friendly

Human biology is a multi-layered complexity of dynamic and interactive networks at the single-cell, multicellular, tissue, organ, system, organismal, environmental as well as social levels. In this context, NCDs are a series of perturbations of afore described complex networks that are deeply rooted in the biology, lifestyle choices, and the engineered/devised environment in which we live today. Given its appearance as user-friendly, biology complexity continues to be, unfortunately, underestimated. While the HGP and the ensuing development of omics solutions have lofty goals, the problem of molecular complexity has been underestimated, and deciphering the genotype-phenotype relationship continues to plague reaching the “magic bullet” goal 51 . For example, Singh and Gupta point out that the unanticipated necessary and unnecessary complexity of molecular machinery and systems in conjunction with evolutionary processes make it extremely difficult, currently, to apply PM effectively. An organism’s genetic redundancy and multiple molecular pathways are complex, related, and integrated and they also affect traits and thus complicate interpretation. It should not be surprising that individuals with similar risk factors for a disease may have different phenotypes 52 , 53 . Genetic backgrounds, gene interaction networks, environments, and histories impact PM making it “uncertain, chance-ridden, and probabilistic” 52 .

The scientific community should be aware that these biological systems could not be compartmentalized as independent and segregated problems in the digital and molecular realm, and therefore are very challenging to be mimicable by the current digital tools. Although impressive strides have been made with the advent of customized artificial intelligence (AI) and machine learning (ML) algorithms that analyze the complexity of these still poorly understood biological networks, they likely will not achieve the status of the “magic bullet” solution in the near future. There is a need for education and training of algorithm-building proficiency experts—a fundamental part of the roll out of advance technological solutions that has not been a major focus of national or global strategies. Perhaps computer science or better yet bioinformatics should become a requisite course in the secondary school system or at least part of an undergraduate curriculum for all students.

It is noticeable that the development of technology and data-driven applications is significantly faster than the progress of the scientific understanding of the complex interactions in biology (e.g., assessing differences between association and causality) and related fields. The realization of AI, ML and big data promises to deliver accurate clinical decisions and robust therapeutic predictions will require first overcoming the major intrinsic challenges related to their origin and cause such as the 4 Vs (volume, velocity, variety, and veracity) 49 , 54 , 55 . The combination of complexity and limitations of big data and omics-based sciences, confounded with health disparities, has revealed that what we dream of as “precision medicine” is still “imprecision medicine” 56 . Beyond these complexities that will be challenges for the foreseeable future is the impediment of the rapidly developing, rapidly changing technology and bioinformatics. These dynamic changes are welcomed because they bring bigger and better ways to identify and use diagnostic and prognostic bioindicators. However, the swift change that is occurring is an indication that the -omics biotechnologies are far from mature and obviously not stable. Technology will have to become somewhat standardized (or stabilized) to differentiate variation in assay performance and noise from -omics contributions and to be able to compare data effectively among the multitude of studies. Moreover, unstable biotechnologies are challenging to implement into operation-oriented diagnostic laboratories as it is costly to invest, requires ever changing quality assurance practices, can create a chaotic environment and the staff in such facilities are users, not innovators of new technologies and will not be able to adjust and troubleshoot as problems arise. While there no doubt will be successes in healthcare developed in the post-genomic era, the belief that “omics and big data” are poised to become routine parts of the HCS may be premature.

Population health and prevention: Simpler solutions matter

This high reliance on drugs has been a landmark that shaped the CHS. Unfortunately, this drug-only strategy was strongly applied to be the cure of multifactorial NCDs (e.g., cardiovascular diseases, cancer, chronic obstructive pulmonary diseases, diabetes, obesity, etc.), the current main global health burden. However, NCDs have complex causes. Their vectors are embedded in multi-generic effects as patients’ genetics (to include genetic imprinting), socio-economic environment, biology, and lifestyle choices 57 , 58 . Socio-economic vectors, a large and often under considered set of factors, for instance, encompass the complex interactions and disparities between economic growth, urbanization, aging, education, globalization, and the pervasiveness of unhealthy products on the market 59 , 60 , 61 . Given such inherent complexity, trying to develop drugs for NCDs using a reductionist approach likely will have limited positive results which at best will serve subcomponents of the population and may not have the global impact desired. The treatment concept for these diseases should move from a drug only strategy carapace to a more comprehensive approach of positive change/intervention in the individual/population socio-economic environment and lifestyle choices following a more holistic approach.

History is replete of simpler solutions with large impact (Fig. 1 ). For example, during the Crimean war in 1854 where there was a shortage of medicines’ supply, the famous British nurse Florence Nightingale significantly reduced the death rates of wounded soldiers from 42 to 2%, and prevented mass infections mainly through improving the hygiene through hand washing, proper ventilation, reducing crowdedness, and sewage evacuation 62 (Fig. 1 ). Also, and following three successive and unexplained tragic outbreaks of Cholera in London, the sewage system proposed by civil engineer Joseph Bazalgette in 1859, suggested by some historians as a “hero of London”, was able to stop the water-borne transmission of disease. The role of the implemented sewage system was to pump the effluent through several interconnecting pipes beyond the metropolitan city 63 , 64 . These examples, among others, clearly pinpoint that simpler and affordable measures outside the realm of HCS and advanced biotechnologies could have significant and sustainable impact on human quality of life, wellbeing, and sustainability.

figure 1

These measures included quarantine during Black Death epidemic 95 , hygiene and social distancing in the Crimean war 62 , the building of mountain sanatoria to cure TB 96 , 97 , 98 , the implementation of a sewage system to overcome London’s Great stink 63 , 64 , and the introduction of water filtration and chlorination systems/technologies to clean potable water in the USA 99 .

COVID-19 and Socio-Economic Disparities

Currently, for instance, the world is facing successive waves of the life-threatening COVID-19 pandemic. Sadly, this coronavirus has affected about a half billion people and killed over 6 million according to WHO 65 . Indeed, humanity is expressing a deep need for science to develop a “cure” with drugs/vaccines and the power of big data more than ever before to overcome this global health issue 32 . This public health crisis has been aggravated by concomitant economic, humanitarian crises, and notable social and health disparity effects 66 , 67 . Now more than two years since the declaration of this pandemic and despite unprecedented planetary networks/initiatives, dedicated mega scale budgets, the intensive use of big data in drug/vaccines development, and the waves of seemingly effective vaccine, the ready access to vaccines is still a struggle in many parts of the world 17 . As mentioned above government dissemination strategies faltered still leaving today large portions of the population to be immunized and some countries of the world lagging well behind others. A balanced strategy of high biotechnology solutions and those other areas that affect socio-economic determinants that impact healthcare of the general population would have been well-served to meet the challenge of combatting this pandemic. Given the rapid mutation rate of SARS-CoV-2, the slow and not well-planned dissemination strategy may contribute to extending the pandemic as opposed to being the hopeful cure to end it. In contrast to individual-level management of the COVID-19 pandemic, it is noteworthy that population-level interventions mainly those targeting the socio-economic determinants of health would have the most impactful and cost-effective outcomes on flattening the pandemic curve 41 , 68 . In the interim, governments and societies have immediately sought refuge in social and lifestyle choices to alleviate the burden of the pandemic and to flatten the uprising infection curves until herd immunity of some sort is reached, i.e., a population-based approach to alleviate the impact. Lockdown, isolation of confirmed cases, quarantine of suspected infected and/or contacted individuals, social distancing, and the simple practice of wearing masks have been among the most effective social measures. Indeed, surges have been related to relaxing of these practices. These actions, together with lifestyle commitments as facemask wearing, frequent hand washing, healthy diet, exercise, and adequate sleep are considered as key tools to reduce the virus’ spread and flatten the epidemiological curve (Fig. 2 ).

figure 2

(1: quarantine/lockdown/curfew; 2: healthy and balanced diet; 3: adequate sleeping; 4: frequent hand washing; 5: cleaning and disinfection of both surfaces and the air; 6: regular domestic exercise; 7: facemask wearing; and 8: social distancing).

Despite big data and digital technologies’ intensive use during this global health crisis, they were quite helpful in the pandemic management (monitoring, surveillance, detection, prediction) and prevention measures 69 , 70 , 71 , 72 . Although some laudable initiatives have developed vaccines against SARS-CoV-2 73 , there is still speculation about their time frame, safety, and effectiveness of future remedies. It seems overly ambitious of the expectations (time frame, levels) for omics and big data to achieve their full potential in health and life sciences in general. Therefore, the current “omics and big data” solution in CHS, which undeniably offers potential benefits, should only be part of a larger and more comprehensive strategy. There needs to be more effort on holistic approaches that include health disparities, social determinants, and lifestyle choices to improve the quality of life. Social and economic systems should rethink cost/benefit analyses to determine the most effective ways to improve healthcare. While omics and digital technologies will have a substantial impact in healthcare and should be pursued, interventions, such as socio-economic determinants, that impact the greater population still will likely have more impact on CHS and must be part of our 21st century healthcare system.

Reporting Summary

Further information on research design is available in the Nature Research Reporting Summary linked to this article.

Data availability

No datasets were generated or analyzed during the current study.

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This research work was funded by Institutional Fund Projects under grant no. (IFPIP: 498-117-1443). Therefore, the authors gratefully acknowledge technical and financial support from the Ministry of Education and King Abdulaziz University, Jeddah, Saudi Arabia.

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Assidi, M., Buhmeida, A. & Budowle, B. Medicine and health of 21st Century: Not just a high biotech-driven solution. npj Genom. Med. 7 , 67 (2022). https://doi.org/10.1038/s41525-022-00336-7

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WEHI spinout aims to revolutionize the development of cancer drugs

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Australia has cemented its role in becoming a major player in the next generation of medicines with the launch of Ternarx – a globally competitive biotechnology company dedicated to finding new treatments for hard-to-treat cancers. 

The WEHI spinout is the first of its kind in Australia dedicated to developing targeted protein degrader medicines and technology, a powerful new tool for destroying disease-causing proteins that cannot be targeted by conventional drugs.

Ternarx is backed by $15 million in funding from the Australian Medical Research Future Fund's (MRFF) Frontier Health and Medical Research initiative and by support from Melbourne-based medical research institute WEHI. 

The company has been officially launched by the Australian Minister for Health and Aged Care, Mark Butler.

While scientists have uncovered many of the drivers of cancer, about 80% of all disease-causing proteins have been considered "undruggable". 

New biotechnology company, Ternarx, aims at changing this statistic through the development of targeted protein degrader (TPD) technology, which is designed to destroy these "undruggable" proteins. 

Unlike conventional drugs that only inhibit the activity of proteins, TPDs can target and destroy disease-causing proteins, completely removing the proteins from the cancer. 

Ternarx is the first company of its kind in Australia, focusing on the development of TPD medicines and technology. 

In 2023, the MRFF's Frontier Health and Medical Research initiative awarded $15 million in funding to establish the Australian Centre for Targeted Therapeutics (ACTT) – a collaboration between experts from WEHI, the Children's Cancer Institute and Monash University. WEHI has now spun out Ternarx to form a globally competitive biotech company and commercialize the ACTT technology. 

It is an honor to officially launch Ternarx, a significant and exciting addition to Australia's growing, high-quality medical and biotech sector. The technology it is pursuing has huge potential to create the next generation of treatments for cancer and other diseases that are currently untreatable.  Ternarx is proof that Australia's health and medical researchers are world leading. With support from the MRFF, our brilliant researchers can turn their ideas into new treatments that have potential to save thousands of lives, not just here but around the world."  Mark Butler, Minister for Health and Aged Care

Unlocking the 'undruggable' 

WEHI director Professor Ken Smith said the landmark initiative would help establish Australia as a leader in this frontier field. 

"With the potential to unlock the 'undruggable', targeted protein degrader technology is one of the most exciting advances in drug discovery and development," Prof Smith said. 

"The establishment of Ternarx is a testament to the wealth of scientific knowledge that exists on our shores, and our ability to remain at the forefront of cutting-edge technologies that have real potential to make a difference to our communities. 

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"To have the greatest impact on human health, we need to continually drive the translation of our discoveries into the new homegrown treatments, diagnostics and devices required to ensure we can live healthier, for longer. 

"We thank the MRFF for continually backing the nation's brightest researchers, helping us to bridge the critical gap between discovery and translation and ensuring that we can confidently tackle our hardest health challenges." 

A new frontier in medicine

Ternarx will initially focus on developing new treatments for neuroblastoma and prostate cancer. 

Neuroblastoma is a childhood cancer that claims more lives of children under five than any other cancer, while prostate cancer is the most commonly diagnosed cancer among Australian men. 

Ternarx CEO, Dr Joanne Boag, said the company will be using and developing novel TPD technology to create new drug candidates and ultimately new ways to treat these cancers and, potentially, other diseases. 

"Neuroblastoma and some forms of prostate cancer urgently need new treatments, as evidenced by poor patient outcomes," Dr Boag said. 

"TPD technology opens up new avenues to attack these and other hard to treat cancers by delivering precision treatment options. 

"This technology could revolutionize treatments for the millions of people in Australia and around the world who continue to live with notoriously difficult-to-treat diseases, including cancer and autoimmune conditions." 

While the initial focus will be cancer, the TPD technology developed by Ternarx has the potential to be applied to a range of disease-causing proteins, including those associated with currently untreatable inflammatory diseases like ulcerative colitis and Crohn's disease, and neurological conditions such as Alzheimer's disease, Huntington's disease and Parkinson's disease. 

Harnessing expertise 

Ternarx will leverage Australia's top cancer experts and research to progress new TPD treatments towards clinical trials, bringing together a core team with deep research expertise as well as biopharmaceutical drug discovery and management experience. 

With further investment, the company has the potential to deliver significant revenue into Australia through co-development and licensing deals, with the TPD market size forecast to grow to USD $3.3 billion by 2030. 

Through its Scientific Advisory Board and other scientific engagements, Ternarx will draw world-leading scientific expertise from Australian scientists Professor John Silke (WEHI), Professor Guillaume Lessene (WEHI), Professor David Komander (WEHI), Professor Michelle Haber (Children's Cancer Institute) and Professor Susan Charman (Monash University). 

The Ternarx management team is composed of high-calibre scientists with experience in the biopharmaceutical sector: 

  • Dr Joanne Boag, CEO 
  • Dianna McKiernan, COO 
  • Dr Nicole Trainor, Lead Chemist 
  • Dr Bernhard Lechtenberg, Structural and Cell Biology Lead (part appointment) 
  • Dr Rebecca Feltham, Target Biology Lead (part appointment) 

The Ternarx Board incorporates senior leaders with a track record in governing and developing innovative biopharmaceutical companies delivering world-class R&D programs: 

  • Dr Victoria Jameson, Business Development lead, WEHI 
  • Dr Amanda Reese, Director, Enterprise, Monash University 
  • Dr Chris Burns, Managing Director and CEO, Amplia Therapeutics 

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  • The Future of Biotech: Innovations and Trends in Science and Research

recent research in medical biotechnology

As a reader interested in healthcare technology, understanding the future of biotech is essential. Biotechnology is constantly shifting with new advancements that have significant enhancements for healthcare. This piece will delve into the most promising innovations and trends in biotech that you need to know about.

Emerging Trends in Biotechnology

One of the most notable trends in biotech is the rise of personalized medicine. This approach tailors medical treatment to individual characteristics, such as genetic makeup, which can lead to more effective and targeted therapies. This trend represents a significant opportunity  for biotech startups  to develop innovative solutions that cater specifically to individual patient needs. 

Advancements in genomic sequencing technologies are also making personalized medicine more accessible and affordable than ever before.

Innovations Driving Biotech Forward

Another key innovation in the field is the development of  CRISPR-Cas9 technology  which is gene-editing tool. Allowing scientists to modify DNA with unprecedented precision, it has opened up the possibility for treating genetic disorders and improving agricultural productivity. In addition to CRISPR, other gene-editing techniques are also being explored, expanding the toolkit available to researchers.

Additionally, advancements in artificial intelligence (AI) are revolutionizing biotech research. AI algorithms can analyze vast amounts of biological data quickly, identifying patterns that would be impossible for humans to detect. This capability accelerates the pace of discovery and enables more accurate predictions about disease progression and treatment outcomes.

The Role of Big Data in Biotech Research

Big data is playing an increasingly important role in biotechnology research. By aggregating and analyzing large datasets from various sources, researchers can gain deeper insights into complex biological processes. This data-driven approach is particularly valuable in areas such as drug discovery and development, where it can help identify potential drug candidates more efficiently.

What's more, integrating big data with other technologies like AI and machine learning enhances its impact. These tools can sift through enormous datasets to uncover hidden correlations and generate hypotheses that guide further experimentation. Consequently, the research process becomes more streamlined and cost-effective.

The increasing availability of large-scale biological datasets, such as genomic and proteomic data, is transforming the way biotech research is conducted. By harnessing the power of big data, researchers can uncover novel insights and patterns that were previously hidden. This data-driven approach enables a more comprehensive understanding of complex biological systems, facilitating the development of innovative therapies and diagnostic tools.

The Impact of Regulatory Changes on Biotech

Regulatory frameworks are evolving alongside technological advancements in biotechnology. Governments worldwide are updating their regulations to keep pace with new developments, ensuring that safety and ethical considerations are addressed. These changes can have significant implications for biotech companies, influencing their research priorities and market strategies.

For instance, recent updates to clinical trial regulations aim to make trials more efficient while maintaining rigorous safety standards. These changes can expedite the approval process for new treatments, benefiting both patients and companies. Staying informed about regulatory trends is crucial for navigating the complex landscape of biotech innovation.

In addition to clinical trial regulations, intellectual property laws also have a significant impact on the biotech industry. Patents play a crucial role in protecting the investments made by companies in developing new technologies and treatments. However, the patent landscape in biotech can be complex, with disputes over patent ownership and infringement being common. Navigating this landscape requires a deep understanding of the legal and regulatory frameworks governing intellectual property in the biotech sector.

In Conclusion

Healthcare is benefitting from biotechnology and the scientific innovations and enhanced research methods it has introduced. As biotech continues to enhance the medical world with new discoveries, the developments are proving key for the health sector and the rise of personalized medicine is helping people everywhere.

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An Introduction to Biotechnology

Varsha gupta.

5 Institute of Biosciences and Biotechnology, Chhatrapati Shahu Ji Maharaj University, Kanpur, UP India

Manjistha Sengupta

6 George Washington University, Washington DC, USA

Jaya Prakash

7 Orthopaedics Unit, Community Health Centre, Kanpur, UP India

Baishnab Charan Tripathy

8 School of Life sciences, Jawaharlal Nehru University, New Delhi, India

Biotechnology is multidisciplinary field which has major impact on our lives. The technology is known since years which involves working with cells or cell-derived molecules for various applications. It has wide range of uses and is termed “technology of hope” which impact human health, well being of other life forms and our environment. It has revolutionized diagnostics and therapeutics; however, the major challenges to the human beings have been threats posed by deadly virus infections as avian flu, Chikungunya, Ebola, Influenza A, SARS, West Nile, and the latest Zika virus. Personalized medicine is increasingly recognized in healthcare system. In this chapter, the readers would understand the applications of biotechnology in human health care system. It has also impacted the environment which is loaded by toxic compounds due to human industrialization and urbanization. Bioremediation process utilizes use of natural or recombinant organisms for the cleanup of environmental toxic pollutants. The development of insect and pest resistant crops and herbicide tolerant crops has greatly reduced the environmental load of toxic insecticides and pesticides. The increase in crop productivity for solving world food and feed problem is addressed in agricultural biotechnology. The technological advancements have focused on development of alternate, renewable, and sustainable energy sources for production of biofuels. Marine biotechnology explores the products which can be obtained from aquatic organisms. As with every research area, the field of biotechnology is associated with many ethical issues and unseen fears. These are important in defining laws governing the feasibility and approval for the conduct of particular research.

Introduction

The term “ biotechnology” was coined by a Hungarian engineer Karl Ereky, in 1919, to refer to the science and methods that permit products to be produced from raw materials with the aid of living organisms. Biotechnology is a diverse field which involves either working with living cells or using molecules derived from them for applications oriented toward human welfare using varied types of tools and technologies. It is an amalgamation of biological science with engineering whereby living organisms or cells or parts are used for production of products and services. The main subfields of biotechnology are medical (red) biotechnology, agricultural (green) biotechnology, industrial (white) biotechnology, marine (blue) biotechnology, food biotechnology, and environmental biotechnology (Fig. 1.1 .). In this chapter the readers will understand the potential applications of biotechnology in several fields like production of medicines; diagnostics; therapeutics like monoclonal antibodies, stem cells, and gene therapy; agricultural biotechnology; pollution control ( bioremediation); industrial and marine biotechnology; and biomaterials, as well as the ethical and safety issues associated with some of the products.

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Major applications of biotechnology in different areas and some of their important products

The biotechnology came into being centuries ago when plants and animals began to be selectively bred and microorganisms were used to make beer, wine, cheese, and bread. However, the field gradually evolved, and presently it is the use or manipulation of living organisms to produce beneficiary substances which may have medical, agricultural, and/or industrial utilization. Conventional biotechnology is referred to as the technique that makes use of living organism for specific purposes as bread/cheese making, whereas modern biotechnology deals with the technique that makes use of cellular molecules like DNA, monoclonal antibodies, biologics, etc. Before we go into technical advances of DNA and thus recombinant DNA technology, let us have the basic understanding about DNA and its function.

The foundation of biotechnology was laid down after the discovery of structure of DNA in the early 1950s. The hereditary material is deoxyribonucleic acid (DNA) which contains all the information that dictates each and every step of an individual’s life. The DNA consists of deoxyribose sugar, phosphate, and four nitrogenous bases (adenine, guanine, cytosine, and thymine). The base and sugar collectively form nucleoside, while base, sugar, and phosphate form nucleotide (Fig. 1.2 ). These are arranged in particular orientation on DNA called order or sequence and contain information to express them in the form of protein. DNA has double helical structure, with two strands being complimentary and antiparallel to each other, in which A on one strand base pairs with T and G base pairs with C with two and three bonds, respectively. DNA is the long but compact molecule which is nicely packaged in our nucleus. The DNA is capable of making more copies like itself with the information present in it, as order or sequence of bases. This is called DNA replication. When the cell divides into two, the DNA also replicates and divides equally into two. The process of DNA replication is shown in Fig. 1.3 , highlighting important steps.

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The double helical structure of DNA where both strands are running in opposite direction. Elongation of the chain occurs due to formation of phosphodiester bond between phosphate at 5′ and hydroxyl group of sugar at 3′ of the adjacent sugar of the nucleotide in 5–3′ direction. The sugar is attached to the base. Bases are of four kinds: adenine ( A ), guanine ( G ) (purines), thymine ( T ), and cytosine ( C ) (pyrimidines). Adenine base pairs with two hydrogen bonds with thymine on the opposite antiparallel strand and guanine base pairs with three hydrogen bonds with cytosine present on the opposite antiparallel strand

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The process of DNA replication. The DNA is densely packed and packaged in the chromosomes. The process requires the action of several factors and enzymes. DNA helicase unwinds the double helix. Topoisomerase relaxes DNA from its super coiled nature. Single-strand binding proteins bind to single-stranded open DNA and prevent its reannealing and maintains strand separation. DNA polymerase is an enzyme which builds a new complimentary DNA strand and has proofreading activity. DNA clamp is a protein which prevents dissociation of DNA polymerase. Primase provides a short RNA sequence for DNA polymerase to begin synthesis. DNA ligase reanneals and joins the Okazaki fragments of the lagging strand. DNA duplication follows semiconservative replication, where each strand serves as template which leads to the production of two complimentary strands. In the newly formed DNA, one strand is old and the other one is new (semiconservative replication). DNA polymerase can extend existing short DNA or RNA strand which is paired to template strand and is called primer. Primer is required as DNA polymerase cannot start the synthesis directly. DNA polymerase is capable of proofreading, that is, correction of wrongly incorporated nucleotide. One strand is replicated continuously with single primer, and it is called as leading strand. Other strand is discontinuous and requires the addition of several primers. The extension is done in the form of short fragments called as Okazaki fragments. The gaps are sealed by DNA ligase. Replication always occurs in 5′–3′ direction

DNA contains whole information for the working of the cell. The part of the DNA which has information to dictate the biosynthesis of a polypeptide is called a “gene.” The arrangement or order of nucleotides determines the kind of proteins which we produce. Each gene is responsible for coding a functional polypeptide. The genes have the information to make a complimentary copy of mRNA. The information of DNA which makes RNA in turn helps cells to incorporate amino acids according to arrangement of letters for making many kinds of proteins. These letters are transcribed into mRNA in the form of triplet codon, where each codon specifies one particular amino acid. The polypeptide is thus made by adding respective amino acids according to the instructions present on RNA. Therefore, the arrangement of four bases (adenine, guanine, cytosine, and thymine) dictates the information to add any of the 20 amino acids to make all the proteins in all the living organisms. Few genes need to be expressed continuously, as their products are required by the cell, and these are known as “constitutive genes.” They are responsible for basic housekeeping functions of the cells. However, depending upon the physiological demand and cell’s requirement at a particular time, some genes are active and some are inactive, that is, they do not code for any protein. The information contained in the DNA is used to make mRNA in the process of “ transcription” (factors shown in Table 1.1 ). The information of mRNA is used in the process of “ translation” for production of protein. Transcription occurs in the nucleus and translation in the cytoplasm of the cell. In translation several initiation factors help in the assembly of mRNA with 40S ribosome and prevent binding of both ribosomal subunits; they also associate with cap and poly(A) tail. Several elongation factors play an important role in chain elongation. Though each cell of the body has the same genetic makeup, but each is specialized to perform unique functions, the activation and expression of genes is different in each cell. Thus, one type of cells can express some of its genes at one time and may not express the same genes some other time. This is called “temporal regulation” of the gene. In the body different cells express different genes and thus different proteins. For example, liver cell and lymphocyte, would express different genes. This is known as spatial regulation of the gene. Therefore, in the cells of the body, the activation of genes is under spatial regulation (cells present at different locations and different organs produce different proteins) and temporal regulation (same cells produce different proteins at different times). The proteins are formed by the information contained in the DNA and perform a variety of cellular functions. The proteins may be structural (responsible for cell shape and size), or they may be functional like enzymes, signaling intermediates, regulatory proteins, and defense system proteins. However, any kind of genetic defect results in defective protein or alters protein folding which can compromise the functioning of the body and is responsible for the diseases. Figure 1.4 shows the outline of the process of transcription and translation with important steps.

Factors involved in transcription process

Eukaryotic transcription
Transcription factor (TF)Functions
TFIIDTATA bindingIt recognizes
Protein (TBP)TATA box
Subunit
TBP associatedRegulate DNA
FactorsBinding by TBP
TFIIBRecognizes TFIIB recognition elements (BRE); positions RNA polymerase (RNA pol)
TFIIFStabilizes RNA pol; attracts TFIIE and TFIIF
TFIIERegulates TFIIH
TFIIHUnwinds DNA at transcription start point; releases RNA polymerase from promoter

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It shows the process of transcription and translation. Transcription occurs in the nucleus and requires the usage of three polymerase enzymes. RNApol I for rRNA, pol II for mRNA, and pol III for both rRNA and tRNA. RNApol II initiates the process by associating itself with seven transcription factors, TFIIA, TFIIB, TFIID, TFIIE, TFIIH, and TFIIJ. After the synthesis, preRNA transcript undergoes processing to form mRNA by removal of introns by splicing and polyadenylation and capping. Protein synthesis is initiated by formation of ribosome and initiator tRNA complex to initiation codon (AUG) of mRNA and involves 11 factors. Chain elongation occurs after sequential addition of amino acids by formation of peptide bonds. Then polypeptide can fold or conjugate itself to other biomolecules and may undergo posttranslational modifications as glycosylation or phosphorylation to perform its biological functions

The biotechnological tools are employed toward modification of the gene for gain of function or loss of function of the protein. The technique of removing, adding, or modifying genes in the genome or chromosomes of an organism to bring about the changes in the protein information is called genetic engineering or recombinant DNA technology (Fig. 1.5 ). DNA recombination made possible the sequencing of the human genome and laid the foundation for the nascent fields of bioinformatics, nanomedicine, and individualized therapy. Multicellular organisms like cows, goats, sheep, rats, corn, potato, and tobacco plants have been genetically engineered to produce substances medically useful to humans. Genetic engineering has revolutionized medicine, enabling mass production of safe, pure, more effective versions of biochemicals that the human body produces naturally [ 20 – 22 ].

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The process of recombinant DNA technology. The gene of interest from human nucleus is isolated and cloned in a plasmid vector. The gene is ligated with the help of DNA ligase. The vector is transformed into a bacterial host. After appropriate selections, the gene is amplified when bacteria multiply or the gene can be sequenced or the gene can be expressed to produce protein

The technological advancements have resulted in (1) many biopharmaceuticals and vaccines, (2) new and specific ways to diagnose, (3) increasing the productivity and introduction of quality traits in agricultural crops, (4) the ways to tackle the pollutants efficiently for sustainable environmental practices, (5) helped the forensic experts by DNA fingerprinting and profiling, (6) fermentation technology for production of industrially important products. The list is very long with tremendous advancements and products which have boosted the economy of biotechnology sector worldwide [ 16 ]. The biotechnology industry and the products are regulated by various government organizations such as the US Food and Drug Administration (FDA), the Environmental Protection Agency (EPA), and the US Department of Agriculture (USDA).

Medical Biotechnology

This fieldof biotechnology has many applications and is involved in production of recombinant pharmaceuticals, tissue engineering products, regenerative medicines such as stem cell and gene therapy, and many more biotechnology products for better human life (Fig. 1.6 ). Biotechnological tools produce purified bio-therapeutic agents on industrial scales. These include both novel agents and agents formerly available only in small quantities. Crude vaccines were used in antiquity in China, India, and Persia. For example, vaccination with scabs that contained the smallpox virus was a practice in the East for centuries. In 1798 English country doctor Edward Jenner demonstrated that inoculation with pus from sores due to infection by a related cowpox virus could prevent smallpox far less dangerously. It marked the beginning of vaccination. Humans have been benefited incalculably from the implementation of vaccination programs.

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Various applications of medical biotechnology

Tremendous progress has been made since the early recombinant DNA technology (RDT) experiments from which the lively—and highly profitable—biotechnology industry emerged. RDT has fomented multiple revolutions in medicine. Safe and improved drugs, accelerated drug discovery, better diagnostic and quick methods for detecting an infection either active or latent, development of new and safe vaccines, and completely novel classes of therapeutics and other medical applications are added feathers in its cap. The technology has revolutionized understanding of diseases as diverse as cystic fibrosis and cancer. Pharmaceutical biotechnology being one of the important sectors involves using animals or hybrids of tumor cells or leukocytes or cells ( prokaryotic, mammalian) to produce safer, more efficacious, and cost-effective versions of conventionally produced biopharmaceuticals. The launch of the new biopharmaceutical or drug requires screening and development. Mice were widely used as research animals for screening. However, in the wake of animal protection, animal cell culture offers accurate and inexpensive source of cells for drug screening and efficacy testing. Pharmaceutical biotechnology’s greatest potential lies in gene therapy and stem cell-based therapy. The underlying cause of defect of many inherited diseases is now located and characterized. Gene therapy is the insertion of the functional gene in place of defective gene into cells to prevent, control, or cure disease. It also involves addition of genes for pro-drug or cytokines to eliminate or suppress the growth of the tumors in cancer treatment.

But the progress so far is viewed by many scientists as only a beginning. They believe that, in the not-so-distant future, the refinement of “targeted therapies” should dramatically improve drug safety and efficacy. The development of predictive technologies may lead to a new era in disease prevention, particularly in some of the world’s rapidly developing economies. Yet the risks cannot be ignored as new developments and discoveries pose new questions, particularly in areas as gene therapy, the ethics of stem cell research, and the misuse of genomic information.

Many bio-therapeutic agents in clinical use are biotech pharmaceuticals. Insulin was among the earliest recombinant drugs. Canadian physiologists Frederick Banting and Charles Best discovered and isolated insulin in 1921. In that time many patients diagnosed with diabetes died within a few years. In the mid-1960s, several groups reported synthesizing the hormone.

The first “bioengineered” drug, a recombinant form of human insulin, was approved by the US Food and Drug Administration (FDA) in 1982. Until then, insulin was obtained from a limited supply of beef or pork pancreas tissue. By inserting the human gene for insulininto bacteria, scientists were able to achieve lifesaving insulinproduction in large quantities. In the near future, patients with diabetes may be able to inhale insulin, eliminating the need for injections. Inhaled insulinproducts like Exubera® were approved by the USFDA; however, it was pulled out and other products like Technosphere® insulin (Afrezza®) are under investigation. They may provide relief from prandial insulin. Afrezza consists of a pre-meal insulinpowder loaded into a cartridge for oral inhalation.

Technosphere technology: The technology allows administration of therapeutics through pulmonary route which otherwise were required to be given as injections. These formulations have broad spectrum of physicochemical characteristics and are prepared with a diverse assortment of drugs with protein or small molecule which may be hydrobhobic or hydrophilic or anionic or cationic in nature. The technology can have its applicability not only through pulmonary route but also for other routes of administration including local lung delivery.

The first recombinant vaccine, approved in 1986, was produced by cloning a gene fragment from the hepatitis B virus into yeast (Merck’s Recombivax HB). The fragment was translated by the yeast’s genetic machinery into an antigenic protein. This was present on the surface of the virus that stimulates the immune response. This avoided the need to extract the antigen from the serum of people infected with hepatitis B.

The Food and Drug administration (FDA) approved more biotech drugs in 1997 than in the previous several years combined. The FDA has approved many recombinant drugs for human health conditions. These include AIDS, anemia, cancers (Kaposi’s sarcoma, leukemia, and colorectal, kidney, and ovarian cancers), certain circulatory problems, certain hereditary disorders (cystic fibrosis, familial hypercholesterolemia, Gaucher’s disease, hemophilia A, severe combined immunodeficiency disease, and Turner’s syndrome), diabetic foot ulcers, diphtheria, genital warts, hepatitis B, hepatitis C, human growth hormone deficiency, and multiple sclerosis. Today there are more than 100 recombinant drugs and vaccines. Because of their efficiency, safety, and relatively low cost, molecular diagnostic tests and recombinant vaccines may have particular relevance for combating long-standing diseases of developing countries, including leishmaniasis (a tropical infection causing fever and lesions) and malaria.

Stem cell research is very promising and holds tremendous potential to treat neurodegenerative disorders, spinal cord injuries, and other conditions related to organ or tissue loss.

DNA analysis is another powerful technique which compares DNA pattern [ 14 ] after performing RFLP and probing it by minisatellite repeat sequence between two or more individuals. Its modification, DNA profiling (process of matching the DNA profiles for STS markers in two or more individuals; see chapter 18), which utilizes multilocus PCR analysis of DNA of suspect and victims, is very powerful and is useful in criminal investigation, paternity disputes, and so many other legal issues. The analysis is very useful in criminal investigations and involves evaluation of DNA from samples of the hair, body fluids, or skin at a crime scene and comparison of these with those obtained from the suspects.

Improved Diagnostic and Therapeutic Capabilities

The sequencing of the human genome in 2003, has given scientists an incredibly rich “parts list” with which to better understand why and how disease happens. It has given added power to gene expression profiling, a method of monitoring expression of thousands of genes simultaneously on a glass slide called a microarray. This technique can predict the aggressiveness of cancer.

The development of monoclonal antibodies in 1975 led to a medical revolution. The body normally produces a wide range of antibodies—the immune system proteins—that defend our body and eliminate microorganisms and other foreign invaders. By fusing antibody-producing cells with myeloma cells, scientists were able to generate antibodies that would, like “magic bullets,” bind with specific targets including unique markers, called antigenic determinants ( epitopes), on the surfaces of inflammatory cells. When tagged with radioisotopes or other contrast agents, monoclonal antibodies can help in detecting the location of cancer cells, thereby improving the precision of surgery and radiation therapy and showing—within 48 h—whether a tumor is responding to chemotherapy.

The polymerase chain reaction, a method for amplifying tiny bits of DNA first described in the mid-1980s, has been crucial to the development of blood tests that can quickly determine exposure to the human immunodeficiency virus (HIV). Genetic testing currently is available for many rare monogenic disorders, such as hemophilia, Duchenne muscular dystrophy, sickle cell anemia, thalassemia, etc.

Another rapidly developing field is proteomics, which deals with analysis and identification of proteins. The analysis is done by two-dimensional gel electrophoresis of the sample and then performing mass spectrometric analysis for each individual protein. The technique may be helpful in detecting the disease-associated protein in the biological sample. They may indicate early signs of disease, even before symptoms appear. One such marker is C-reactive protein, an indicator of inflammatory changes in blood vessel walls that presage atherosclerosis.

Nanomedicine is a rapidly moving field. Scientists are developing a wide variety of nanoparticles and nanodevices, scarcely a millionth of an inch in diameter, to improve detection of cancer, boost immune responses, repair damaged tissue, and thwart atherosclerosis. The FDA has approved a paclitaxel albumin-stabilized nanoparticle formulation (Abraxane® for injectable suspension, made by Abraxis BioScience) for the treatment of metastatic adenocarcinoma of the pancreas. Nanoparticles are being explored in heart patients in the USA as a way to keep their heart arteries open following angioplasty.

Therapeutic proteins are those, which can replace the patients naturally occurring proteins, when levels of the natural proteins are low or absent due to the disease. High-throughput screening, conducted with sophisticated robotic and computer technologies, enables scientists to test tens of thousands of small molecules in a single day for their ability to bind to or modulate the activity of a “target,” such as a receptor for a neurotransmitter in the brain. The goal is to improve the speed and accuracy of therapeutic protein or potential drug discovery while lowering the cost and improving the safety of pharmaceuticals that make it to market.

Many of the molecules utilized for detection also have therapeutic potential too, for example, monoclonal antibodies. The monoclonal antibodies are approved for the treatment of many diseases as cancer, multiple sclerosis, and rheumatoid arthritis. They are currently being tested in patients as potential treatments for asthma, Crohn’s disease, and muscular dystrophy. As the antibodies may be efficiently targeted against a particular cell surface marker, thus they are used to deliver a lethal dose of toxic drug to cancer cells, avoiding collateral damage to nearby normal tissues.

Agricultural Biotechnology

The manhas made tremendous progress in crop improvement in terms of yield; still the ultimate production of crop is less than their full genetic potential. There are many reasons like environmental stresses (weather condition as rain, cold, frost), diseases, pests, and many other factors which reduce the ultimate desired yield affecting crop productivity. The efforts are going on to design crops which may be grown irrespective of their season or can be grown in frost or drought conditions for maximum utilization of land, which would ultimately affect crop productivity [ 24 ]. Agricultural biotechnology aims to introduce sustainable agriculturalpractices with best yield potential and minimal adverse effects on environment (Fig. 1.7 ). For example, combating pests was a major challenge. Thus, the gene from bacterium , the Bt gene, that functions as insect-resistant gene when inserted into crop plants like cotton, corn, and soybean helps prevent the invasion of pathogen, and the tool is called . This management is helpful in reducing usage of potentially dangerous pesticides on the crop. Not only the minimal or low usage of pesticides is beneficial for the crop but also the load of the polluting pesticides on environment is greatly reduced [ 24 ].

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Various applications of agricultural biotechnology

Resistance to Infectious Agents Through Genetic Engineering

  • The gene comes from the soil bacterium .
  • The gene produces crystal proteins called Cry proteins. More than 100 different variants of the Bt toxins have been identified which have different specificity to target insect lepidoptera. For eg., CryIa for butterflies and CRYIII for beetles.
  • These Cry proteins are toxic to larvae of insects like tobacco budworm, armyworm, and beetles.
  • The Cry proteins exist as an inactive protoxins.
  • These are converted into active toxin in alkaline pH of the gut upon solubilization when ingested by the insect.
  • After the toxin is activated, it binds to the surface of epithelial cells of midgut and creates pores causing swelling and lysis of cells leading to the death of the insect (larva).
  • The genes (cry genes) encoding this protein are isolated from the bacterium and incorporated into several crop plants like cotton, tomato, corn, rice, and soybean.

The proteins encoded by the following cry genes control the pest given against them:

  • Cry I Ac and cry II Ab control cotton bollworms.
  • Cry I Ab controls corn borer.
  • Cry III Ab controls Colorado potato beetle.
  • Cry III Bb controls corn rootworm.
  • A nematode infects tobacco plants and reduces their yield.
  • The specific genes (in the form of cDNA) from the parasite are introduced into the plant using -mediated transformation.
  • The genes are introduced in such a way that both sense/coding RNA and antisense RNA (complimentary to the sense/coding RNA) are produced.
  • Since these two RNAs are complementary, they form a double-stranded RNA (ds RNA).
  • This neutralizes the specific RNA of the nematode, by a process called RNA – interference.
  • As a result, the parasite cannot multiply in the transgenic host, and the transgenic plantis protected from the pest.

These resistant crops result in reduced application of pesticides. The yield is high without the pathogen infestations and insecticides. This also helps to reduce load of these toxic chemicals in the environment.

The transformation techniques and their applications are being utilized to develop rice, cassava, and tomato, free of viral diseases by “International Laboratory for Tropical Agricultural Biotechnology” (ILTAB). ILTAB in 1995 reported the first transfer of a resistance gene from a wild-type species of rice to a susceptible cultivated rice variety. The transferred gene expressed and imparted resistance to crop-destroying bacterium Xanthomonas oryzae . The resistant gene was transferred into susceptible rice varieties that are cultivated on more than 24 million hectares around the world [ 6 ].

The recombinant DNA technology reduces the time between the identification of a particular gene to its application for betterment of crops by skipping the labor-intensive and time-consuming conventional breeding [ 3 ]. For example, the alteration of known gene in plant for the improvement of yield or tolerance to adverse environmental conditions or resistance to insect in one generation and its inheritance to further generations. Plant cell and tissue culture techniques are one of the applications where virus-free plants can be grown and multiplied irrespective of their season on large scale (micropropogation), raising haploids, or embryo rescue. It also opens an opportunity to cross two manipulated varieties or two incompatible varieties (protoplast culture) for obtaining best variety for cultivation.

With the help of technology, new, improved, and safe agricultural products may emerge which would be helpful for maintaining contamination-free environment. Biotechnology has the potential to produce:

  • High crop yields [ 4 ]
  • Crops are engineered to have desirable nutrients and better taste (e.g., tomatoes and other edible crops with increased levels of vitamin C, vitamin E, and/or beta-carotene protect against the risk of some prevalent chronic diseases and rice with increased iron levels protects against anemia)
  • Insect- and disease-resistant plants
  • Genetic modification avoids nonselective changes
  • Longer shelf life of fruits and vegetables

The potential of biotechnology may contribute to increasing agricultural, food, and feed production, protecting the environment, mitigating pollution, sustaining agricultural practices, and improving human and animal health. Some agricultural crops as corn and marine organisms can be potential alternative for biofuel production. The by-products of the process may be processed to produce other chemical feedstocks for various products. It is estimated that the world’s chemical and fuel demand could be supplied by such renewable resources in the first half of the next century [ 5 ].

Food Biotechnology

Food biotechnology is an emerging field, which can increase the production of food, improving its nutritional content and improving the taste of the food. The food is safe and beneficial as it needs fewer pesticides and insecticides. The technology aims to produce foods which have more flavors, contain more vitamins and minerals, and absorb less fat when cooked. Food biotechnology may remove allergens and toxic components from foods, for their better utility [ 6 , 7 ].

Environmental Biotechnology

Environmental biotechnology grossly deals with maintenanceof environment, which is pollution-free, the water is contamination-free, and the atmosphere is free of toxic gases. Thus, it deals with waste treatment, monitoring of environmental changes, and pollution prevention. Bioremediation in which utilization of higher living organisms (plants: phytoremediation) or certain microbial species for decontamination or conversion of harmful products is done is the main application of environmental biotechnology. The enzyme bioreactors are also being developed which would pretreat some industrial and food waste components and allow their removal through the sewage system rather than through solid waste disposal mechanisms. The production of biofuel from waste can solve the fuel crisis (biogas). Microbes may be engineered to produce enzymes required for conversion of plant and vegetable materials into building blocks for biodegradable plastics. In some cases, the by-products of the pollution-fighting microorganisms are themselves useful. For example, methane can be derived from a form of bacteria that degrades sulfur liquor, a waste product of paper manufacturing. This methane thus obtained is used as a fuel or in other industrial processes. Insect- and pest-resistant crops have reduced the use and environmental load of insecticides and pesticides. Insect-protected crops allow for less potential exposure of farmers and groundwater to chemical residues while providing farmers with season-long control.

Industrial Biotechnology

The utilizationof biotechnological tools (bioprocessing) for the manufacturing of biotechnology-derived products (fuels, plastics, enzymes, chemicals, and many more compounds) on industrial scale is industrial biotechnology. The aim is to develop newer industrial manufacturing processes and products, which are economical and better than preexisting ones with minimal environmental impact. In industrial biotechnology, (1) microorganisms are being explored for producing material goods like fermentation products as cheese; (2) biorefineries where oils, sugars, and biomass may be converted into biofuels, bioplastics, and biopolymers; (3) and value-added chemicals from biomass. The utilization of modern techniques can improve the efficiency and reduces the environmental impacts of industrial processes like textile, paper, pulp, and chemical manufacturing. For example, development and usage of biocatalysts, such as enzymes, to synthesize chemicals and development of antibiotics and better tasting liquors and their usage in food industry have provided safe and effective processing for sustainable productions. Biotechnological tools in the textile industry are utilized for the finishing of fabrics and garments. Biotechnology also produces spider silk and biotech-derived cotton that is warmer and stronger and has improved dye uptake and retention, enhanced absorbency, and wrinkle and shrink resistance.

Biofuels may be derived from photosynthetic organisms, which capture solar energy, transform it in other products like carbohydrates and oils, and store them. Different plants can be used for fuel production:

  • Bioethanol can be obtained from sugar (as sugarcane or sugar beet) or starch (like corn or maize). These are fermented to produce ethanol, a liquid fuel commonly used for transportation.
  • Biodiesel can be obtained from natural oils from plants like oil palm, soybean, or algae. They can be burned directly in a diesel engine or a furnace, or blended with petroleum, to produce fuels such as biodiesel.
  • Wood and its by-products can be converted into liquid biofuels, such as methanol or ethanol, or into wood gas. Wood can also be burned as solid fuel, like the irewood.

In these kinds of biological reaction, there are many renewable chemicals of economic importance coproduced as side streams of bioenergy and biofuels as levulinic acid, itaconic acid, and sorbitol. These have tremendous economic potential and their fruitful usage would depend upon the collaboration for research and development between the government and the private sector.

Enzyme Production

The enzymeshave big commercial and industrial significance. They have wide applications in food industry, leather industry, pharmaceuticals, chemicals, detergents, and research. In detergents the alkaline protease, subtilisin (from Bacillus subtilis ), was used by Novo Industries, Denmark. The production of enzymes is an important industrial application with world market of approximately 5 billion dollars. The enzymes can be obtained from animals, plants, or microorganisms. The production from microorganisms is preferred as they are easy to maintain in culture with simple media requirements and easy scale-up. The important enzymes for the industrial applications are in food industry, human application, and research. A few animal enzymes are also important as a group of proteolytic enzymes, for example, plasminogen activators, which act on inactive plasminogen and activate it to plasmin, which destroys fibrin network of blood clot. Some of the plasminogen activators are urokinase and tissue plasminogen activators (t-PA). Urokinase (from urine) is difficult to obtain in ample quantity; thus, t-PA is obtained from cells grown in culture medium. Streptokinase (bacterial enzyme) is also a plasminogen activator but is nonspecific and immunogenic.

Enzyme engineering is also being tried where modifications of specific amino acid residue are done for improving the enzyme properties. One of the enzymes chymosin (rennin) coagulates milk for cheese manufacturing.

The enzymes can be produced by culturing cells, growing them with appropriate substrates in culture conditions. After optimum time the enzymes may be obtained by cell disruption (enzymatic/freeze–thaw/osmotic shock) followed by preparative steps (centrifugation, filtration), purification, and analysis. The product is then packaged and ultimately launched in the market.

After their production, they can be immobilized on large range of materials (agar, cellulose, porous glass, or porous alumina) for subsequent reuse. Some of the important industrial enzymes are α-amylase (used for starch hydrolysis), amyloglucosidase (dextrin hydrolysis), β-galactosidase (lactose hydrolysis), aminoacylase (hydrolysis of acylated L-amino acids), glucose oxidase (oxidation of glucose), and luciferase (bioluminescence). Some of the medically important enzymes are urokinase and t-PA for blood clot removal and L-asparaginase for removal of L-asparagine essential for tumor growth and thus used for cancer chemotherapy in leukemia.

Exploring Algae for Production of Biofuels

The energyrequirement of present population is increasing and gradually fossil fuels are rapidly depleting. Thus, renewable energy sources like solar energy and wind-, hydro-, and biomass-based energy are being explored worldwide. One of the feedstocks may be microalgae, which are fast-growing, photosynthetic organisms requiring carbon dioxide, some nutrients, and water for its growth. They produce large amount of lipids and carbohydrates, which can be processed into different biofuels and commercially important coproducts. The production of biofuels using algal biomass is advantageous as they (1) can grow throughout the year and thus their productivity is higher than other oil seed crops, (2) have high tolerance to high carbon dioxide content, (3) utilize less water, (4) do not require herbicides or pesticides with high growth potential (waste water can be utilized for algal cultivation), (5) can sustain harsh atmospheric conditions, and (6) do not interfere with productivity of conventional crops as they do not require agricultural land. The production of various biofuels from algae is schematically represented in Fig. 1.8 .

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Object name is 346823_1_En_1_Fig8_HTML.jpg

Different biofuel productions by using microalgae. The algae use sunlight, CO2, water, and some nutrients

Algae can serve as potential source for biofuel production; however, biomass production is low. The production has certain limitations, as cultivation cost is high with requirement of high energy [ 1 ].

Marine or Aquatic Biotechnology

Marine or aquatic biotechnology also referred to as “blue biotechnology” deals with exploring and utilizing the marine resources of the world. Aquatic or marine life has been intriguing and a source of livelihood for many since years. As major part of earth is acquired by water, thus nearly 75–80 % types of life forms exist in oceans and aquatic systems. It studies the wide diversity found in the structure and physiology of marine organisms. They are unique in their own ways and lack their equivalent on land. These organisms have been explored and utilized for numerous applications as searching new treatment for cancer or exploring other marine resources, because of which the field is gradually gaining momentum and economic opportunities [ 19 ]. The global economic benefits are estimated to be very high. The field aims to:

  • Fulfill the increasing food supply needs
  • Identify and isolate important compounds which may benefit health of humans
  • Manipulate the existing traits in sea animals for their improvement
  • Protect marine ecosystem and gain knowledge about the geochemical processes occurring in oceans

Some of the major applications are discussed:

  • Aquaculture: Aquaculture refers to the growth of aquatic organisms in culture condition for commercial purposes. These animals may be shellfish, finfish, and many others. Mariculture refers to the cultivation of marine animals. Their main applications are in food, food ingredients, pharmaceuticals, and fuels, the products are in high demand, and various industries are in aquaculture business, for example, crawfish farming (Louisiana), catfish industry (Alabama and Mississippi Delta), and trout farming (Idaho and West Virginia).
  • Transgenic species of salmon with growth hormone gene has accelerated growth of salmons.
  • Molt-inhibiting (MIH) from blue crabs leads to soft-shelled crab.
  • : Anovel protein antifreeze protein (AFP) was identified. AFPs were isolated from Northern cod (bottom-dwelling fish) living at the Eastern Canada coast and teleosts living in extremely cold weather of Antarctica. AFPs have been isolated from Osmerus mordax (smelt), Clupea harengus (herring), Pleuronectes americanus (winter flounder), and many others. Due to antifreeze properties (lowering the minimal freezing temperature by 2–3 °C), the gene has potential for raising plants which are cold tolerant (e.g., tomatoes).
  • Medicinal applications : For osteoporosis, salmon calcitonin (calcitonin is thyroid hormone promoting calcium uptake and bone calcification) with 20 times higher bioactivity is available as injection and nasal spray.
  • Hydroxyapatite ( HA ): Obtained from coral reefs and is an important component of bone and cartilage matrix. Its implants are prepared by Interpore Internationals which may be used for filling gaps in fractured bones.

Many anti-inflammatory, analgesic, anticancerous compounds have been identified from sea organisms which can have tremendous potential for human health.

Tetrodotoxin (TTX) is the most toxic poison (10,000 times more lethal than cyanide) produced by Japanese pufferfish or blowfish ( Fugu rubripes ). TTX is being used to study and understand its effect on sodium channels which can help guide the development of drugs with anesthetic and analgesic properties.

Other Products

  • Taq polymerase from Thermus aquaticus which is used in PCR reactions and obtained from hot spring Archaea.
  • Collagenase (protease) obtained from Vibrio is used in tissue engineering and culturing.

Transgenic Animals and Plants

In the early1980s, inserting DNA from humans into mice and other animals became possible. The animals and plants which have foreign gene in each of their cells are referred to as transgenic organisms and the inserted gene as transgene. Expression of human genes in these transgenic animals can be useful in studies, as models for the development of diabetes, atherosclerosis, and Alzheimer’s disease. They also can generate large quantities of potentially therapeutic human proteins. Transgenic plants also offer many economic, safe, and practical solutions for production of variety of biopharmaceuticals. The plants have been engineered to produce many blood products (human serum albumin, cytokines), human growth hormone, recombinant antibodies, and subunitvaccines.

The usage of transgenic plants for the production of recombinant pharmaceuticals might open new avenues in biotechnology. As plants can be grown inexpensively with minimal complicated requirements, thus they may have tremendous therapeutic potential. The plants have been engineered to produce more nutrients or better shelf life. The transgenic plants have been created which have genes for insect resistance (Bt cotton, soybean, corn). Now billion acres of land is used for cultivation of genetically engineered crops of cotton, corn, and soybean as they have higher yield and are pest resistant. However, due to social, ethical, and biosafety issues, they have received acceptance as well as rejections at many places and health and environment-related concerns in many parts of the world [ 8 ].

Response to Antibiotic Resistance

Antibiotics areone of the broadly used therapeutic molecules produced by certain classes of microorganisms (bacteria and fungi) which can be used in diverse clinical situations to eliminate bacteria, improve symptoms, and prevent number of infections. Antibiotics have various other applications apart from clinical aspects. They can be used for the treatment of tumors and treatment of meat, in cattles and livestocks, in basic biotechnological work. However, their effectiveness is a matter of concern as bacteria which are continuously exposed to certain antibiotics might become resistant to it due to accumulation of mutations. These days antibiotic-resistant bacteria have increased and some of them have developed multiple drug resistance. Thus, it has become very difficult to initiate therapy in diseases like tuberculosis and leprosy. Biotechnology is solving the urgent and growing problem of antibiotic resistance. With the help of bioinformatics—powerful computer programs capable of analyzing billions of bits of genomicsequence data—scientists are cracking the genetic codes of bacteria and discovering “weak spots” vulnerable to attack by compounds identified via high-throughput screening. This kind of work led in 2000 to the approval of Zyvox (linezolid), an antibiotic to reach the market in 35 years.

Lytic bacteriophage viruses that infect and kill bacteria may be another way to counter resistance. First used to treat infection in the 1920s, “phage therapy” was largely eclipsed by the development of antibiotics. However, researchers in the former Soviet Republic of Georgia reported that a biodegradable polymer impregnated with bacteriophages and the antibiotic Cipro successfully healed wounds infected with a drug-resistant bacterium.

After exposure of strontium-90, three Georgian lumberjacks from village Lia had systemic effects, and two of them developed severe local radiation injuries which subsequently became infected with Staphylococcus aureus . Upon hospitalization, the patients were treated with various medications, including antibiotics and topical ointments; however, wound healing was only moderately successful, and their S. aureus infection could not be eliminated. Approximately 1 month after hospitalization, treatment with PhagoBioDerm (a wound-healing preparation consisting of a biodegradable polymer impregnated with ciprofloxacin and bacteriophages) was initiated. Purulent drainage stopped within 2–7 days. Clinical improvement was associated with rapid (7 days) elimination of the etiologic agent, and a strain of S. aureus responsible for infection was resistant to many antibiotics (including ciprofloxacin) but was susceptible to the bacteriophages contained in the PhagoBioDerm preparation [ 11 ].

The Challenges for the Technology

Gene therapy.

Some biotechapproaches to better health have proven to be more challenging than others. An example is gene transfer, where the defective gene is replaced with a normally functioning one. The normal gene is delivered to target tissues in most cases by virus that is genetically altered to render it harmless. The first ex vivo gene transfer experiment, conducted in 1990 at the National Institutes of Health (NIH), on Ashanti DeSilva who was suffering from severe combined immunodeficiency (SCID) helped boost her immune response and successfully corrected an enzyme deficiency. However, treatment was required every few months. However, 9 years later, a major setback occurred in gene therapy trial after the death of 18-year-old Jesse Gelsinger suffering from ornithine transcarbamylase (OTC) deficiency due to intense inflammatory responses followed by gene therapy treatment. There were some positive experiences and some setbacks from gene therapy trials leading to stricter safety requirements in clinical trials.

Designer Babies

The fancyterm designer baby was invented by media. Many people in society prefer embryos with better traits, intellect, and intelligence. They want to select embryo post germline engineering. This technique is still in infancy but is capable of creating lot of differences in the society thus requires appropriate guidelines.

Genetically Modified Food

Genetically modifiedcrops harboring genes for insect resistance were grown on billion of acres of land. These crops became very popular due to high yield and pest resistance. However, some of the pests gradually developed resistance for a few of these transgenic crops posing resistant pest threat. The other technologies as “traitor” and “terminator” technologies pose serious risk on crop biodiversity and would impart negative characters in the crop (they were not released due to public outcry).

Pharmacogenomics

Scientists do not believe they will find a single gene for every disease. As a result, they are studyingrelationships between genes and probing populations for variations in the genetic code, called single nucleotide polymorphisms, or SNPs, that may increase one’s risk for a particular disease or determine one’s response to a given medication. This powerful ability to assign risk and response to genetic variations is fueling the movement toward “individualized medicine.” The goal is prevention, earlier diagnosis, and more effective therapy by prescribing interventions that match patients’ particular genetic characteristics.

Tissue Engineering

Tissue engineering is one of the emerging fields with tremendous potential to supply replacement tissue and organ option for many diseases. Lot is achieved, lot more need to be achieved.

Ethical Issues

The pursuit of cutting-edge research “brings us closer to our ultimate goal of eliminating disability and disease through the best care which modern medicine can provide.” Understanding of the genetics of heart disease and cancer will aid the development of screening tools and interventions that can help prevent the spread of these devastating disorders into the world’s most rapidly developing economies.

Biotechnology is a neutral tool; nevertheless, its capabilities raise troubling ethical questions. Should prospective parents be allowed to “engineer” the physical characteristics of their embryos? Should science tinker with the human germ line, or would that alter in profound and irrevocable ways what it means to be human?

More immediately, shouldn’t researchers apply biotechnology—if they can—to eliminate health disparities among racial and ethnic groups? While genetic variation is one of many factors contributing to differences in health outcome (others include environment, socioeconomic status, health-care access, stress, and behavior), the growing ability to mine DNA databases from diverse populations should enable scientists to parse the roles these and other factors play.

Biotechnology along with supportive health-care infrastructure can solve complicated health problems. Accessibility to the new screening tests, vaccines, and medications and cultural, economic, and political barriers to change must be overcome. Research must include more people from disadvantaged groups, which will require overcoming long-held concerns, some of them have had about medical science.

Biotechnology has been a significant force which has improved the quality of lives and has incalculably benefitted human beings. However, technology does have prospects of doing harm also due to unanticipated consequences. Each technology is subjected to ethical assessment and requires a different ethical approach. Obviously the changes are necessary as technology can have major impact on the world; thus, a righteous approach should be followed. There is uncertainty in predicting consequences, as this powerful technology has potential to manipulate humans themselves. Ethical concerns are even more important as the future of humanity can change which require careful attention and consideration. Therefore, wisdom is required to articulate our responsibilities toward environment, animals, nature, and ourselves for the coming future generations. We need to differentiate what is important technologically rather that what technology can do. For an imperative question, that is, whether this can be achieved, the research must answer “Why should it be achieved”? Who would it benefit?

Issues Related to Safety

  • As the new GM crops are entering the market, the issue regarding their consumption, whether they are safe, without any risk, is one of the important concerns [ 2 ]. Though the results related to safety and usage are well reported (as compared to conventional crops), unknown fear from these products makes them non acceptable at many places [ 20 ].
  • As insect- and pest-resistant varieties are being prepared and used as Bt genes in corn and cotton crops, there exists a risk of development of resistance insect population. Another important factor is that these resistant crops may harm other species like birds and butterfly.
  • The development of more weeds may occur as cross-pollination might result in production of weeds with herbicide resistance which would be difficult to control.
  • The gene transfers might cross the natural species boundary and affect biological diversity.
  • The judgment of their usage would depend upon the clear understanding of risks associated with safety of these products in determining the impact of these on environment, other crops, and other animal species.

Future of the Technology

With the understanding of science, we should understand that genetic transfers have been occurring in animals and plant systems; thus, the risk of the biotechnology-derived products is similar as conventional crops [ 12 ].

The biotechnology products would be acceptable to many if they are beneficial and safe. People are willing to buy crops free of pesticides and insecticides. Nowadays people are also accepting crops grown without the usage of chemical fertilizers or pesticides, which are high in nutritive values.

The labeling of the product is also an ethical issue as some believe that labeling any product as biotechnology product might be taken by consumer as warning signs; however, others believe that labeling should be done as consumer has every right to know what he is consuming [ 9 ]. The products may be acceptable if consumers can accept the food derived from biotechnology weighing all pros and cons and, if the price is right, has more nutritive values, is good in taste, and is safe to consume [ 10 ].

Biotechnology is at the crossroads in terms of fears and thus public acceptance [ 15 ]. Surprisingly the therapeutic products are all accepted and find major place in biopharmaceutical industry, but food crops are still facing problems in worldwide acceptance. The future of the world food supply depends upon how well scientists, government, and the food industry are able to communicate with consumers about the benefits and safety of the technology [ 13 , 16 ]. Several major initiatives are under way to strengthen the regulatory process and to communicate more effectively with consumers by conducting educational programs [ 18 , 23 ].

Chapter End Summary

  • The advantages of biotechnology are so broad that it is finding its place in virtually every industry. It has applications in areas as diverse as pharmaceuticals, diagnostics, textiles, aquaculture, forestry, chemicals, household products, environmental cleanup, food processing, and forensics to name a few.
  • Biotechnology is enabling these industries to make new or better products, often with greater speed, efficiency, and flexibility.
  • With the applications of recombinant DNA technology, more safer and therapeutic drugs are produced. These recombinant products do not elicit unwanted immunological response which is observed when the product is obtained from other live or dead sources. Many of these therapeutics are approved for human usage, and many of them are in the phase of development.
  • Immunological and DNA-based techniques like PCR (polymerase chain reaction) are used for early diagnosis of disorders. PCR and NAAT with microarray can be utilized for the diagnosis of many diseases, and it can detect mutations in gene.
  • The technology holds promise through stem cell research and gene therapy and holds applications in forensic medicine.
  • The technique may be helpful in developing useful and beneficial plants. It overcomes the limitations of traditional plant breeding. The techniques of plant tissue culture, transgenics, and marker-assisted selections are largely used for selecting better yielding varieties and imparting quality traits in plants.
  • Food industries. Production of single-cell protein, spirulina, enzymes, and solid-state fermentations
  • Increase and improvement of agricultural production
  • Production of therapeutic pharmaceuticals
  • Production of vaccines and monoclonal antibodies
  • Cultivation of virus for vaccine production

Multiple Choice Questions

  • All of the above
  • Vitamin D and calcium
  • Growth hormone
  • Tissue plasminogen activator
  • Factor VIII
  • Genetically modifying organism
  • Production of therapeutics
  • Production of better diagnosis
  • Increase in yield of crops
  • Improved crop varieties
  • Lesser fertilizers and agrochemicals
  • All of these
  • It is resistant to it.
  • The toxin is enclosed in vesicle.
  • The toxin is present in inactive form.
  • None of these.
  • Gene therapy
  • Replacement protein therapy
  • Stem cell therapy
  • The productivity would improve.
  • The usage of chemical agent would be reduced.
  • The environment and crop would be insecticide free.
  • All of the above.
  • Detoxifying waste material
  • Burying waste material
  • Burning waste material
  • None of these

(1) In all the cells of our body, all the genes are active.

(2) In different cells of our body, different genes are active.

(3) Gene expression is spatially and temporally regulated.

  • All 1, 2, and 3 are correct.
  • 1 and 2 are correct.
  • 1 and 3 are correct.
  • 2 and 3 are correct.
  • Inoculation with monoclonal antibody was able to prevent small pox.
  • Inoculation with pus from sores due to cowpox could prevent small pox.
  • Attenuated vaccine was able to prevent small pox.
  • None of the above.
  • 1. (c); 2. (a); 3. (c); 4. (d); 5. (d); 6. (d); 7. (c); 8. (a); 9. (d); 10. (a); 11. (d); 12. (b)

Review Questions

  • Q1. What are cry proteins? What is their importance?
  • Q2. Give some applications of biotechnology in agriculture.
  • Q3. What is your opinion about labeling of biotechnology-based food product as rDNA technology derived product?
  • Q4. What are applications of biotechnology in maintaining environment?
  • Q5. What is medical biotechnology?
  • Q6. What are the challenges faced by biotechnology industry?
  • Q7. What do you think about GM crops?

Some Related Resources

  • http://ificinfo.health.org/backgrnd/BKGR14.htm
  • http://www.bio.org/aboutbio/guide1.html
  • http://www.bio.org/aboutbio/guide2000/guide00_toc.html
  • http://www.bio.org/aboutbio/guide3.html
  • http://www.bio.org/aboutbio/guide4.html
  • http://www.dec.ny.gov/energy/44157.html
  • http://www.ers.usda.gov/whatsnew/issues/biotech/define.htm
  • http://www.nal.usda.gov/bic/bio21
  • http://www.nature.com/nbt/press_release/nbt1199.html
  • www.angelfire.com/scary/intern/links.html
  • www.bio-link.org/library.htm
  • www.biospace.com
  • www.dnai.org
  • www.fiercebiotech.com
  • www.iastate.edu
  • www.icgeb.trieste.it
  • www.ncbi.nlm.nih.gov

10 Recent Biotechnology Advances In Medicine

In surgical rooms, doctors can now operate on patients remotely from their computer screens, guiding robotic arms to an accuracy of a few nanometers. Genetic laboratories equipped with DNA splicing enzymes, a mere sequence of polypeptide chains, can make wonders happen. The entire genetic makeup of human beings can be deconstructed into understandable genetic codes. Medical biotechnology has moved forward by leaps and bounds in the last few decades.

Biotechnology Breakthroughs In Medicine

Table of Contents

1. Stem Cell Research

Stem cells can keep dividing infinitely and have the capacity to differentiate into different types of body cells during the early development of an organism. In a laboratory, researchers can program these stem cells to differentiate into specific types of cells. This is where the innovation of biotechnology steps in. Imagine an individual with the degenerative spinal disorder that severely impacts their quality-of-life. With the help of stem cell research, it might be possible to grow these stem cells in vitro , in a lab setting, and then implanted back into the affected individual’s body. This would help restore their cognitive acuity, vision, hearing, and other physical features. This may sound far-fetched and like a plot from a sci-fi movie, but the preliminary results have been promising.

2. Human Genome Project

Often lauded as the one of greatest feat of exploration in human history, the  Human Genome Project  (HGP) was an international  scientific research  project coordinated by the National Institutes of Health and the U.S. Department of Energy. It was officially launched in 1990 with the goal of determining the sequence of nucleotide  base pairs  that make up human  DNA . In April 2003, the researchers announced that they had completed a preliminary sequencing of the entire human genome. This work of the HGP has allowed researchers to begin to understand the blueprint for building a person. As researchers learn more about the functions of genes and proteins, it has aided them in identifying genes that cause diseases.

3. Targeted Cancer Therapies

Currently, established standard chemotherapies are toxic for healthy cells. Targeted cancer therapies are drugs that work either by interfering with the function of specific molecules or by only targeting known cancerous cells, in order to minimize damage to healthy cells. According to the National Cancer Institute, “Eventually, treatments may be individualized based on the unique set of molecular targets produced by the patient’s tumor.”

4. 3D visualization and augmented reality for surgery

Surgery is brutal on a human body, and medical breakthroughs that make the surgical and healing process more efficient is always welcomed. Biotechnology has now made it possible for doctors to view an entire 3D image of the inside of a patient’s body through the use of MRI and CT scans. This allows each organ to be precisely projected so that the surgeon can make small, targeted incisions to minimize bodily trauma to the patient. Furthermore, augmented reality would allow pertinent information to be displayed directly overlaid over the relevant body parts.

5. HPV vaccine

Human Papilloma Virus (HPV) is one of the causative agents of cervical cancer. It is the second most lethal cancer in women, second only to breast cancer, killing 275,000 women worldwide every year. Therefore, a successful HPV vaccination is considered a major medical accomplishment. The U.S. Food and Drug Administration (FDA) has approved HPV vaccines like Gardasil and Cervarix for use among females between 9 – 26 years of age.

6. Face Transplants

8. 3d printed organs.

Artificial limbs have been in use for centuries, and there has been a steady improvement in the mobility and versatility of bionic limbs. Now new advances in bionic technology and  3D printing  have taken it even further. It has made it possible to artificially construct  internal organs like  heart ,  kidney , and  liver . Doctors have been able to implant these into individuals that need them successfully.

9. Nerve Regeneration

10.  brain signals to audible speech.

Scientists are working on creating a device that can translate brain signals to audible speech using a voice synthesizer. This would serve as an incredible tool in communicating with individuals paralyzed with the disease or traumatic injuries. Furthermore, scientists have found that they can use these devices on epileptic patients to isolate the source of their seizures.

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Medical Biotechnology: Advancement and Ethics

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recent research in medical biotechnology

What is Medical Biotechnology?

Medical biotechnology is a branch of medicine that uses living cells and cell materials to research and then produce pharmaceutical and diagnosing products. These products help treat and prevent diseases. From the Ebola vaccine to mapping human DNA to agricultural impacts, medical biotechnology is making huge advancements and helping millions of people.

Some of the most recent uses of biological tech is work in genetic testing, drug treatments, and artificial tissue growth. With the many advancements in medical biotechnology, there are new concerns that arise. From funding to ethics, there are many things to determine and regulate when it comes to this fast-paced industry. Learn about the many technical biology advancements and the concerns surrounding them.

Major Medical Biotechnology Advancements

From cancer research to agriculture advancements, medical biotechnology has many promising avenues of technological growth that have the potential to help many people.

CRISPR technology or CRISPR-Cas9 utilizes a protein called Cas9, which acts like a pair of molecular scissors and can cut DNA. CRISPRs are specialized stretches of DNA and are used in medical biotechnology as a tool to edit genomes. This allows scientists to alter DNA and modify gene functions, often called genetic engineering. There are many applications, like correcting genetic defects, treating diseases, preventing the spread of diseases, improving crops, and more. But the science of altering genomes has many ethical concerns surrounding it. From the ability to mutate genes and the unknowns surrounding gene mutation, CRISPR is a controversial area of biomedical science. Some new studies even show that perhaps CRISPR technology can create tumors and cancer with DNA deletions that aren’t controlled or precise. Of course, pharmaceutical companies and other scientific organizations that develop and utilize CRISPR technology are trying to downplay the concerns and issues, so the reality of the benefits and damage of the technology is somewhat unknown.

Tissue Nanotransfection

New science may have the ability to heal people with a single touch.  Sound too good to be true? It’s not. Tissue nanotransfection works by injecting genetic code into skin cells, which turns those skin cells into the other types of cells required for treating diseases. In some lab tests, one touch of TNT completely repaired the injured legs of mice over a period of a few weeks by turning skin cells into vascular cells. And reportedly, this biotech can work on other types of tissue besides skin. The potential for this type of gene therapy is huge, from helping car crash victims to active duty soldiers. Medical biotechnology has made this advancement possible, and the continued research and testing will only help improve this tech and adopt it across hospitals and medical centers.

Recombinant DNA Technology

Recombinant DNA technology  is combining DNA molecules from two different species and then inserting that new DNA into a host organism. That host organism will produce new genetic combinations for medicine, agriculture, and industry. There are many examples of recombinant DNA technology being utilized, from biopharmaceuticals and diagnostics to energy applications like biofuel to agricultural biotechnology with modified fruits and veggies. The genetically modified products are able to perform better than the regular medicine or produce. Recombinant agriculture is able to be more pest resistant or weather resistant; recombinant medicine like insulin is able to better work with bodies, etc. Because of the many benefits that recombinant DNA holds for a variety of products, researchers are optimistic about the future it has within biosciences and in other industries as well.

Genetic Testing from 23andMe

Genetic and ancestry kits are popular these days, and they are beneficial for more than just helping people understand their genetics and heritage. New studies are showing that saliva kits are able to test for things like breast cancer by looking at gene mutations. Certain races are also more likely to inherit certain mutations or human diseases, and knowing what races make up your genetic material can help you be prepared. While 23andMe test results shouldn’t be a reason to make decisions about treatments, understanding your heritage and how that could impact your health is valuable. 23andMe is also authorized to analyze for a variety of diseases, including Parkinson’s and Alzheimer's.

HPV Vaccine

You’ve probably heard of the Human Papilloma Virus (HPV) and how it’s linked to cervical cancer—which is the second most lethal form of cancer for women, next to breast cancer. Statistics show that cervical cancer kills 275,000 women annually, which is why a vaccine for HPV is so important. The good news is there are now two vaccines on the market—Cervarix and Gardasil—that have been approved by the U.S. Food and Drug Administration for use in women from ages 9 to 26.

Stem Cell Research

Biotechnology plays a big part in supporting stem cell research, which supports the exploration of growing stem cells in a lab setting or in vitro. This could help in situations where patients may be suffering from a disease or disorder where implanting stem cells could help restore their vitality and give them a new lease on life. How does it work? Because stem cells can repeatedly divide and transform into other types of body cells, biotechnologists can learn how to work with their unique profiles to encourage growth of specific types of cells. Though research is ongoing, it’s reported that the results show hope for the future of this unique medical approach.

recent research in medical biotechnology

Medical and Ethical Issues of Biotechnology

While there are great advancements and positives to medical biotechnology, anything this fast-growing and powerful is bound to come with some concerns and issues. Medical biotechnology is a controversial medical topic, with medical ethical issues associated.

Risk to Human Life in Clinical Trials

A huge risk of medical biotechnology is its impact during clinical trials. Because it’s such new tech, people can and have gotten hurt—and even died—during trials of the technology. Because of these risks, extensive research should be performed before even thinking of introducing tech to human subjects, and those who are participating in a trial should be extremely aware of any and all possibilities. Unfortunately, the paradox is that many times people who are sick are willing to try new things for the chance to get cured. This means researchers and doctors have a huge ethical responsibility to truly outline for a patient what the costs may be and respect their ultimate decision.

High Cost May Exclude the Poor

While medical biotechnology has huge potential to make medicine more efficient and easy, what’s the cost? This technology is often hugely expensive compared to traditional treatments. There is an ongoing give and take about finding new medical advancements and the cost it takes to do research and then market the findings for purchase. There is also the concern that high costs of tech treatments can exclude an entire class of people from being able to utilize them. This is also a huge give and take, with science and medicine having a responsibility to help all patients—not just those who are wealthy enough to buy the best care.

Privacy Concerns

Privacy is an ongoing issue in our technology world, but reading someone’s DNA seems to be a giant privacy breach. Imagine a doctor looks at a young child’s DNA and finds out they are likely to develop a heart disease or terminal issue. Does their employer have the right to know that? Should this information impact their ability to get a house or insurance? HIPAA offers some protection, but as medical biotechnology continues to advance the ability to read genes, insurance companies, doctors, and governments will have to come up with new programs and privacy tactics to match all the new needs that will arise.

Some Groups Oppose Stem Cell Research

Medical biotechnology is kind of a hot-button political issue, with presidential candidates even being asked about their position. The idea of working with fetal tissue, or other tissue, to learn about regrowth conjures images of Frankenstein’s monster. Scientists and researchers have been cautioned multiple times to be ethical and moral when doing this research. For example, using human tissue for research can be seen as ethical, while using an embryo’s tissue can be seen as unethical because it can damage the embryo. It is still early in the stem-cell research process, but as technology and research continue to advance in that area, scientists will have to consider moral and ethical lines even more.

Bioterrorism is a National Concern

Medical biotechnology has been used for security measures to help prevent a large number of people from possible bioterrorism. But the development of these projects takes away funding and time from curing known diseases. It becomes a real question of how to divide resources among projects and knowing where the resources are most needed. It’s difficult because we don’t know if people will die from bioterrorism but with so many people being concerned, it seems like a worthwhile place to spend time and money.

Any way you look at it, there are a number of concerns when it comes to medical biotechnology, and as we continue to make advancements, these ethical considerations will have to be made.

recent research in medical biotechnology

Role of Nurses in the Biotechnology Industry

Nurses have an ongoing role in medical biotechnology because of their direct experience with patient care. Nurses are able to use their knowledge and experience in hospitals and clinics to understand and demonstrate how medicines and drugs would impact large populations. Beyond knowing the science, they have the human element that researchers sometimes lack. They are able to understand how a patient would respond to a potential treatment and can help researchers consider new approaches to technology and adoption practices.

Nurses who have leadership and management experience can also help support researchers by keeping them on track with goals and checkpoints, ensuring that projects are moving along smoothly and key information is being conveyed to management. In instances where patients are part of the research, nurses can gain deeper insights from patients about their experiences in trials and how they’ve been affected. By being fluent in medical terminology and having the ability to effectively connect with patients, nurses can help bridge the gap between the two worlds and share valuable information between patients and researchers.

Role of Healthcare Leaders in the Biotechnology Industry

Because the biotech industry is constantly shifting and changing, strong leadership is needed to help navigate those changes and support researchers in their work. This is where healthcare managers come in. With their experience in operational management, these leaders can assist with streamlining processes and addressing the needs of a variety of stakeholders, while their knowledge of data-driven decision making can support researchers crunching the numbers associated with their work. An understanding of financial management can keep projects on budget, while experience in healthcare information technology is also a valuable asset to the biotech world. And with a background in marketing, healthcare leaders can also be key in communicating findings, both internally and externally.

Medical biotechnology is a field that is exploding and along with its potential for saving lives, it raises some ethical questions. As the field continues to grow, people from all types of industries are going to be required to make decisions to help regulate this field.

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Bharat Biotech adds ICMR as co-owner of Covid-19 vaccine patent

Bharat biotech has added the indian council of medical research (icmr) as co-owner of the covid-19 vaccine patent..

Bharat Biotech adds ICMR as co-owner of Covid-19 vaccine patent

Bharat Biotech has added the Indian Council of Medical Research (ICMR) as co-owner of the Covid-19 vaccine patent. Notably, Bharat Biotech was working on developing the Covid-19 vaccine as a top priority to ensure product availability at the earliest. The Covid vaccine development of Bharat Biotech International Limited (BBIL) was faced with multiple challenges and all organizations were in a rush to develop vaccines and file the appropriate patents, prior to any other entity or prior to any data being published in journals.

Bharat Biotech's covid vaccine application was filed in the above circumstances and since BBIL-ICMR agreement copy, being a confidential document, was not accessible. Hence, ICMR was not included in the original application, the press release said. Though this was purely unintentional, such mistakes are not uncommon for the Patent office and therefore, Patent Law provides provisions to rectify such mistakes, the release added.

"BBIL has great respect for ICMR and is thankful to ICMR for their continuous support on various projects therefore as soon as this inadvertent mistake was noticed, BBIL has already started the process to rectify it by including ICMR as co-owner of the patent applications for Covid-19 vaccine," the press release said. It further informed that necessary legal documents are being prepared for it and BBIL will file those documents in the Patent office as soon as they are ready and signed.

Notably, these actions are in accordance with the Memorandum of Understanding (MoU) signed between ICMR-NIV Pune and BBIL for joint development of the Covid-19 vaccine in April 2020, the press release informed. (ANI)

(This story has not been edited by Devdiscourse staff and is auto-generated from a syndicated feed.)

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How a Baltimore neuroscience study is rewriting Black America’s relationship with medical research

By Alia Sajani June 19, 2024

A Black person, wearing gloves, slides a piece of specimen under a microscope in a lab — coverage from STAT

P riscilla Agnew-Hines will never be able to forget that day in early 2020. On March 26, just weeks after Covid-19 officially became a global pandemic, her son died from an overdose.

Larry, 41, was a chef, a drummer for his gospel church, and the son who challenged Priscilla’s barbecue skills during summer cookouts. He also struggled with addiction. That, she knew. But what made him more prone to addiction?

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“What part of the brain triggers mental illness?” Priscilla asked during a recent interview. “If we continue to be quiet, no one will understand the process of mental illness.” So when she learned about researchers looking into the role of genetics in neurological conditions among African Americans, Priscilla was hopeful. Looking for answers, she donated her son’s brain to the study.

Priscilla was among the more than a hundred Black Baltimorians who donated the brains of their deceased loved ones for a groundbreaking initiative that’s seeking to rebuild the medical research community’s tattered relationship with Black Americans.

The study , published in the journal Nature Neuroscience last month, is the first major undertaking from the African Ancestry Neuroscience Research Initiative — a collaboration between Morgan State University, a historically Black research university in Baltimore, the Lieber Institute for Brain Development, and local community leaders. Founded in 2019, the initiative has sought to understand the biological underpinnings of some neurological conditions that are more prevalent among those with African American ancestry.

Researchers from the Lieber Institute, housed at Johns Hopkins University, found that genetics, to some degree, could explain the higher prevalence of conditions like Alzheimer’s and stroke among Black Americans, or the lower prevalence of Parkinson’s. They also speculated that environmental factors, and their impact on gene expression, might better explain higher incidence of mental health conditions like schizophrenia and depression.

The findings could someday lead to personalized therapies informed by genetic ancestry. The researchers, who worried that studies like theirs might rekindle old myths and give validity to a biological basis for race, said the focus should be on how environmental stressors and lived experiences impact gene expression. This interplay of environment and genetics could make people more, or less, prone to certain diseases.

recent research in medical biotechnology

Bianca Jones Marlin, a neuroscientist at the Zuckerman Institute at Columbia University who studies how learned information is passed down generations through genetics, lauded the researchers’ efforts to center African Americans in their study. Marlin said while the findings deepen neuroscience’s understanding of how environmental factors affect genes in the brain, she wished the researchers had zeroed in more on the impact of specific environmental factors, especially social and emotional stressors like racism, which has impacted the African American community for generations.

Still, Marlin is hopeful that the study will inspire future research to investigate how socio-emotional stressors impact gene expression, potentially predisposing Black Americans to certain diseases. By taking into account the social determinants of health , a public health concept that accounts for how biology is impacted by the environment, researchers may gain insight into the policy changes needed to improve health outcomes in the African American community.

Related: A preacher’s new calling: Connecting neuroscience researchers as a way to advance social justice

The landmark study was made possible by the more than 100 brains (and 400 tissue samples from various brain regions) from deceased Baltimorians who self-identified as African American — an achievement in itself given the long history of racism and abuse that has marked Black Americans’ relationship with biomedical research.

In the 1800s, the pseudoscience of phrenology, the idea that bumps present on skulls could identify mental capabilities, was used to justify racism and slavery. More recently in 1951, Henrietta Lacks’ cells were collected by her physician during a cervical cancer biopsy at Johns Hopkins University. Known as HeLa cells, Lacks’ fast growing cancer cells are now used extensively in biomedical research, but were first grown in the lab by her physician without her consent. And, even decades after the infamous Tuskegee Syphilis Study , which started in 1932, a majority of Black Americans still believe that “medical researchers experiment on Black people without their knowledge or consent,” a recent Pew Research Center survey found.

Whether it is due to Black Americans’ mistrust, or because they were excluded, neuroscience research cohorts are typically dominated by participants with European descent. As a result, large genetic databases commonly used in brain research are limited in their use to investigate the disparities in neurological diseases — Black Americans are 20% more likely to experience major mental health problems , and twice as likely to develop Alzheimer’s disease .

In the Lieber Institute study, researchers first collected and sorted brain tissues based on self-reported race, hoping to understand how the lived experience of being African American in the U.S. impacted gene expression. Then, they determined genetic ancestry by analyzing the differences in specific genetic markers — African Americans can have a mix of African and European ancestry as a result of the long history of migration and slave trade.  

To avoid playing into old stereotypes about biological differences between races, researchers sought help from Black neuroscientists. Scientists from Black in Neuro , a nationwide effort of Black scientists established in 2020 during the Black Lives Matter movement, worked closely with the researchers on how to communicate the findings.

Related: Genetic variant common among West African descendants contributes to large cardiovascular disease burden

The researchers found that environmental factors — that could include everything from water quality and air pollution, to racism — impacted neurological health outcomes among people of African descent. Structural changes to DNA mediated by environmental factors, called epigenetics,   accounted for 15% of disease prevalence, while genetics accounts for 60% of differences between people of African and European ancestry.

They also found that genes that determine the body’s immune response, and the structure of blood vessels, were more likely to be elevated in people of African descent compared to people of European descent. The role of the immune system in affecting neurological diseases has recently gained the attention of the scientific community — since stress can affect the immune system, it may be the mechanism that makes some neurological diseases worse in Black Americans, a community that has a long history of experiencing discrimination.

The researchers found that genetics can explain only up to 26% of the likelihood of African Americans experiencing ischemic stroke, 27% for Parkinson’s disease and 30% for Alzheimer’s disease.

While the new findings advance neuroscience’s understanding of the disparities in disease prevalence among those with African-American ancestry, experts told STAT that the study itself is a model for more inclusive medical research.

“We reasoned that if we could demonstrate the success of this model in Baltimore (a city with a largely Black population and a long history of racial trauma and mistrust of medical institutions), we could institute a model that is suitable to be applied throughout neglected communities across the nation,” Alvin C. Hathaway Sr., who co-founded the African Ancestry Neuroscience Research Initiative, wrote in an editorial comment published along with the study.

Hathaway, who retired as the pastor of Union Baptist Church in Baltimore, was a crucial link in researchers’ ability to earn the trust of the African American community. During the 2020 racial reckoning after the murder of George Floyd, Hathaway said he realized going to protests wasn’t enough. Following a conversation with a member of the church, Hathaway decided that bringing more Black Americans into biomedical research was his new calling.

After the early success of the initiative’s first study, Hathaway is now focused on expanding the effort to more historically Black universities in other parts of the country.

For Priscilla, Larry’s mother, the study offered some closure, knowing that her son was part of an effort that could someday result in better medical care for those struggling with neurological and psychiatric conditions. She is now training to become a recovery coach, wanting to help others, like Larry, who are struggling with addiction.

About the Author Reprints

Alia sajani.

AAAS Mass Media Fellow

Alzheimer’s

neuroscience

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  1. Biotechnology

    Biotechnology articles from across Nature Portfolio. Biotechnology is a broad discipline in which biological processes, organisms, cells or cellular components are exploited to develop new ...

  2. The first gene-editing treatment: 10 Breakthrough Technologies 2024

    Biotechnology and health The first gene-editing treatment: 10 Breakthrough Technologies 2024 Sickle-cell disease is the first illness to be beaten by CRISPR, but the new treatment comes with an ...

  3. Research articles

    Nature Biotechnology ( Nat Biotechnol) ISSN 1546-1696 (online) ISSN 1087-0156 (print) Read the latest Research articles from Nature Biotechnology.

  4. Fresh from the biotech pipeline: record-breaking FDA approvals

    Nature Biotechnology 42 , 355-361 ( 2024) Cite this article. New drug approvals reached an all-time high in 2023, with five gene therapies, the first CRISPR-Cas9-edited therapy and a disease ...

  5. Human Molecular Genetics and Genomics

    In 1987, the New York Times Magazine characterized the Human Genome Project as the "biggest, costliest, most provocative biomedical research project in history." 2 But in the years between the ...

  6. Articles

    Research on the targeted improvement of the yield of a new VB 12-producing strain, Ensifer adhaerens S305, based on genomic and transcriptomic analysis. Vitamin B 12 (VB 12) has a wide range of applications and high economic value. In this study, a new strain with high VB 12 production potential, Ensifer adhaerens S305, was

  7. Ethics and Highly Innovative Research on Brain Diseases

    Disclosure Forms (nejmp2310377_disclosures.pdf) Download. 205.90 KB. Now Reading: Despite exciting advances, conducting brain research and appropriately implementing interventions or insights ...

  8. Protecting Human Health through Biotechnology Breakthroughs and

    After nearly 10 years of development and testing, this biotechnology breakthrough became the core of a new life science platform with therapeutic applications for many diverse diseases. Visit our Newsroom. Office of News and Public Information. E-mail: [email protected]. Tel.: 202-334-2138.

  9. Biotech

    Biotech February 1, 2024 A-fib—a Rapid, Irregular Heartbeat—Can Kill You, but New Tech Can Spot It A fluttering heartbeat called A-fib can lead to stroke, but smartwatches can detect it, and ...

  10. Genomics in medicine: A new era in medicine

    Core Tip: The field of Genomics is the future of medicine, as evidenced by the unprecedented research and clinical application which pushed the time boundaries for the coronavirus disease 2019 mRNA vaccines.However the path to unleashing the potential from genomic tools is far from perfect. A thorough research with international collaboration and cooperation is a necessity and the need of the ...

  11. Existing high blood pressure drugs may prevent epilepsy, Stanford

    The finding comes out of an analysis of the medical records of more than 2 million Americans taking blood pressure medications. The study , published June 17 in JAMA Neurology , suggests that the drugs, called angiotensin receptor blockers, could prevent epilepsy in people at highest risk of the disease, including older adults who have had strokes.

  12. Biotechnology

    Read the latest news focusing on Biotech drug developments, clinical research and pharmaceuticals.

  13. Innovative system enhances biological-artificial interactions in

    Recent research in bioelectronics and neuromorphic engineering has facilitated the development of new-generation neuroprostheses for brain repair. However, realizing their full potential requires ...

  14. Big data: Historic advances and emerging trends in biomedical research

    Current Research in Biotechnology. Volume 4, 2022, Pages 138-151. ... Healthcare and medical research are shifting focus to improving disease outcomes through finding hidden associations or patterns ... However, based on the current trends in biomedical research, the incorporation of big data analytics is expected to become more frequent, thus ...

  15. Cell Press: Trends in Biotechnology

    We are pleased to announce that Trends in Biotechnology—the multidisciplinary journal from Cell Press—will publish original research across applied life sciences that examines bio-based solutions to real-world problems.. Trends in Biotechnology is a high-impact journal with a 40-year legacy. Our highly-cited review articles provide a foundation for an exciting new chapter for the journal.

  16. Biotechnology News, Research

    MGI Tech Co., Ltd. ("MGI"), a company committed to building core tools and technologies that drive innovation in life science, recently set a new record for sequencing applications by ...

  17. Leqembi and Alzheimer's: What to Know About the New Drug, Treatment and

    As the F.D.A. considers a new Alzheimer's medication, we asked experts how the rollout of a similar drug has gone. By Dana G. Smith Over the last three years, a new class of Alzheimer's drug ...

  18. Ingestible microbiome sampling pill technology advances

    Tufts University. "Ingestible microbiome sampling pill technology advances." ScienceDaily. ScienceDaily, 12 June 2024. <www.sciencedaily.com / releases / 2024 / 06 / 240612140911.htm>. Significant ...

  19. Current Research in Biotechnology

    Current Research in Biotechnology is a peer-reviewed gold open access (OA) journal and upon acceptance all articles are permanently and freely available. It is a companion to the highly regarded review journal Current Opinion in Biotechnology (2018 CiteScore 8.450) and is part of the Current Opinion and Research (CO+RE) suite of journals. All ...

  20. A Clinical Diagnostic Test for Calcium Release Deficiency Syndrome

    Case-control study including individual cases of calcium release deficiency syndrome (CRDS), 3 patient control groups, and genetic mouse models assesses the cardiac repolarization response on an electrocardiogram after brief tachycardia and a pause as a clinical diagnostic test for CRDS.

  21. Medicine and health of 21st Century: Not just a high biotech ...

    The "magic bullet" era. Drug therapy began centuries ago with the use of plant extracts and progressively evolved into the development of purified and targeted materials for a wide range of ...

  22. How biotech can revolutionize healthcare for the future

    Biotech and its applications are rapidly evolving and have the potential to revolutionize industries, including healthcare. But forward-thinking businesses, governments and academia need to work together to realize biotechnology's full promise. C4IR Serbiarecently launched at the Biotech Future Forum as biotech was revealed to be the country's ...

  23. WEHI spinout aims to revolutionize the development ...

    New biotechnology company, Ternarx, aims at changing this statistic through the development of targeted protein degrader (TPD) technology, which is designed to destroy these "undruggable" proteins ...

  24. The Future of Biotech: Innovations and Trends in Science and Research

    The Future of Biotech: Innovations and Trends in Science and Research. Biotechnology is rapidly advancing, bringing with it groundbreaking innovations and trends. As the field evolves, staying updated on the latest developments is crucial. This article explores what lies ahead for biotech, focusing on new scientific discoveries and research ...

  25. An Introduction to Biotechnology

    Medical Biotechnology. This fieldof biotechnology has many applications and is involved in production of recombinant pharmaceuticals, tissue engineering products, regenerative medicines such as stem cell and gene therapy, and many more biotechnology products for better human life (Fig. 1.6). Biotechnological tools produce purified bio ...

  26. Current research in biotechnology: Exploring the biotech forefront

    In this latest addition of biotechnology literature analysis, we aimed to unveil the latest trends (since 2017) in biotechnology research. By analyzing the research literature, we identified the latest popular research themes, major contributors in terms of institutions, countries/regions, and journals. 2. Materials and methods.

  27. 10 Recent Biotechnology Advances In Medicine

    7. CRISPR. Clustered Regularly Interspersed Short Palindromic Repeats is a relatively new gene-editing system that has been hailed as a groundbreaking tool in medical research.Of its many uses, HIV research is one of them. Researchers can now keep up with the constant genetic mutations by actively testing newly found mutations and constantly editing them to tweak targeted therapies.

  28. Medical Biotechnology: Advancement and Ethics

    Medical biotechnology is a branch of medicine that uses living cells and cell materials to research and then produce pharmaceutical and diagnosing products. These products help treat and prevent diseases. From the Ebola vaccine to mapping human DNA to agricultural impacts, medical biotechnology is making huge advancements and helping millions ...

  29. Bharat Biotech adds ICMR as co-owner of Covid-19 vaccine patent

    SHARE. Bharat Biotech has added the Indian Council of Medical Research (ICMR) as co-owner of the Covid-19 vaccine patent. Notably, Bharat Biotech was working on developing the Covid-19 vaccine as a top priority to ensure product availability at the earliest. The Covid vaccine development of Bharat Biotech International Limited (BBIL) was faced ...

  30. Baltimore study rewrites Black America's relationship with medical research

    The researchers found that genetics can explain only up to 26% of the likelihood of African Americans experiencing ischemic stroke, 27% for Parkinson's disease and 30% for Alzheimer's disease ...