Category Archives: Molecular Genetics

Media Advisory

Friday, November 19, 2021

NPC1 leads to difficulty controlling movements, liver and lung disease, impaired swallowing, intellectual decline and death.

Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in Niemann-Pick disease type C1 (NPC1), a rare genetic disorder affecting children and adolescents, suggests a study in mice by scientists at the National Institutes of Health.

The study was conducted by Forbes D. Porter, M.D., Ph.D., of NIHs Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and colleagues in the National Human Genome Research Institute and National Institute of Arthritis and Musculoskeletal and Skin Disease. It appears in Molecular Genetics and Metabolism. The study was supported in part by a grant from the Ara Parseghian Medical Research Foundation.

NPC1 results from an impaired ability to move cholesterol through cells, leading to difficulty controlling movements, liver and lung disease, impaired swallowing, intellectual decline and death. Much of the movement difficulties in NPC1 result from gradual loss of brain cells known as Purkinje neurons. The researchers found that mice with a form of NPC1 have a diminished ability to lower levels of glutamate a brain chemical that stimulates neurons after it has bound to a neurons surface. High levels of glutamate can be toxic to cells. The researchers believe the buildup of glutamate contributes to the brain cell loss seen in the disease. Riluzole blocks the release of glutamate and hence delays the progression of ALS in people.

In the current study, mice with NPC1 survived 12% longer when treated with riluzole, compared to untreated mice. The researchers believe that riluzole or similar drugs may provide a way to slow disease progression in patients with NPC1.

Forbes D. Porter, M.D., Ph.D., NICHD Clinical Director, is available for comment.

Cougnoux, A., et al Reduction of glutamate toxicity: a novel therapeutic approach for Niemann-Pick disease, type C1. Molecular Genetics and Metabolism. 2021.

This media availability describes a basic research finding. Basic research increases our understanding of human behavior and biology, which is foundational to advancing new and better ways to prevent, diagnose, and treat disease. Science is an unpredictable and incremental process each research advance builds on past discoveries, often in unexpected ways. Most clinical advances would not be possible without the knowledge of fundamental basic research. To learn more about basic research, visit https://www.nih.gov/news-events/basic-research-digital-media-kit.

About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): NICHD leads research and training to understand human development, improve reproductive health, enhance the lives of children and adolescents, and optimize abilities for all. For more information, visit https://www.nichd.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Repurposed ALS drug shows promise in mouse model of rare childhood genetic disorder - National Institutes of Health

Global Molecular Diagnostics Market to Reach $24,228. 0 Million by 2031. Market Report Coverage - Molecular Diagnostics Market Segmentation.

New York, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Molecular Diagnostics Market - A Global and Regional Analysis: Focus on Product, Testing Location, Application, Technology, and End User - Analysis and Forecast, 2021-2031" - https://www.reportlinker.com/p06184570/?utm_source=GNW Products (Kits and Consumables, Systems, Software and Other Products) Testing Location (Laboratory Testing and Point-of-Care (POC) Testing) Application (Core Molecular Diagnostics, Reproductive Genetics, Companion Diagnostics (CDx), Liquid Biopsy, and Others) Technology (Polymerase Chain Reaction (PCR), Next-Generation Sequencing, Isothermal Nucleic Acid Amplification Technology (INAAT), Microarray, In-situ Hybridization (ISH), Immunohistochemistry (IHC) and Other Technologies) End User (Hospitals, Diagnostics Centers, Out-Patient Clinics/General Practitioners, Research Laboratories, and Other End Users)

Regional Segmentation

North America: U.S., Canada Europe: Germany, France, Italy, U.K., Spain, and Rest-of- Europe Asia-Pacific: Japan, China, India, Australia, Singapore, and Rest-of-Asia-Pacific Latin America: Brazil, Mexico, Rest-of-Latin America Rest-of-the-World (RoW)

Market Growth Drivers

A Highly Increasing Prevalence of Infectious Diseases and Various Types of Cancer, Globally Growth in the Biomarker Identification Market and Advancements in Molecular Techniques Increase in Awareness and Acceptance of Personalized Medicines on a Global Level Significant External Funding for Executing Research and Development Exercises

Market Challenges

Uncertain Reimbursement Scenario Lack of High-Complexity Testing Centers Complex Regulatory Frameworks Delaying the Approval of New Molecular Diagnostic Tests

Market Opportunities

Massive Scope for Adoption of Molecular Diagnostics in Emerging Nations Rise of Next-Generation Ultrasensitive Molecular Diagnostics Novel Revenue Streams

Key Companies Profiled

Abbott, Agilent Technologies, Inc., Becton, Dickinson and Company (BD), bioMrieux SA, Bio-Rad Laboratories, Inc., Danaher, F. Hoffmann-La Roche Ltd, Guardant Health, HTG Molecular Diagnostics, Inc., Illumina, Inc., Invivoscribe, Inc., ICON plc, LungLife AI, Inc., QIAGEN, QuantuMDx Group Ltd., Siemens Healthcare GmbH, Thermo Fisher Scientific Inc.

Key Questions Answered in this Report: How is each segment of the market expected to grow during the forecast period 2021-2031, and what is the anticipated revenue to be generated by each segment? What are the major market drivers, challenges, and opportunities in the global molecular diagnostics market? What are the underlying structures resulting in the emerging trends within the global molecular diagnostics market? How is each segment of the global molecular diagnostics market expected to grow during the forecast period, and what will be the expected revenue generated by each of the segments by the end of 2031? What are the key developmental strategies implemented by the major players to sustain in the competitive market? What are the key regulatory implications in developed and developing regions for molecular diagnostics? Who are the leading players with significant offerings to the global molecular diagnostics market? What is the current market dominance for each of these leading players? What would be the compound growth rate witnessed by the leading players in the market during the forecast period 2021-2031? Which molecular diagnostic product type has the most promising growth? What are the key applications in the global molecular diagnostics market? What are the major segments of these applications? What technologies are dominating these application segments? What are the major technologies that are employed in the global molecular diagnostics market? Which is the dominating technology? Who are the primary end-users of the global molecular diagnostics market? Which is the fastest-growing end-user segment in the global molecular diagnostics market? Who are the key manufacturers in the global molecular diagnostics market, and what are their contributions? Also, what is the growth potential of each major molecular diagnostics manufacturer? What is the scope of the global molecular diagnostics market in North America, Europe, Asia-Pacific, Latin America, and Rest-of-the-World? Which molecular diagnostics application and end user dominate these regions? What are the emerging trends in the global molecular diagnostics market? How are these trends revolutionizing the diagnostic procedure? Which technologies are anticipated to break through the current molecular diagnostic regime? Which companies are anticipated to be highly disruptive in the future and why? What are the regulatory procedures that are required to unify the approval process for emerging molecular diagnostics? How will these enhance the reimbursement scenario? What are the gaps in regularizing optimum molecular diagnostic adoption in regular healthcare routines? How are these gaps being tackled?

Market Overview

Diagnostic tests provide critical insights at every stage of medical care - right from the genetic tests providing information about personalized cancer treatment to the microbial culture identifying accurate antibiotics against an infection. The importance of diagnostic tests throughout the entire procedure (i.e., prevention, detection, diagnosis, treatment, and successful management of health conditions) of treating an individual for a particular disease is as consequential as the treatment itself. It has been subjected to extensive research for further refinement of the same.

Our healthcare experts have found the molecular diagnostics market to be one of the stable markets, and the global market is predicted to grow from $10,914.6 million in 2020 to $24,228.0 million in 2031 and is expected to grow with a CAGR of 7.38% during the forecast period 2021-2031.

Factors fueling the growth of the market include a highly increasing prevalence of infectious diseases and various types of cancer, an increase in awareness and acceptance of personalized medicines on a global level, and significant external funding for executing research and development exercises.Despite rapid advanced industry growth, several key issues need to be addressed to facilitate future growth.

Uncertain reimbursement scenarios, lack of high-complexity testing centers, and complex regulatory frameworks delaying the approval of new molecular diagnostic tests are hampering the market growth. Further, some of the opportunities, such as massive scope for the adoption of molecular diagnostics in emerging nations and the rise of next-generation ultrasensitive molecular diagnostics and novel revenue streams, provide growth to the market.

Within the research report, the market has been segmented based on product, testing location, application, technology, end user, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive Landscape

The exponential rise in the prevalence of infectious diseases and various types of cancer globally has created a buzz among companies to invest in advanced technologies such as molecular diagnostics.

Based on region, North America holds the largest share, owing to improved healthcare infrastructure, rise in per capita income, and improvised reimbursement policies in the region. However, the Asia-Pacific and Europe regions are anticipated to grow at the fastest CAGR during the forecast period.

Countries Covered North America U.S. Canada Europe Germany Italy France U.K. Spain Rest-of-Europe Asia-Pacific China India Singapore Japan Australia Rest-of-Asia-Pacific Latin America Brazil Mexico Rest-of-Latin America Rest-of-the-World (RoW)Read the full report: https://www.reportlinker.com/p06184570/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Here is the original post:
Molecular Diagnostics Market - A Global and Regional Analysis: Focus on Product, Testing Location, Application, Technology, and End User - Analysis...

ANN ARBOR, Mich. & BOSTON--(BUSINESS WIRE)--First paragraph, first sentence of release should read: Penrose TherapeuTx, a privately held preclinical stage small molecule drug development company ... (instead of Penrose TherapeuTx, a privately held clinical stage small molecule drug development company ...).

The updated release reads:

PENROSE THERAPEUTX APPOINTS PHARMACEUTICAL LEADER DAVID SHERRIS, PH.D., AS PRESIDENT AND CHIEF EXECUTIVE OFFICER

Veteran industry executive will spearhead the clinical development and commercialization of oncology treatments leveraging Penroses novel mitochondrial modifying agent (MMA) technology

Penrose TherapeuTx, a privately held preclinical stage small molecule drug development company committed to improving patient lives through innovative oncology therapies, today announced the appointment of David Sherris, Ph.D. as President and Chief Executive Officer. Dr. Sherris has over 30 years of experience in translational medicine involving basic research, pharmaceuticals, and diagnostics, and has held numerous senior roles at prominent pharmaceutical companies including Serono, Centocor, Unilever Research and Mateon Therapeutics (formerly OXiGENE).

Penrose TherapeuTx, through unprecedented tissue-driven pharmacologic insights, has pioneered a next-generation class of MMAs which create a selective mitochondrial vulnerability that ultimately leads to cancer cell metabolic collapse. The platform has shown promising preclinical results and the company has developed a lead candidate, RP-0320, which is set to advance into clinical research in the coming year.

With Dr. Sherriss drug development skills, his experience in business development and securing funding for biotechnology companies, we feel confident that he will bring Penrose TherapeuTx to its next phase of success, said Mark de Souza, Chief Executive Officer and Board Member of parent company Tribar Healthcare Partners. We are pleased to bring on such an accomplished scientist and biotech entrepreneur.

Dr. Sherris is a proven serial entrepreneur having developed biotechnology companies from the ground up, devising corporate strategy, enacting programs, successfully established partnerships within the pharmaceutical industry and carrying companies through funding and acquisition. Holding senior positions in public and private companies, Dr. Sherris has led the development of novel therapies from the bench to the clinic. He has published multiple scientific papers and holds patents in a wide range of therapeutic areas including oncology, ophthalmology, dermatology, neurology, gene therapy and infectious disease.

Im excited to lead such an innovative biotechnology company and work with the talented team dedicated to advancing Penroses mitochondrial modifying agents. A first-in-class, small molecule drug technology attacking tumor cells leading to their necrotic death is an exciting and novel means to tackle cancer, said Dr. Sherris, Chief Executive Officer and President of Penrose TherapeuTx. I am grateful for the excellent work Mr. de Souza has done in his role establishing a solid foundation for the company. Im committed to working with the Penrose team, the U.S. Food & Drug Administration and other global regulatory agencies to bring this technology into the next stage of clinical development and ultimately to the market.

Prior to Penrose TherapeuTx, Dr. Sherris was President and Chief Executive Officer of GenAdam Therapeutics, Paloma Pharmaceuticals and VasculoMedics, private biotech companies. The former of which was acquired by PrimeCell and the latter two by RestorGenex, now Diffusion Pharmaceuticals. Dr. Sherris also had senior leadership positions at public biotech companies as Chief Operating Officer and Vice President of Research and Development at OXiGENE, along with Serono, Centocor and Unilever Research.

Dr. Sherris received his Ph.D. in Biochemistry and Molecular Genetics from the University of Utah, held a postdoctoral position in cellular immunology at the Jackson Laboratory and a faculty position in the Department of Medicine, Division of Clinical Immunology at the Mt. Sinai Medical Center, New York, NY.

About Penrose TherapeuTx

Penrose TherapeuTx is a U.S.-based pharmaceutical company focused on developing innovative small-molecule therapies for the treatment of advanced cancers. Penrose has pioneered the development of a novel Mitochondrial Modifying Agent therapeutic platform designed to generate therapies for difficult to treat cancers through a unique cooperative mechanism of action. Our approach has potential broad applicability across both hematologic and solid tumors. Learn more at http://www.penrosetherapeutx.com.

Excerpt from:
CORRECTING and REPLACING Penrose TherapeuTx Appoints Pharmaceutical Leader David Sherris, Ph.D., as President and Chief Executive Officer - Business...

Science in the early 20th century was undergoing many simultaneous revolutions. Radiological dating numbered the years of Earths existence in the billions, and eons of sediment demonstrated its geological evolution. The biological theory of evolution had become accepted, but mysteries remained about its selection mechanism and the molecular biology of genetics. Remnants of life dated far, far back, beginning with simple organisms. These ideas came to a head with the question of abiogenesis: could the first life have sprung from non-living matter?

In 1952, a graduate student named Stanley Miller, just 22 years old, designed an experiment to test whether the amino acids that form proteins could be created under the conditions thought to exist on the primordial Earth. Working with his Nobel Prize-winning advisor Harold Urey, he performed the experiment, which is now told time and again in textbooks all over the world.

The experiment mixed water and simple gases methane, ammonia, and hydrogen and shocked them with artificial lightning within a sealed glass apparatus. Within days, a thick colored substance built up at the bottom of the apparatus. This detritus contained five of the basic molecules common to living creatures. Revising this experiment over the years, Miller claimed to find as many as 11 amino acids. Subsequent work varying the electrical spark, the gases, and the apparatus itself created another dozen or so. After Millers death in 2007, the remains of his original experiments were re-examined by his former student. There may have been as many as 20-25 amino acids created even in that primitive original experiment.

The Miller-Urey experiment is a daring example of testing a complex hypothesis. It is also a lesson in drawing more than the most cautious and limited conclusions from it.

In the years following the original work, several limitations curbed excitement over its result. The simple amino acids did not combine to form more complex proteins or anything resembling primitive life. Further, the exact composition of the young Earth did not match Millers conditions. And small details of the setup appear to have affected the results. A new study published last month in Scientific Reports investigates one of those nagging details. It finds that the precise composition of the apparatus housing the experiment is crucial to amino acid formation.

The highly alkaline chemical broth dissolves a small amount of the borosilicate glass reactor vessel used in the original and subsequent experiments. Dissolved bits of silica permeate the liquid, likely creating and catalyzing reactions. The eroded walls of the glass may also boost catalysis of various reactions. This increases total amino acid production and allows the formation of some chemicals which are not created when the experiment is repeated in an apparatus made of Teflon. But, running the experiment in a Teflon apparatus deliberately contaminated with borosilicate recovered some of the lost amino acid production.

The Miller-Urey experiment was based on a complicated system. Over the years, many variables were tweaked, such as the concentration and composition of gases. For the purpose of demonstrating what might be plausible that is, whether biomolecules can be created from inorganic materials it was stunningly successful. But there wasnt a good control. We now see that might have been a pretty big mistake.

One of the elements of art in science is to divine which of innumerable complexities matter and which do not. Which variables can be accounted for or understood without testing, and which ones can be cleverly elided by experimental design? This is a borderland between hard science and intuitive art. It is certainly not obvious that glass would play a role in the outcome, but it apparently does.

A more certain and careful form of science is to conduct an experiment that varies one and only one variable at a time. This is a slow and laborious process. It can be prohibitively difficult for testing complex hypotheses like, Could life evolve from non-life on the early Earth? The authors of the new work performed just such a single-variable test. They ran the entire Miller-Urey experiment multiple times, varying only the presence of silicate glass. The runs performed in as glass vessel produced one set of results, while those using a Teflon apparatus produced another.

Systematically marching through each potential variable, one at a time, might be called brute force. But there is art here too, namely, in deciding which single variable out of many possibilities to test and in what way. In this case, we learned that glass silicates played an important role in the Miller-Urey experiment. Perhaps this means that silicate rock formations on the early Earth were necessary to produce life. Maybe.

Here is the original post:
What the famous Miller-Urey experiment got wrong - Big Think

(Salt Lake City, UT) The Utah Department of Health (UDOH) has sponsored COVID-19 testing since the beginning of the pandemic and has added hours, locations, and more tests to the testing options across the state. Tests offered through the UDOH are free and no appointment is necessary. Tests include Abbott ID NOW COVID-19 Rapid Molecular test (this is a Nucleic Acid Amplification TestNAAT), RT-PCR, and rapid antigen. PCR results are usually available within 24-48 hours; rapid PCR and rapid antigen results are usually available within 15-60 minutes. And, now, theres a test-at-home option for some people.

Live in rural Utah, or

Are unable to access a community testing site

Register onlineto see if you qualify for the free mail-based PCR test. It takes about four days to get your test results after registering. This includes the time it takes for you to get the test in the mail, take your sample at home, and mail it back to Fulgent Genetics. The test will be shipped to you by FedEx and must be returned to Fulgent Genetics by FedEx. Test results from the mail-based PCR tests are reported to the Utah Department of Health by Fulgent Genetics.

The UDOH also partnered with NOMI Health (TestUtah) to offer two designated lanes for travel testing In Salt Lake City and St. George. The testing locations are open from 7 a.m. to 7 p.m., Monday through Sunday. Testing for non-travelers is still available at both locations for all other members of the public. Not all travel destinations will accept the types of tests offered at these testing locations. Travelers are responsible for making sure they meet testing or vaccination requirements of their travel destination. For more information on travel testing and requirements, visithttps://coronavirus.utah.gov/travel/.

Excerpt from:
COVID-19 Testing in Utah is Easier than Ever | Utah Department of Health - Utah Department of Health

Biotech is a broad church. The term can be applied toany business that manufactures or uses products with a biological base and it can be difficult to keep up with the many scientific buzzwords in this rapidly expanding field. Help is at hand.

If you thought mining terminology was a minefield, the rich vein of biotech buzzwords will have you reaching for the dictionary.

It can be difficult to keep up with biotech terms when the field - fuelled by a raft of discoveries in genetics, biochemistry, molecular biology and other sciences that fall into the category - is expanding as quickly as the universe itself.

Biotech originated with the study of living organisms, which covers a vast array of sectors including agriculture, medicine, food and industrial technology.

The term can be applied toany business that manufactures or uses products with a biological base thismayincludetextiles,wood pulp products,food, biofuels and therapeutic drugs.

Gene technology drives a large part of modern biotech, with new technologies and products being developed every year.

This by-no-means complete list is designed to helpthe average investordistinguish theiralleles from theirantigens, andnavigate the world of biotechnology.

Active immunity:A type of immunitywhere resistance to a disease is built up byeither having the disease or receiving a vaccineforit.

Allele:One of two or more versions of a gene anindividual inherits two alleles for each gene, one from each parent. If the two alleles are the same, the individual ishomozygousfor that gene.

Antibody:A protein produced by the immune system that binds to a specific antigen.

Antigen/immunogen:Asubstance, usually a protein,that when recognised as 'foreign by the immune system will trigger an immune response, stimulating the production ofantibodies.When introduced into a vertebrate organism, an antigenis bound by the antibody or a T cell receptor.

Autologous cells:Cellsthat are taken from an individual, cultured (or stored), and, possibly, genetically manipulated before being introducedback into the original donor.

Biologics:Abiotechnology drug, such as a protein expressed by an engineered cell, or an antibody produced using recombinant DNA.

Biomanufacturing:The use of living cells to produce a biological product for examplea therapeutic proteinthat might be used in cancer treatment.

Biomarker:A measurable physiological event, such as the expression of a gene.Biomarkers are often used to indicate the presence or progression of a disease.

Biosimilar:Amedicine or biologic productachieved using a different processthan the one that originally produced theeffect, but which has a similar structure and function.

Blind trial:A trial where the participants do not know which treatment or intervention they have been allocated.

Cell:Thebuilding blocks of any living organism. Each celltypically containsgenetic material, an energy-producing system, and protein-makingability,encased in amembrane.

CAR T celltherapy:Chimeric antigen receptor (CAR) T-cell therapy uses immuneor white bloodcells, calledT cells,to fight cancer byaltering themso they can find and destroy cancer cells.

Companion diagnostic:A diagnostic used by physiciansto inform their prescribing decision.

CRADA(cooperativeresearch anddevelopmentagreement):Adeal in which two companiesagree tojoin resources to move a product concept or technology ahead big pharma will oftenpartner with emerging biotechcompaniesin a CRADA.

CRO(clinical researchorganisation):A supplier firm that offers contract servicesin testing, clinical trialsandmanufacturing.

Cytokinesis:Cytoplasmic division and other changes exclusive of the nuclear division thatoccurs throughmitosisormeiosis.

Cytology:The study of the structure and function of cells.

Deoxyribonucleic acid (DNA):DNA is a double-stranded helix held together by bonds between pairs of nucleotidesthe molecule that encodes genetic information.

Diagnostic:A test used to identifythe presence ofa disease or disorder, or to monitor the progression of treatment.Aroutine diagnosticis a broad screening tool;a specialty diagnostic screens for a specific disease.

Di-sulphidebond:A chemical bond thatstabilisesthe three-dimensional structure of proteins, andthereforethe protein's normal function.

Endpoint:In clinical trials, an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial.Endpointsare usually identified in the study objectives.

Epigenetic modification: Chemical modification made to a nucleotide base. Epigenetic modification can change the degree to which a gene is expressed. This occurs as part of normal development but irregularities are associated with various diseases.

Fission:Asexual reproductionwhichinvolvesthe division of a single-celled organism into two newequally sizedsingle-celled individuals.

Fusion protein:A proteinthat isexpressedby a gene developed through recombinant DNA methods from parts of genes encoding two or more proteins.

Genetically Modified Organism (GMO):Anyorganism whose DNA has been altered using genetic engineering techniques.

Genome:All of the genetic material in the chromosomes of a particular organism.

Genomicediting:A type of genetic engineering in which DNA is inserted, replacedor removed from agenome.

Haploid:A cell with half the usual number of chromosomes, or only one chromosome set. Sex cells are haploid.

Hapten:A small molecule which, when chemically-coupled to a protein, acts as an immunogen and stimulates the formation of antibodies not only against the two-molecule combination but also against thehapten.

Homozygous:Having two identical alleles for a particular gene.

Inducer:Asubstancethat increases the rate of enzyme synthesis, usually by blocking the action of the corresponding repressor.

Immune checkpoint inhibitors:Therapeutics that lift the inhibitions on T-cells in order to make them more likely to attack cancer cells.

INK(invariant natural killer)T cells:Cells thatrecogniselipid antigens presented bya cell-surface molecule known as CD1d. They have been shown to have important roles in many diverse immune responses.

Knockout:A technique usedto inactivate a particular gene in order to define its function.

Large-moleculedrug:Protein-basedtherapeutics these contain molecules that are too large to enter cells.

Leukocyte:Acolourlesscell in the blood, lymph and tissues that is an important component of the bodys immune system, which isalsoknown as awhite blood cell.

Lipoproteins:Agroupof serum proteins that transportfatsand cholesterol in the bloodstream. Abnormalities in lipoprotein metabolismcan feature inheart diseases.

Master cellbank:Asingle pool of cells that generally has been prepared from the selected cell clone under defined conditions.

Microbiome:An ecosystem ofmicrobes, their genomes and environmental interactions in a particular environment.

Mitosis:Process of cell reproduction whereby the daughter cells are identical in chromosome number to the parent cells.

Meiosis:Meiosis is the division of a germ cell into four cells, each with half the number of chromosomes of the parent cell.

Monoclonalantibody:An antibody produced by a single clone of cells, which therefore consistently binds to the same epitope of an antigen.

Mutationbreeding:Genetic change caused naturally or using mutagens. Stable mutations in genes are passed on to offspring, while unstable mutations are not.

Nanotechnology:The understanding and control of matterat dimensions between approximately 1 and 100nanometres, with myriad applications across science,energy and evendefence.

NDA:A new drug application is the mechanism by which drug companies formally propose that theUSFood and DrugAdministration (FDA) approve a new pharmaceutical for sale and marketing.

Nucleotide:A nucleotide is the basic building block of nucleic acids.RNAandDNAare polymers made of long chains of nucleotides.

Nutraceutical:Term coined by the Foundation for Innovation in Medicine in 1991referring toany substance that may be considered a food or part of a food and provides medical or health benefits, including the prevention and treatment of disease.

Orphandrug:Drug developed for a condition that affects fewer than 200,000 individuals(in the US).

Peptide:Peptides areshort strings of amino acids, typically comprising up to 50 amino acids. Amino acids are also the building blocks of proteinsbut peptides may be easier for the body to absorb than proteins because they are smaller and moreeasilybroken down.

Pharmacodynamics:The study of theeffect ofadrugon the bodyas it relates to increasing dose.

Pharmacogenomics:Thestudy ofthe correlation between an individuals genetic make-upand their response to drug treatment. Some drugs work well in some patient populations and notothers.

Pharmacokinetics:Thestudy of the time course of drugs lifespan in the body, from absorption to distribution, metabolism and excretion.

Phase 1clinicaltrials:The first stage of the testing of a new drug or process on human participants.Investigators areinterested in learning aboutsafety,side effects and dosage levels.

Phase 2clinicaltrials:Smalltrials on people who have themedical condition under investigation. The objective is to determine whether the experimental drug provides a beneficial effect.There is usually a control group the receives a placebo alongside thetreatment group who receive the drug.

Phase 3clinicaltrials:Larger,carefully controlled long-term studies on patients to determine whether the drug will be effective in normal medical settings.During Phase 3, information is gleaned onlong-term side effects and safety.

Pre-clinical testing:This generally means testing in animals, which in medicine generally happens asa precursor toPhase 1 to 3testing.

Quantitative Reverse Transcriptase PCR:A technique used to measure the levels of gene expression of a particular gene.

Rationaldrugdesign:The development oftherapeutic drugsbased on an understanding of the underlying disease mechanism.

Receptor:A region of tissuefound on the surface of a cell that responds to a specific chemical messenger, such as a neurotransmitter, antigen or hormone.

Recombinant:Relatestoanorganism, cell, or genetic material formed by recombination.

RNA:Ribonucleic acid (RNA) isa molecule similar to DNA.Molecular biology suggests that the primary role of RNA isto convert the information stored in DNA into proteins.

RNAinterference (RNAi):A technique usedin scienceto block the expression of a particular protein.

Selective mediumnutrient:Matter thatwill support the growth of specific organisms while inhibiting the growth of others.

Sequence:The order of nucleotides in a DNA or RNA molecule, or the order of amino acids in a protein.

Signallingmolecule:A molecule that binds to a cellular receptor, whichresults in the cell starting or stopping the production of a particular protein.

Small molecules:Molecules madebychemistry into traditional pills, or by training cells to produce proteins that can be used as medications.

Spike protein:Aspike protein, used in vaccines,is aproteinthat forms a large structure known as aspikeorpeplomerprojecting from the surface of the enveloped virus. It is located on the outside of the virus sothe immune system canrecogniseit easily.

Stemcell:Stem cells are undifferentiated human cells that have the ability to self-renew and develop into many different cell typesin response to extracellular cues.

T-Cell:An immune system cellthat can be usedtorecognisespecific pathogensbecause ofthe shape of its cell-surface receptor.

Targetvalidation:Determining if targeting a particular moleculethat mightbeinvolved in a disease mechanism will be a safe and effective means of therapy.

Transgenicorganism:An organism whose genome has been altered by the incorporation of foreign DNA.

Unipotent:A moleculecapable of developing into only one type of cellor tissue.

Unblinded results:Unblinding, sometimes referred to as code-break, is the process by which the treatment details that form part of a study are made available eitheron purpose or by accident.

Upstreamprocessing:The phase of biomanufacturing consistingof establishing cell banks and seeding and scaling up cell cultures.

Viral vaccines:Vaccines consisting of live rather than deador separated parts of viruses. The virus itself is genetically engineered so that it elicits the immune response to the viral pathogen without causing the disease itself.

Vector:A vehicle for the transfer of DNA from one organism to another.

Working cell bank: A vialed collection of cells that are derived from the master cell bank.

X-chromosome:A chromosome associated with sex determination -the female has two X-chromosomes,whilethe male has one X-chromosome and oneY-chromosome.

Xenobiotic:A chemical compoundnot produced by living organisms amanufactured chemical compound.

Y-chromosome:The partner of the X-chromosome in the male of many animal species.

Zygote:A diploid cell formed by the fusion of two haploid gametes during fertilization the first cell of the neworganism.

More:
How to speak biotech: An A-Z guide - Proactive Investors Australia

VANCOUVER, BC, Nov. 18, 2021 /PRNewswire/ --Advances in the collective genetic understanding of diseases, and the ability to identify disease biomarkers, is ushering in a new era of personalized medicine. Technologies such as CRISPR/Cas9 are also paving the way for improved, more tailored treatments targeted to a specific genetic marker of a disease. As our understanding of the molecular underpinnings of disease continue to improve, so, too, will the technologies at our disposal to treat them.

We've already seen the benefits of this type of personalized medicine in the cancer realm. Using a person's (or disease's) genes to drive cancer therapy is known as precision medicine. Precision medicine can help doctors identify high-risk cancer patients, choose treatment options, and evaluate treatment effectiveness. Precision medicine can also be used to prevent certain types of cancer, diagnose certain types of cancers early (leading to earlier treatment and better outcomes), and diagnose specific types of cancer more correctly.

As targeted therapies continue to advance, we will continue to see their impacts flow beyond that of the cancer realm. One area in which interest is ramping up is neurodegenerative diseases, which are chronic, progressive diseases affecting the brain and its constituent cells. Neurologic disease can be genetic, or caused by a stroke or brain tumor. Examples of neurodegenerative disease include Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease. These diseases have a genetic component, with specific genes playing a role in the development and progression of disease, especially in rare forms. Neurodegenerative conditions, like cancer, are devastating and costly. Collectively, neurodegenerative conditions cost people in the United States $655 billion in 2020.

Can we apply concepts from targeted therapies developed for cancer to create better outcomes for patients suffering from neurodegenerative diseases? What's more, can precision medicine be used to treat other large unmet needs in the field of neurology, such as neuropsychiatry, pain, epilepsy, sleep disorders, and stroke?

Precision medicine in neuroscience and neurology is where many companies have dedicated their time and efforts. Three companies trading on the NASDAQ in this space that investors should research are Alnylam, Ionis Pharmaceuticals, and Regeneron.

Neuroscience research companies are clamoring to make use of the plethora of cellular and molecular biology data that is emerging about drugs and the patients who use them. There is much more information to be gleaned from diseases and patients than the genetics, which may not reveal information about the ways that genes are formally transcribed and expressed. Emerging technologies, therefore, also look at the RNA profiles of a drug response, patient, or disease state, called transcriptomics; and the set of proteins expressed by a cell, tissue, or organism, called proteomics. While a challenge with gene therapy is reimbursement by insurance providers, research is underway that can make gene therapies more common, and pave the way for more established insurance structures.

RNA targeting is an active area of research for neurodegenerative disease, with companies such as Skyhawk Therapeutics, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, and Takeda involved. By modifying genetic transcription via RNA technologies, these companies hope to develop novel treatments for disorders of the central nervous system. The study of RNA profiles in a given cell, tissue, or organism is known as transcriptomics, and this area will likely heat up as these researchers work to develop pioneering RNA technologies to target neurodegenerative disease.

Proteomics, or the study of the proteins expressed by a cell, tissue, or organism, will also play a role in precision medicine for neurological disorders. In June 2021, the United States Food and Drug Adminstration approved the first therapy addressing the underlying biology of Alzheimer's disease. The drug, Biogen's Aducanumab, is a monoclonal antibody therapy that works by clearing a substance known as beta-amyloid, a protein that scientists believe causes Alzheimer's, from the brain. The drug, which was found to exhibit a unique proteomic profile upon treatment in mice, was the first approved for Alzheimer's in 20 years, and while it is thought to be effective in a limited number of Alzheimer's disease cases (namely, people in the early stages of Alzheimer's), it represents a step forward in neurodegenerative disease research.

The FDA's approval of Aduhelm, which was under an accelerated timeframe, has created more interest in the area of Alzheimer's and Parkinson's disease treatments. Scientists believe that a protein called tau is more closely associated with dementia than beta-amyloid, so they are also seeking to develop drugs targeting tau protein. In the realm of Parkinson's disease, research is underway to target a compound called alpha-synuclein, which, like amyloid beta and tau protein in Alzheimer's, is associated with cognitive decline in Parkinson's disease. There are a number of approaches in development to target tau. Investors can expect many more biotech companies and venture firms moving into this space to develop innovative and alternative treatments.

This work is not without significant challenges. One obstacle in neurodegenerative research is creating drugs that can bypass the brain's blood-brain-barrier, which keeps the brain safe from toxic substances or pathogens that would otherwise make their way into the brain. Another challenge is the fact that neurodegeneration affects a subset of neurons, which may have different levels of vulnerability to such disease. It is not yet fully clear which factors predispose certain neurons to develop pathology over others.

Yet as drug discovery continues to leverage the latest techniques in genomics, transcriptomics, and proteomics, and combinations of these technologies, this will unlock new potential for companies to create novel, increasingly personalized, therapies. For example, advances in genomics may provide insight into how neurodegeneration occurs in the brain.

Drug discovery in neurodegeneration also overlaps with that of other diseases, due to common disease pathways. For example, phosphatidylinositol 3-Kinase (PI3K) inhibitors are implicated not only in COVID-19 and breast cancer, but also Parkinson's Disease. Stem cell therapies, which could benefit patients suffering from many conditions, can also have significant applications in the neurodegenerative realm. Stem cells could potentially be used to restore lost brain tissue, or to release compounds such as anti-inflammatory factors and growth factors supporting repair of the nervous system. Stem cell therapies, which are already in use for conditions such as cancer, could thereby restore function to neurodegenerative patients. Therefore, advances made in the treatment of other disease states could potentially innovate the field of neurodegeneration as well.

PRLog ID: http://www.prlog.org/12894142

SOURCE Braeden Lichti

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Braeden Lichti - Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases - PRNewswire

Last year, as Americans facing the threat of COVID-19 hunkered down and masked up, the flu seemed to go into hibernation. At the height of a typical flu season, up to 1 visit in 20 to emergency departments is for the illness. But during the most recent flu season, it accounted for less than 1 of every 1,000 emergency room visits.

Experts expect that the flu will make a comeback this winter, circulating along with other seasonal respiratory viruses as well as the coronavirus. Heres what you need to know to protect yourself and your family.

Influenza is a notoriously difficult virus to predict. The past year with fewer cases means there might be a lower level of immunity in the general population, says Lynnette Brammer, an epidemiologist in the influenza division at the Centers for Disease Control and Prevention. This could affect children more than adults because adult immune systems have had decades of exposure to different flu viruses.

On top of that, COVID-19 will still be with us, especially because new and more contagious variants of the coronavirus that causes COVID-19 are continually emerging. So its possible we could see the flu and COVID-19 spreading at the same time, a situation feared by some scientists last year.

It really comes down again to behavior and whether people continue taking the steps to avoid respiratory diseases that so effectively limited the spread of flu last season, says Sarah Cobey, an associate professor of ecology and evolution at the University of Chicago.

Consider vaccines as the first line of defense. Theyre not available for all seasonal respiratory viruses, including many that cause the common cold. But you can be vaccinated against two of the riskiest viruses that will be circulating: influenza and the coronavirus.

In a May study published by the CDC, researchers found the Pfizer and Moderna vaccines were 94% effective at preventing hospitalizations for COVID-19 among people ages 65 and older. In comparison, the flu vaccines strength might seem low: The shot was 39% effective at keeping people from needing to see a doctor for a case of the flu during the 2019-2020 flu season, the last year for which CDC data is available.

But just as with the coronavirus vaccines, flu shots also reduce your risk of serious illness or hospitalization if you do get sick. For example, the CDC estimates that in the 2019-2020 flu season, vaccinations averted about 61,000 hospitalizations among those 65 and older and 105,000 hospitalizations overall. So take these steps now:

Get a coronavirus vaccine. If you havent had a shot yet, get one as soon as you can. The CDC says you can even get a COVID-19 shot and a flu vaccine during the same visit. And millions of people are now eligible for a booster shot. Get one if you are.

65 or older? Seek out the best flu vaccines. Two have been shown to provide better protection for older adults compared with the standard vaccine, and theyre available only for those 65 and older. The Fluzone High-Dose contains four times the amount of viral antigen (the molecule that stimulates an immune response) as the standard shot.

The other vaccine, Fluad, is made with an additive thats designed to prompt a stronger response from the immune system. If you cant get one of those shots, a standard flu vaccine is still better than none at all.

Time your flu jab right. Flu vaccine effectiveness wanes over the course of the season, especially for older adults. Get your shot as soon as you can if you havent yet, and remember that it takes a few weeks for protection to kick in.

If youre over 65, go for a pneumococcal vaccine if you havent yet. The bacteria Streptococcus pneumoniae is one of the most common causes of bacterial pneumonia. Vaccines are available against this bacteria, which can also cause sinus infections and meningitis. The CDC recommends that everyone 65 and older receive a dose of PPSV23 (Pneumovax 23).

With COVID-19, the flu and colds likely to circulate together this winter, youll need to practice additional virus-fighting habits.

Hang on to your mask. Whatever the current rules are where you live, remember that wearing a mask can help protect you from COVID-19 including breakthrough infections and may shield you from other respiratory viruses. (Even with a mask, keep a distance from anyone coughing or sniffling.) While the flu can spread through surfaces and large droplets (as from a sneeze), it can also be transmitted via small particles in the air, just like the novel coronavirus.

The CDC doesnt actively recommend mask use for preventing the flu, but if you have any respiratory symptoms or are headed into a crowded environment an airplane, a busy store, a big event wearing a mask is a reasonable precaution to take, says Seema Lakdawala, an assistant professor in microbiology and molecular genetics at the University of Pittsburgh School of Medicine, who studies flu transmission.

Thats especially true if youre at higher risk for severe disease because of your age or an underlying condition.

Wash your hands. Cleaning your hands regularly with soap and water for at least 20 seconds helps prevent a wide range of diseases, not only those caused by respiratory viruses. When you dont have access to a sink, use hand sanitizer with at least 60% alcohol.

Stay home when youre sick. Many people developed at least one good habit during the pandemic: staying home the minute they had any respiratory symptoms, Lakdawala says. That meant not going to work, visiting friends or even stopping in a store. This probably helped limit the transmission of many viruses in addition to the coronavirus, she adds.

If you start to notice symptoms of any viral illness fever, cough, chills, sore throat, runny nose, congestion seek out testing, for the novel coronavirus at least. The flu, COVID-19 and colds can cause symptoms that make telling one disease from the others difficult. Thats one reason its important to get tested early and take precautions such as isolating yourself from others.

Early testing can also help facilitate prompt treatment, which is critical for the flu and COVID-19. If you have the flu and are at an increased risk of complications (because of age or an underlying illness such as asthma or diabetes), antiviral medications are recommended.

These can help reduce the severity of symptoms, but meds are most effective if you start taking them within two days of noticing the first signs. Early treatment with monoclonal antibodies and other medication may improve your prognosis if you have COVID-19.

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For this winter, here are the best ways to avoid COVID-19, the flu and colds - The Spokesman-Review

As a young child, Professor Michele Nishiguchi would dive off the couch and into the shag carpet reefs of her family home, mimicking the turns and leaps of intrepid explorer and environmentalist Jacques Cousteau. This love of the ocean would remain with Nishiguchi as she progressed through her academic career.

Now a professor in the Department of Molecular and Cell Biology in the Schoolof Natural Sciences, her lab studies the relationship between ocean microbes and Bobtail squids.

Looking around the lab, with its ocean microbes in frosty freezer vials and precious squid specimens preserved in jars, one would think Nish and her team are marine biologists. But Nish, as she likes to be known, describes herself as an evolutionary biologist who studies the impact of microbes on organisms and how microbes interact with the world around them.

So why ocean microbes and squid?

Their relationship is dynamic and fascinating. The two organisms are so evolutionarily far apart but still able to communicate and interact with each other for mutual benefit and survival, Nish said.

At UC Merced, Nish and her team research the dynamic relationships between the Bobtail squid and Vibrio bacteria. There are approximately 20 labs nationally that use this species/host model but only Nishs lab is investigating all the different species of Vibrio and their coevolution with their squid hosts across various locations.

Just like the beneficial microbes living in the human gut and affecting digestion and nutrient uptake, Bobtail squid harbor special ocean microbes, bacteria in the genus Vibrio. These bacteria are taken up by the squid into specialized light organs, where they have a safe niche to multiply and provide the squid with a luminescent glow. Its a delicate balance that requires communication between the bacteria and the squid: Too much growth could harm the host and too little means no benefit.

Though humans dont gain luminescence, we do strengthen our gut health when we consume probiotic bacteria from foods such as yogurt or kombucha. The bacteria break down food molecules that we cant on our own. They colonize our gastrointestinal tracts and deter disease-causing, harmful microbes from establishing. As with the squid, even healthy bacteria can overgrow and cause harm, leading to gut irritation, inflammation and sometimes, chronic disease.

By studying the relationship between Bobtail squids and various Vibrio bacteria species, Nishs lab hopes to discover how communication between squid and bacteria take place, how the relationship between host and microbe evolves over time, and what genes might regulate these interactions.

Only certain species of Vibrio can thrive and colonize the Bobtail squids light organ, indicating an evolved preference for one species or strain over the other, Nish and her students discovered. Examining this relationship sheds light on how host organisms manage and select beneficial microbes and what communication mechanisms are involved.

For Nish, coming back to California meant coming full circle She is a product of the UC system and a proud recipient of three UC degrees: her bachelors degree at UC Davis, masters degree at UC San Diego, and PhD at UC Santa Cruz.

But she has been all over the world studying squid. She relocated her lab from New Mexico to UC Merced during the COVID-19 pandemic, setting up a new squid hatchery to raise the model organism, reestablishing protocols and restarting experiments from scratch. Some lab members drove the precious frozen bacteria cell strains, preserved on dry ice, from New Mexico to California.

Just as there is more than meets the eye with the Bobtail squid, Nish and her team are multifaceted, exhibiting expertise from a range of fields such as microbiology and evolutionary genetics. Nish hopes eventually, her labs findings could lay the foundation for answering questions about humans and microbes.

Perhaps, one day, the bobtail squid and Vibrio will help us answer complex questions regarding our own relationships with microbes, Nish said.

Excerpt from:
Exploring Microbial Interactions with Glowing Squid | Newsroom - UC Merced University News

Nancy Hopkins, an MIT professor who has made significant strides in molecular biology and a tireless advocate for gender equity in science, was named the recipient of STATs 2021 Biomedical Innovation Award on Tuesday.

Its very easy to forget how much progress there has been because we havent arrived where wed like to be. So we see the problems that still lie ahead. But you periodically have to pause and say, Oh, my gosh, look how far we came, said Hopkins at the 2021 STAT Summit, where she was honored for her work. The STAT award, now in its third year, honors biology and medicine researchers whose work has helped define their field. Hopkins was selected by STAT editors with input from outside experts, and received the award during the annual STAT Summit, a three-day event focused on health care. She is the second woman to receive the award, which was given last year to CRISPR researcher Jennifer Doudna.

Were in a period of dramatic advances and daunting problems, said Matthew Herper, STATs senior medicine writer and editorial director of events. Society needs every great scientist we can train. And that means that we need women every bit as much as men. Hopkins advocacy in this area has been instrumental in starting to create a more level playing field a project on which there is still much to do.

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By the mid-1990s, Hopkins had worked at MIT for 20 years, but still found herself one of a small proportion of women faculty in science at MIT. She had seen no woman professor become head of an MIT science department, center, or lab. There were no women administrators in science or engineering.

I thought it was a choice, once the door opened, Hopkins said. And I think we were all surprised to discover that behind the door that had opened were a whole series of obstacles that we really hadnt thought about.

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Over time, her illusions faded so all she could see was a perplexing problem. So she did what any good scientist does: She studied it. She surveyed female colleagues about their experiences at MIT and then went big, chairing a committee that produced landmark reports exposing gender-based discrimination, structural sexism, and pay disparities across MIT departments.

But unlike some work that emerges from higher ed committees only to languish in a file cabinet, the committees work forced MIT administrators to confront reality by making the gender inequity issue in each of its schools front and center. In response, deans and provosts aggressively recruited women for faculty positions, opened an on-campus day care and fixed pay gaps treating the problem as a structural issue instead of a case-by-case situation. The findings were highlighted in the media, prompted other institutions to interrogate themselves, and sparked conversations about pervasive discrimination against women in the world beyond academia.

If one were to ask what was the most important factor in change to date, it would have to be the Reports that documented the problems and led to the engagement of administrators in solving them, Hopkins wrote about MITs progress in a subsequent report.

Hopkins has continued to push for the inclusion and recognition of women in science, technology, engineering, and math fields. As a biology professor and researcher, she watched the biotech industry be born and boom and saw how women were systematically excluded from it, barred from contributing their expertise. So Hopkins worked with MITs Sangeeta Bhatia and Susan Hockfield, and co-founded the Boston Biotech Working Group to foster women faculty as founders and board members of biotechnology companies.

While working to make science a viable career for more women, Hopkins was also doing groundbreaking genetics research. As a young scientist, she focused on identifying RNA tumor virus genes and unraveling how they were correlated to the severity of cancers. She turned her attention a decade later to a different study of cancer, becoming one of the first scientists to use zebrafish as a model to understand how genetic mutations contribute to the development of diseases such as cancer. What started as an attempt by Hopkins to switch fields in search of a more equitable department led to major scientific breakthroughs, even though she was denied adequate space and resources for her zebrafish work.

Social change is a fascinating thing, she told STAT. Why does it take so long? Were changing peoples brains. I think these unconscious biases are so deeply embedded in our brains. Why did it take me 20 years to figure out what was going on? You come in with the belief that science is a meritocracy.

Hopkins is a member of the National Academy of Sciences and the National Academy of Medicine, and has also been awarded the Harvard Centennial Medal, UCSF Medal, and MD Andersons Margaret L. Kripke Legend Award, among other honors. She is a 2021 fellow with the American Association for Cancer Research Academy.

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Nancy Hopkins wins STAT Biomedical Innovation Award - STAT