Category Archives: Human Genetics

The latest report study collected and published by Apex Market Research evaluates the historic and present-day scenario of the Gaucher Disease Drugs market to precisely measure its growth potential. The report study on Gaucher Disease Drugs market offers in-depths details about market revenue, trends, share, key growth factors, potential restraints/treats, and opportunities that are generating the platform for the growth of the global Gaucher Disease Drugs market. The report provides useful information about the current market scenario and its revenue for base year. Also offers a perceptive about how the Gaucher Disease Drugs Market would perform over the forecast period of 2021 to 2028. The global Gaucher Disease Drugs market size is projected to reach around US$ xx Million from its value of US$ xx Million in 2020. During the conjecture period of 2021 to 2028, the global Gaucher Disease Drugs market is anticipated to grow at a CAGR of xx% over the forecast period.

Checkout Free Report Sample of Gaucher Disease Drugs Market Report for Better Understanding: https://www.apexmarketsresearch.com/sample/1064275

Major Industry Competitors:

COVID-19 Impact analysis

The current report published by Apex Market Research covers a detailed analysis of ongoing COVID-19 pandemic conditions on market performance. The effect of COVID-19 pandemic is evaluated at all steps of the market that will provide useful insights to the reader regarding current ups and downs of the market revenue, effect of the sale of the product, government policies, supply chain, and distributors scenario. With these stats, a reader can understand and predict the market flow over the forecast period. Also, it will help an investor to take progressive steps in terms of investments or expansion of business by understanding the high demand zones.

Regional Analysis

The regional analysis covered in the Gaucher Disease Drugs market report bifurcates the market in North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The report provides a detailed analysis at regional as well as country-level analysis, that covers recent market trends in the region, major driving factors for the growth of the market in a particular country for the respective region, and opportunities in the untapped market. Regional stats provided in the systematic form will easily help start-ups and small market players to understand the market expansion opportunities.

Segmentation

The detailed segmentation in the report is constructed by understanding every element of the market. The market segment and their subsegments offer comprehensive information to the reader that will help to understand the most lucrative segment in the market and the reasons for the slow growth or decline in other market segments. Statistical analysis provided in segmentations by graphical presentations will assist reader for easy understanding of market scenario this will help to make effective strategic decisions for investments.

By Disease TypeType IType IIType IIITreatment TypeEnzyme Replacement TherapyERTSubstrate Reduction TreatmentSBT

Enquire Here for, Report Enquire, Discount and Customization: https://www.apexmarketsresearch.com/enquiry/1064275

Manufacturers Information

The report on Gaucher Disease Drugs market covers ten key market players operating on a global platform. These market players are selected based on their net sale/revenue, geographical presence, mergers & acquisitions, business segments, market shares, and R&D spending. The competitive analysis in the report will help an investor to know their closest competitors, their global presence, and their marketing strategies. This will assist the reader in better decision-making to stay ahead in the competitive market.

Who will get benefit of this report?

Large manufacturers, small and medium investors, private equity firms, government organizations, suppliers, as well as retailers that are operating in the Gaucher Disease Drugs market can get the benefit from the report published by Apex Market Research. The report provides market analysis in a simplified manner that is easy to read and understand for the users. This will also assist startups to understand every component of the global Gaucher Disease Drugs market.

Why should buy this report from Apex?

The report on Gaucher Disease Drugs market by Apex Market Research is a methodical assessment involving various factors that are relevant to the market growth and dynamics. The important facts and data covered in the report for Gaucher Disease Drugs market for the forecast period of 2021-2028 will assist as a valuable document for the user looking for guidance in decision making to reinforce their current position in the market or planning to enter the market.

Access Full Report information with TOC, here: https://www.apexmarketsresearch.com/report/gaucher-disease-drugs-market-1064275/

Report Methodology

The global Gaucher Disease Drugs market report is constructed based on a detailed analysis of current market trends, investor presentations, government reports, industry insights, driving factors, opportunities, revenue pockets, geographical market scenario, etc. All this information is collected via thorough secondary research which is again validated by intense primary research.

About Us

We at Apex Market Research aim to be global leaders in qualitative and predictive analysis as we put ourselves in the front seat for identifying worldwide industrial trends and opportunities and mapping them out for you on a silver platter. We specialize in identifying the calibers of the markets robust activities and constantly pushing out the areas which allow our clientele base in making the most innovative, optimized, integrated and strategic business decisions in order to put them ahead of their competition by leaps and bounds.

Contact Us

1st Floor, Harikrishna Building,Samarth Nagar, New Sanghvi,Pune- 411027 India+91-8149441100 (GMT Office Hours)+17738002974sales@apexmarketsresearch.com

Link:
Gaucher Disease Drugs Market by Manufacturers, Regions, Type and Application, Forecast To 2026 Genzyme Corporation, Pfizer, Inc., Shire Human...

Personalized nutrition has emerged in recent years as a key potential solution to a variety of diseases that originate in the gut, and Nebraska scientists are mining microbiome data to determine how individuals digestive systems might respond to different nutritionalapproaches.

Ultimately, these findings could help scientists and doctors recommend specific types of foods say, yogurts to individuals to nurture beneficial bacteria in their guts and stave off diseases such as diabetes andobesity.

Yanbin Yin, associate professor of food science at the University of NebraskaLincoln, has received a four-year, $1.2 million grant from the National Institutes of Health to continue his research. Yins lab develops computational models and informatics tools to identify carbohydrate-active enzymes CAZymes in the gut microbiome that can build, modify and break down various complex carbohydrates. These CAZymes are made by our gut bacteria to fully digest fibers in our diets. Bacterial digestion of these fibers produces metabolites, such as short-chain fatty acids, that have a significant influence on their human hosts health, Yinsaid.

However, every human individual has different microbiome compositions and thus may respond to the same dietary fibers differently, Yin said. We aim to develop machine learning tools to help predict which human individuals may respond to which dietary fibers by analyzing their gut microbiome DNAsequences.

Yin and his co-investigator, Yuzhen Zhou, assistant professor of statistics at Nebraska, will develop new computer software that can automate the data mining of these sequencing data for CAZymes and their gene clusters. Finding these gene clusters will contribute to addressing two fundamental personalized nutritionquestions:

Creating new bioinformatics tools for predicting carbohydrate use will contribute to the arising microbiome-based personalized nutrition practice and aid in the development of therapeutic options to prevent and treat human metabolicdisorders.

The software Yin is creating can analyze and classify CAZymes at a more detailed level than ever before. It looks for key features within genetic code to distinguish among different CAZyme groups and predict how the enzymesfunction.

The computer algorithms learn and improve as data is added. Yin started his research with a CAZyme database, CAZy, compiled from the scientific literature and maintained by other researchers. Hes been using the database to train his CAZyme discovery software and has packaged the software into a free, user-friendly website, dbCAN, for CAZymeresearchers.

Yin has more than 10 years of experience in developing CAZyme bioinformatics tools and maintains the well-recognized CAZyme annotation database and web server. His CAZyme bioinformatics research has been funded by an NSF CAREER award.

Yins work is affiliated with the Nebraska Food for Health Center, launched in September 2016, which brings together faculty researchers from the University of NebraskaLincoln, the University of Nebraska Medical Center and the University of Nebraska at Omaha to tie gastrointestinal and biomedical research to agriculture, plant and animal breeding, andgenetics.

View post:
Researchers intend to discover microbiome's role in personalized nutrition - Nebraska Today

BACKGROUND

Charles Darwins The Descent of Man, published on 24 February 1871, laid the grounds for scientific studies into human origins and evolution. We look at the advances in our understanding of these processes through the lenses of modern speciation theory. Applying this theory to specific cases requires one to identify and understand the nature of (i) the ancestor and various preexisting adaptations and traits that it possessed that allowed or simplified the speciation process, (ii) evolutionary forces responsible for major differences between the emergent species and its close relatives, and (iii) the most salient adaptations characteristic of the new species and its evolutionary history (such as genetic, morphological, behavioral, spatial, and temporal).

Modern research shows that we share many developmental, physiological, morphological, cognitive, and psychological characteristics as well as about 96% of our DNA with the anthropoid apes. We now know that since our last common ancestor with the other apes 6 million to 8 million years ago, human evolution followed the path common for other species with diversification into closely related species and some subsequent hybridization between them. Since Darwin, a long series of unbridgeable gaps have been proposed between humans and other animals. They focused on tool-making, cultural learning and imitation, empathy, prosociality and cooperation, planning and foresight, episodic memory, metacognition, and theory of mind. However, new insights from neurobiology, genetics, primatology, and behavioral biology only reinforce Darwins view that most differences between humans and higher animals are of degree and not of kind. What makes us different is that our ancestors evolved greatly enhanced abilities for (and reliance on) cooperation, social learning, and cumulative culturetraits emphasized already by Darwin. Cooperation allowed for environmental risk buffering, cost reduction, and the access to new resources and benefits through the economy of scale. Learning and cumulative culture allowed for the accumulation and rapid spread of beneficial innovations between individuals and groups. The enhanced abilities to learn from and cooperate with others became a universal tool, removing the need to evolve specific biological organs for specific environmental challenges. These human traits likely evolved as a response to increasing high-frequency climate changes on the millennial and submillennial scales during the Pleistocene. Once the abilities for cumulative culture and extended cooperation were in place, a suite of subsequent evolutionary changes became possible and likely unavoidable. In particular, human social systems evolved to support mothers through the recruitment of males and nonreproductive females. The most distinctive feature of our species, language, appeared arguably driven by selection for simplifying cooperation. Reliance on social learning and conformity led to the emergence of new factors constraining and driving human behavior, such as morality, social norms, and social institutions. These forces often act against the immediate biological or material interests of individuals, promoting instead the interests of the society as a whole or of its powerful segments. Continuous engagement in cooperation has led to the evolution of strong coalitionary psychology, which can bring us together whenever we perceive that our identity group faces outside threats. Coalitionary psychology also has an undesirable byproduct: often negative or even hostile reaction to others who differ from us in their looks, behaviors, beliefs, caste, or class.

Our society faces challenges, including climate change; various types of inequality; economic crises; political, cultural, and religious conflicts; and pandemics. Similar challenges have repeatedly arisen and were dealt with in the past with varying success. What makes the current situation different is not only the scale of societal threats but also that modern science can provide guidance on how to respond to them. Adequately answering these challenges requires understanding humans social behavior and the roles of cooperation, social learning, and culture for human decision-making. Evolutionary perspective is already helping to synthesize the contributions of social sciences, including anthropology, psychology, economics, political science, and history. The impact of Descent on the social sciences and on the development and implementation of different policies by practitioners and policymakers to improve our society will only grow.

(Left) Organic evolution and (right) cultural evolution, as depicted in Alfred L. Kroebers 1923 textbook Anthropology: Cultural Patterns and Processes. Biological inheritance is rigid from parents to offspring in eukaryotes, and species mostly do not exchange genes. Culture is potentially acquired from anyone in a persons social network, and ideas spread rather readily from culture to culture.

Original post:
Modern theories of human evolution foreshadowed by Darwin's Descent of Man - Science Magazine

Polaris Market Research conducts an in-depth analysis of the Gaucher Disease Treatment Market to evaluate business solutions, evaluation, R&D, Industry shares, size, benefits, advantages, scope and operations. The report provides in-depth analysis and development of the industrys major manufacturers, major driving factors, opportunities, challenges, international suppliers, risks and entry barriers. It also provides competitive analysis on the latest technology, trends, innovation, future scope, industry share, sales, revenue generation, investment analysis and overall business scenarios.

The Gaucher Disease Treatment market research report also provides an analysis of the market share of important stakeholders. This qualitative and quantitative analysis include key products, key differentiating factors, revenue share, market size, market conditions, and leading manufacturers strategies. The report will also soon cover key agreements, associations and global partnerships to change market dynamics on a global scale.

Get More Details of the report (Download Sample Copy) : https://www.polarismarketresearch.com/industry-analysis/gaucher-disease-treatment-market/request-for-sample

The research goals of the Gaucher Disease Treatment Market are:

Competitive structure and analysis of Gaucher Disease Treatment Market:

1) Constant Growth, Expanding Margins

Some companies have excellent growth records from 2014 to 2020. Some of these companies have shown tremendous growth in sales and revenue, while net income in the same period has doubled as performance and gross profit margins increase.

The Gaucher Disease Treatment report further performs functional analysis, which contains detailed information on the companys manufacturing base, production volume, size, value chain, and product specifications.

2) Devised Growth Plans & Rising Competition?

Gaucher Disease Treatment market participants have established a strategy to provide a large number of new product launches within several markets around the world. To confirm the comprehensive practice, some player profiles worth reviewing are Acetelion Pharmaceutical (J&J Ltd.), Shire Human Genetics Therapies, Inc., Erad Therapeutic Inc., and JCR Pharmaceuticals Co Ltd.

3) Status Of The Market In TodayS World

Although the market segmentation has made considerable gains and may not be so encouraging in recent years, the situation may be better if manufacturers can take plan-driven initiatives earlier. It is different from the past, but it is estimated that the investment cycle in the United States will continue to evolve, and these companies will have many growth opportunities by 2021. This seems to be good news for today, but we can expect greater returns beyond leading players in the industry.

Market Segmentation:

Gaucher Disease Treatment Market Size and Forecast by Disease type, 2018 2025

Gaucher Disease Treatment Market Size and Forecast by Treatment type, 2018 2025

Overview of the Regional Outlook of this Market:

The Gaucher Disease Treatment report provides information about the market area, which is further subdivided into sub-regions and countries/regions. In addition to market share in each country and subregion, this chapter of this report also contains information about profit opportunities. This chapter of the report mentions the share and market growth rate of each region, country, and sub-region in the estimated time period.

Impact of COVID-19:

The report provides insights on COVID-19, which considers changes in consumer behavior and demand, purchasing patterns, rerouting of the supply chain, current dynamics of market forces, and important government interventions. Considering the impact of COVID-19 on the market, the updated research provides insights, analysis, estimates and forecasts.

Finally, the Gaucher Disease Treatment market report provides insights and expert analysis of key market trends and behaviors, as well as an overview of market data and major brands. The Gaucher Disease Treatment market report provides easy-to-digest all data information to promote future innovation and provide business support for every entrepreneur and established enterprise.

If You Have Any Query, Ask Our Experts @ https://www.polarismarketresearch.com/industry-analysis/gaucher-disease-treatment-market/speak-to-analyst

About Polaris market Research

Polaris market Research is a global market research and consulting company. We provide unmatched quality of offerings to our clients present globally. The company specializes in providing exceptional market intelligence and in-depth business research services for our clientele spread across industry verticals.

Contact Us

Corporate Sales, USA

Polaris market Research

Phone: 1-646-568-9980

Email:sales@polarismarketresearch.com

Web:www.polarismarketresearch.com

Visit link:
Gaucher Disease Treatment Market Study Report | Market Analysis, Drivers, Restraints, Opportunities, and Threats : Acetelion Pharmaceutical (J&J Ltd.)...

This year, vaping and other key respiratory issues join COVID-19 on the agenda for the ATS 2021 International Conference.

Vaping really fell under the radar. And I actually think it would be worth mentioning that it hasn't gone away, said Andrew J. Halayko, PhD, ATSF, FCAHS, professor of physiology and pathophysiology and internal medicine at the University of Manitoba, in Canada, and chair of the international conference.

The conference begins Friday, May 14, formally opening with a keynote address on Saturday from Anthony Fauci, MD, titled, COVID-19 in 2021: Lessons Learned and Remaining Challenges. Fauci is President Joe Biden's medical adviser and head of the National Institute of Allergy and Infectious Diseases.

In an interview with The American Journal of Managed Care, Halayko gave a broad overview of what attendees can expect to hear during the meeting.

Asthma management guidelines

This session, called Three in One: Updates on Asthma Management From 3 Guidelines Committees, was postponed from last year, Halayko said, to ensure that it received due attention. It will highlight the most recent recommendations from the US National Asthma Education and Prevention Program and the Global Initiative for Asthma, as well as guidelines from the European Respiratory Society and the ATS.

Biologics and biomarkers

A number of presentations and posters will focus on various aspects of biologics and biomarkers for severe asthma, which affects about 10% of patients.

What we're learning is that a really good antibody therapy for asthma doesn't fit all, and it's really important to endotype or phenotype the patient, Halayko said. Given the expense of newer therapies, its vital to know which subset of patients they will help the most, he added.

COPD

Every talk in the chronic obstructive pulmonary disease (COPD) section is starred as a hot topic, Halayko said, partly in recognition of the fact that it's time to move forward with a better understanding of disease pathology, but also therapies where we really have not made significant progress. And we are starting to get to the point where precision medicine and large data studies, omics studies, are coming forward.

Vaping and cannabis

There are more than 2 dozen posters on vaping and cannabis, the long-term pulmonary effects of which are not known. Previous studies have linked vaping to more severe effects from COVID-19. The 2019 e-cigarette or vaping-associated lung injury epidemic is continuing, and various presentations and posters will discuss the types of injuries seen, the chemicals involved, recovery trajectories, and more.

Racism and health equity

The president of ATS, Juan C. Celedn, MD, DrPH, division director, Pulmonology; the Niels K. Jerne Professor of Pediatrics; and professor of medicine, epidemiology, and human genetics, chairs a symposium asking if racial and ethnic disparities in respiratory health can be eliminated. Social determinants of health, universal health care, improving access to lung cancer screening, and other areas are up for discussion by the panel.

Read more here:
5 Things to Watch for at the ATS 2021 International Conference - AJMC.com Managed Markets Network

BETHESDA, Md., May 13, 2021 /PRNewswire/ --The National Coordinating Center for the Regional Genetics Network (NCC) is excited to announce the second annual Public Health Genetics Week from May 24-28, 2021. The goal of Public Health Genetics Week is to increase awareness and to celebrate the field of public health genetics.

Each day of Public Health Genetics Week will have a different theme:

Individuals and organizations are encouraged to participate in the week by using the hashtags #PHGW and #PublicHealthGenetics across their social media platforms.

The following events will also occur throughout the week:

Ken Burns Presents The Gene: An Intimate HistoryVirtual Screening

In collaboration with WETA Washington, D.C., virtual daily screenings of Ken Burns Presents The Gene: An Intimate History ("THE GENE") will be held to celebrate Public Health Genetics Week. The landmark four-hour documentary seriesweaves together science, history, and personal stories to present a historical biography of the human genome, while also exploring groundbreaking breakthroughs for diagnosis and treatment of genetic diseases, and the complex web of moral, ethical and scientific questions raised by developments in genetics.

Listed below is the virtual screening schedule. More information, including registration information, can be found at https://phgw.org/thegene.

For more information about the film, visit https://pbs.org/thegene.

Social Media Events

Other Activities

OnPHGW.org, everyone can find more information about the daily themes, social media events, and social media tools (such as daily social media images, GIFs, social media banners, and more).

For questions or comments about Public Health Genetics Week, please contact [emailprotected]and be sure to follow NCC (@nccrcg) onFacebook,Instagram,LinkedIn,TikTok, andTwitterfor the latest updates on the week.

About the National Coordinating Center for the Regional Genetics Networks (NCC)

Funded since 2004 by the Health Resources and Services Administration/Maternal and Child Health Bureau (MCHB) to the American College of Medical Genetics and Genomics (ACMG), NCC's mission is to improve access to genetic services for underserved populations. In collaboration with the seven Regional Genetics Network (RGNs) and the National Genetics Education and Family Support Center (NGEFSC), NCC achieves this mission by working in the following focus areas: genetics and genomics education; genetics policy education; telemedicine; and data collection and evaluation. Learn more about the efforts of the NCC athttps://nccrcg.org.

NCC Funding Acknowledgement

This project is supported by the Health Resources and Services Administration (HRSA) of the U.S. Department of Health and Human Services (HHS) under Cooperative Agreement #UH9MC30770-01-00 from June 2020 to May 2024 for $800,000 per award year.

This information or content and conclusions are those of the author and should not be construed as the official position or policy of, nor should any endorsements be inferred by HRSA, HHS or the U.S. government.

About Ken Burns Presents The Gene: An Intimate History

Ken Burns Presents The Gene: An Intimate Historyis a production of Florentine Films and WETA Washington, D.C., in association with Ark Media. Executive Producer and Senior Creative Consultant: Ken Burns. Written by Geoffrey C. Ward; and Barak Goodman & David Blistein. Based on the book "The Gene: An Intimate History" by Dr. Siddhartha Mukherjee. Narrator: David Costabile. Senior Producer: Barak Goodman. Directors: Chris Durrance and Jack Youngelson. Executive Producers: Dr. Siddhartha Mukherjee, Dalton Delan, Tom Chiodo, John F. Wilson and Anne Harrington. Production funding has been provided by Genentech, 23andMe, Cancer Treatment Centers of America, Alfred P. Sloan Foundation, Gray Foundation, American Society of Clinical Oncology (ASCO) & Conquer Cancer Foundation, Judy and Peter Blum Kovler Foundation, Craig and Susan McCaw Foundation, and the Corporation for Public Broadcasting. The Outreach and Education Partner is National Institutes of Health, National Human Genome Research Institute. Outreach support is provided by Foundation Medicine.

About WETA Washington, D.C.

WETA Washington, D.C., is a leading producer of new content for public television in the United States. WETA productions and co-productions include PBS NewsHour; Washington Week; documentaries by filmmaker Ken Burns, including Ken Burns Presents The Gene: An Intimate History, Cancer: The Emperor of all Maladies, Hemingway and the forthcoming MuhammadAli;and series and specials by scholar Henry Louis Gates, Jr., including Finding Your Roots, Reconstruction: America After the Civil War and The Black Church: This Is Our Story, This Is Our Song. WETA's multi-year campaign Well Beings addresses the critical health needs of Americans. More information on WETA and its programs and services is available at weta.org. On social media, visit facebook.com/wetatvfmon Facebook and follow @WETAtvfmon Twitter.

Related Images

image1.png

SOURCE National Coordinating Center for the Regional Genetics Networks (NCC)

Original post:
National Coordinating Center for the Regional Genetics Network (NCC) Announces the Second Annual Public Health Genetics Week, May 24-28, 2021 -...

Igor Golovniov/SOPA Images/LightRocket via Getty Images

Third Rock Ventures has closed a Series A financing round thatraised $82 million, funding which will be used to launchFlare Therapeutics, a new biotech startup focused on developing precision treatments for cancer and other diseases. Significant contributions were made to the Series A by Boxer Capital, Nextech Invest, Casdin Capital, Invus Financial Advisors, and Eventide Asset Management.

Flare uses a switch-site based drug discovery approach, which helped the company develop a pipeline of therapeutic programs that target pivotal cancer drivers such as transcription factor dysregulation and mutations. The new financing from the Series A will support its ability to advance its lead precision oncology program toward the clinic.

Flare currently has three lead precision oncology programs. The first program is expected to move into the clinic in 2023 or 2024, while the following two programs are expected to enter the clinic in the two years following.

Abbie Celniker, Ph.D., Flares interim Chief Executive Officer, and Third Rock Ventures partner, said in a statement that the new startup company was created to pursue the mission of conquering transcription factors which have been one of the most sought-after targets of drug developers based on the central role they play in cancer and other diseases.

Transcription factors appear to play a central role in cancer, among other diseases, according to emerging data and scientific discoveries published over the past ten years. Yet, transcription factors have continued to be elusive for finding targetable sites for drug discovery, with less than 1% of transcription factors successfully targeted for medicines, according to a statement made by Flares Scientific Co-Founder, Fraydoon Rastinejad, Ph.D., a professor of biochemistry and structural biology at the University of Oxford.

The novel drug discovery paradigm established by Flare uses an approach that targets transcription factors. According to the company, this strategy is based primarily on a broader understanding of the cooperative communication and allosteric interaction among the elements of the transcriptional molecular complex, in contrast to the previous scientific focus on only single, isolated transcription factor domains.

Recent work by the companys Scientific Co-Founders has helped Flare gain a greater understanding of the molecular mechanisms involved in targeting transcription factors. This understanding, according to Flare, has helped its research team to recognize the broad potential to generalize these principles to the switch site as a focal point for drugging transcription factors in a new way.

Scientific Co-Founder of Flare, Steven McKnight, Ph.D., a professor at the University of Texas, Southwestern, noted, Maturation of the fields of human genetics and the biochemistry of gene regulation now point us towards opportunities for therapeutic intervention using conventional, small molecule drugs.

Likely, Flare will also plan to use the new Series A funding to grow its staff to 25 employees. Currently, the company employs 12 to 15 full-time staffers, mainly relying on contractors and partners to fulfill its operational needs.

Follow this link:
Third Rock Ventures Launches Flare Therapeutics With $82 Million Series A - BioSpace

Research that we have part-funded has led to the discovery of 179 kidney genes responsible for high blood pressure.

Scientists who led the study, which has been published in Nature Genetics, say that this sheds new light on our understanding of genes associated with the condition.

High blood pressure is a key risk factor for heart and circulatory diseases, such as heart attack and stroke, and can run in families. However, the exact ways in which genes can cause people to inherit the condition is not clear.

The study found how certain information inherited in DNA can translate into an increased likelihood of high blood pressure, through changes in the activity of certain kidney genes.

The research, led by Professor Maciej Tomaszewski at The University of Manchester, was possible through access to huge datasets of human DNA and biological material known as RNA from possibly the worlds largest source of human kidney tissue samples.

Around 80 per cent of the 179 genes discovered by the team have never been associated with high blood pressure before. Some of these genes can be targeted by existing medicines, creating new opportunities to treat the condition.

Professor James Leiper, our Associate Medical Director, said: We have known for many years that the kidney is a major regulator of blood pressure, but our understanding of precisely how the kidney controls blood pressure is incomplete.

The identification of this large set of genes that appear to directly affect blood pressure fills in an important missing piece of that puzzle. The researchers have also found a subset of these genes that are a potential new target for the treatment of hypertension.

This is important because many people taking existing medications still struggle to control their blood pressure. If doctors have more tools to work with then it will help stop thousands of lives being lost each year from this potentially preventable condition.

The study was also supported by Kidney Research UK.

OUR RESEARCH INTO HIGH BLOOD PRESSURE

Read more:
Genes responsible for high blood pressure discovered in the human kidney - British Heart Foundation

PITTSBURGH, May 12, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, announced today the appointment of Gerald (Gerry) J. McDougall to the Company's Board of Directors. For more than 25 years, Mr. McDougall has been the driving force behind large-scale strategic alliances, joint ventures, and industry partnerships across the healthcare industry to advance innovations in precision medicine and cancer.

"Gerry's deep expertise, passionate commitment to improve the human condition, and vast network have been the foundation for numerous transformational alliances in life sciences and healthcare, and his ability to create synergistic combinations of people, ideas, and resources is exceptional," said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. "The Board and I look forward to working closely with Gerry as we advance NeuBase's comprehensive approach to precision genetic medicine to address previously untreatable diseases."

"Genetics are the foundation for understanding and treating rare and common diseases including cancers, and the ability to precisely modulate gene function is key to developing new medicines for the many diseases that still have no treatment options," said Mr. McDougall. "I have dedicated my career to coalescing divergent approaches to achieve precision care, and I believe NeuBase can unify the field of precision genetic medicine with its PATrOL technology platform."

Mr. McDougall spent almost his entire career as a senior partner at PwC where he built and led the firm's Global Health Science consulting practice before retiring. He has worked across the entire ecosystem of the healthcare industry and advised an array of Fortune 500 companies, including leading global pharmaceutical companies. Mr. McDougall has been instrumental around the globe in building public-private partnerships to address human health imperatives. These include the creation and maturation of the Translational Genomics Research Institute (TGen), Arizona's renowned bio-cluster; the design and launch of the Multiple Myeloma Research Consortium (MMRC); the strategic plan for the California Institute of Regenerative Medicine (CIRM) and the Country of Luxemburg's biotechnology commercialization ecosystem.

About NeuBase Therapeutics, Inc.NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines which can be designed to increase, decrease, or change gene function, as appropriate, to resolve genetic defects that drive disease. NeuBase's targeted PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. Forward-looking statements include, among others, those related to the anticipated strategic guidance and assistance that the Company's new director will provide to support the Company's comprehensive approach to precision genetic medicine to address previously untreatable diseases. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission (the SEC), may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the timing of the availability of data from the Company's clinical trials; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.comOP: (617) 430-7576

NeuBase Media Contact:Jessica Yingling, Ph.D.Little Dog Communications Inc.jessica@litldog.com+1 (858) 344-8091

Continue reading here:
NeuBase Therapeutics Appoints Gerald J. McDougall to Board of Directors - BioSpace

Visit the News Hub

Study to look for genetic pathways that lead to formation of plaques, tangles

A $10.7 million, five-year grant will support a comprehensive study in which whole genome sequencing will be used to address critical gaps in knowledge about Alzheimer's disease. The project is led by researchers at Washington University School of Medicine in St. Louis and the University of Pittsburgh Graduate School of Public Health.

Despite decades of research and investment, the genetic underpinnings of Alzheimers disease are still largely unknown, stymieing efforts at drug development and early diagnosis. To change that, a new grant will support the first comprehensive study to use whole genome sequencing to address critical gaps in knowledge about the disease. The $10.7 million, five-year project is led by researchers at Washington University School of Medicine in St. Louis and the University of Pittsburgh Graduate School of Public Health.

Funded by the National Institute on Aging of the National Institutes of Health (NIH), the research team plans to identify the genetic variants, genes and pathways that lead to formation of plaques and tangles, two specific signs of disease called biomarkers that begin appearing in the brains of people with Alzheimers 15 to 25 years before they show symptoms.

Cruchaga

Genetic studies of measurable traits such as plaques and tangles provide advantages over other classic case-control studies, because these traits appear earlier and are more closely related to the biology behind the disease, said Carlos Cruchaga, PhD, a co-principal investigator of the study and the Reuben Morriss III Professor of Neurology at Washington University School of Medicine. In addition, studying these traits is more likely to lead to the identification of druggable targets along the genetic pathways that lead to disease. This genetic information can help us better predict disease risk at the individual patient level.

Cruchaga, also a professor of psychiatry, is working with co-principal investigator Ilyas Kamboh, PhD, a professor of human genetics and epidemiology at Pitt Public Health. Together, they plan to study as many as 5,000 participants at high risk for Alzheimers. The researchers will gather biomarker data to identify genetic variants that appear decades before clinical symptoms of the disease.

All of the clinical trials to find a drug to stop Alzheimers disease have failed because theyve focused on patients who already have developed the disease, so they already had high levels of plaques and tangles, said Kamboh. Once you have the plaques and tangles, it seems to be an irreversible process, so were focused on the preclinical stage of the disease.

According to the World Health Organization, Alzheimers disease is the most common form of dementia, with about 50 million cases worldwide and 6 million new cases each year. It is one of the major causes of disability and dependency among older people.

The plaques and tangles in the brain associated with Alzheimers can be thought of like cholesterol in the arteries of the heart and its association with heart disease, Kamboh explained. Cholesterol can quietly accumulate over years along the walls of the coronary arteries without causing symptoms until it causes a heart attack and does irreversible damage to the heart. Some genes predispose people to accumulate more cholesterol, and understanding that can allow people to take medication and make lifestyle changes that reduce the risk of heart disease. It also can prompt pharmaceutical companies to develop drugs that target the genetic pathways that lead to the formation of cholesterol deposits.

The new project will look for the genetic underpinnings of the plaques and tangles known to define Alzheimers disease and that formed due to abnormal accumulation of amyloid beta and tau proteins, respectively. Both can be detected early in the brains of living people through neuroimaging and the testing of cerebrospinal fluid.

In the past, we could detect these plaques and tangles only after death, through a brain autopsy, Kamboh said. Now we can identify them while people are living.

But those imaging and fluid-collection techniques are expensive and can be invasive.

New methods are now being developed to detect the presence of abnormal amyloid beta and tau proteins in less expensive blood tests, Cruchaga said. We hope that by learning more about the genes associated with the plaques and tangles, we might uncover underlying pathways that lead to Alzheimers disease and discover potential drug targets.

This study is supported by the National Institute on Aging of the National Institutes of Health (NIH). Grant number R01 AG064877.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, consistently ranking among the top medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

See the original post:
WashU, Pitt awarded $10.7 million for Alzheimer's disease research Washington University School of Medicine in St. Louis - Washington University...