StemCell Therapy

Los Angeles, United State: QY Research recently published a research report titled, Global Stem Cell Cryopreservation Equipment Market Research Report 2020 The research report is collated on the basis of historic and forecast data derived by using primary and secondary methodologies by researchers. The global Stem Cell Cryopreservation Equipment market is one of the fastest-growing markets and is expected to witness substantial growth in the forecast years. Reader are provided easy access to thorough analysis on the various aspects such as opportunities and restraints affecting the market. The Stem Cell Cryopreservation Equipment report clearly explains the trajectory this market will take in the forecast years.

The Stem Cell Cryopreservation Equipment research report has presented an analysis of various factors influencing the markets current growth. Drivers, restraints, and trends are elaborated to understand their positive or negative effects. This Stem Cell Cryopreservation Equipment report section is aimed at providing readers with a thorough information about the potential scope of various applications and segments. These estimates are based on the current trends and historic milestones.

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This section of the Stem Cell Cryopreservation Equipment report offers a thorough and comprehensive information about the various manufacturers in the market. The major manufacturers covered in the report hold significant share that demands a microscopic look. It provides vital information about various strategies implemented by these manufacturers to combat competition and expand their footprint in the Stem Cell Cryopreservation Equipment market. It also surveys the current trends adopted by the manufacturers to innovate their product for the future. This Stem Cell Cryopreservation Equipment report is structured in such a way so as to help the reader understand the market and make business decisions accordingly.

Key Players Mentioned in the Global Stem Cell Cryopreservation Equipment Market Research Report:

Chart, Worthington Industries, Cesca Therapeutics, Shengjie Cryogenic Equipment, Sichuan Mountain Vertical, Qingdao Beol

Global Stem Cell Cryopreservation Equipment Market Segmentation by Product:Liquid PhaseVapor Phase

Global Stem Cell Cryopreservation Equipment Market Segmentation by Application:Cord Blood Stem Cell CryopreservationOther Stem Cell Cryopreservation

The Stem Cell Cryopreservation Equipment Market research report briefs on segments such as product type and end users. The product type segment gives an understanding about various products available in the Stem Cell Cryopreservation Equipment market. It also gives information on what is the scope and potential of each product. Also, the segment presents an elaborate information on end users. Understanding end users is of utmost importance as it aids in identifying marketable areas.

Key questions answered in the report: What is the growth potential of the Stem Cell Cryopreservation Equipment market? Which product segment will grab a lions share? Which regional market will emerge as a frontrunner in coming years? Which application segment will grow at a robust rate? What are the growth opportunities that may emerge in Stem Cell Cryopreservation Equipment industry in the years to come? What are the key challenges that the global Stem Cell Cryopreservation Equipment market may face in future? Which are the leading companies in the global Stem Cell Cryopreservation Equipment market? Which are the key trends positively impacting the market growth? Which are the growth strategies considered by the players to sustain hold in the global Stem Cell Cryopreservation Equipment market?

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Table od Content:1.1 Research Scope1.2 Market Segmentation1.3 Research Objectives1.4 Research Methodology1.4.1 Research Process1.4.2 Data Triangulation1.4.3 Research Approach1.4.4 Base Year1.5 Coronavirus Disease 2019 (Covid-19) Impact Will Have a Severe Impact on Global Growth1.5.1 Covid-19 Impact: Global GDP Growth, 2019, 2020 and 2021 Projections1.5.2 Covid-19 Impact: Commodity Prices Indices1.5.3 Covid-19 Impact: Global Major Government Policy1.6 The Covid-19 Impact on Stem Cell Cryopreservation Equipment Industry1.7 COVID-19 Impact: Stem Cell Cryopreservation Equipment Market Trends

2 Global Stem Cell Cryopreservation Equipment Quarterly Market Size Analysis2.1 Stem Cell Cryopreservation Equipment Business Impact Assessment COVID-192.1.1 Global Stem Cell Cryopreservation Equipment Market Size, Pre-COVID-19 and Post- COVID-19 Comparison, 2015-20262.1.2 Global Stem Cell Cryopreservation Equipment Price, Pre-COVID-19 and Post- COVID-19 Comparison, 2015-20262.2 Global Stem Cell Cryopreservation Equipment Quarterly Market Size 2020-20212.3 COVID-19-Driven Market Dynamics and Factor Analysis2.3.1 Drivers2.3.2 Restraints2.3.3 Opportunities2.3.4 Challenges

3 Quarterly Competitive Assessment, 20203.1 Global Stem Cell Cryopreservation Equipment Quarterly Market Size by Manufacturers, 2019 VS 20203.2 Global Stem Cell Cryopreservation Equipment Factory Price by Manufacturers3.3 Location of Key Manufacturers Stem Cell Cryopreservation Equipment Manufacturing Factories and Area Served3.4 Date of Key Manufacturers Enter into Stem Cell Cryopreservation Equipment Market3.5 Key Manufacturers Stem Cell Cryopreservation Equipment Product Offered3.6 Mergers & Acquisitions, Expansion Plans

4 Impact of Covid-19 on Stem Cell Cryopreservation Equipment Segments, By Type4.1 Introduction1.4.1 Liquid Phase1.4.2 Vapor Phase4.2 By Type, Global Stem Cell Cryopreservation Equipment Market Size, 2019-20214.2.1 By Type, Global Stem Cell Cryopreservation Equipment Market Size by Type, 2020-20214.2.2 By Type, Global Stem Cell Cryopreservation Equipment Price, 2020-2021

5 Impact of Covid-19 on Stem Cell Cryopreservation Equipment Segments, By Application5.1 Overview5.5.1 Cord Blood Stem Cell Cryopreservation5.5.2 Other Stem Cell Cryopreservation5.2 By Application, Global Stem Cell Cryopreservation Equipment Market Size, 2019-20215.2.1 By Application, Global Stem Cell Cryopreservation Equipment Market Size by Application, 2019-20215.2.2 By Application, Global Stem Cell Cryopreservation Equipment Price, 2020-2021

6 Geographic Analysis6.1 Introduction6.2 North America6.2.1 Macroeconomic Indicators of US6.2.2 US6.2.3 Canada6.3 Europe6.3.1 Macroeconomic Indicators of Europe6.3.2 Germany6.3.3 France6.3.4 UK6.3.5 Italy6.4 Asia-Pacific6.4.1 Macroeconomic Indicators of Asia-Pacific6.4.2 China6.4.3 Japan6.4.4 South Korea6.4.5 India6.4.6 ASEAN6.5 Rest of World6.5.1 Latin America6.5.2 Middle East and Africa

7 Company Profiles7.1 Chart7.1.1 Chart Business Overview7.1.2 Chart Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.1.3 Chart Stem Cell Cryopreservation Equipment Product Introduction7.1.4 Chart Response to COVID-19 and Related Developments7.2 Worthington Industries7.2.1 Worthington Industries Business Overview7.2.2 Worthington Industries Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.2.3 Worthington Industries Stem Cell Cryopreservation Equipment Product Introduction7.2.4 Worthington Industries Response to COVID-19 and Related Developments7.3 Cesca Therapeutics7.3.1 Cesca Therapeutics Business Overview7.3.2 Cesca Therapeutics Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.3.3 Cesca Therapeutics Stem Cell Cryopreservation Equipment Product Introduction7.3.4 Cesca Therapeutics Response to COVID-19 and Related Developments7.4 Shengjie Cryogenic Equipment7.4.1 Shengjie Cryogenic Equipment Business Overview7.4.2 Shengjie Cryogenic Equipment Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.4.3 Shengjie Cryogenic Equipment Stem Cell Cryopreservation Equipment Product Introduction7.4.4 Shengjie Cryogenic Equipment Response to COVID-19 and Related Developments7.5 Sichuan Mountain Vertical7.5.1 Sichuan Mountain Vertical Business Overview7.5.2 Sichuan Mountain Vertical Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.5.3 Sichuan Mountain Vertical Stem Cell Cryopreservation Equipment Product Introduction7.5.4 Sichuan Mountain Vertical Response to COVID-19 and Related Developments7.6 Qingdao Beol7.6.1 Qingdao Beol Business Overview7.6.2 Qingdao Beol Stem Cell Cryopreservation Equipment Quarterly Production and Revenue, 20207.6.3 Qingdao Beol Stem Cell Cryopreservation Equipment Product Introduction7.6.4 Qingdao Beol Response to COVID-19 and Related Developments

8 Supply Chain and Sales Channels Analysis8.1 Stem Cell Cryopreservation Equipment Supply Chain Analysis8.1.1 Stem Cell Cryopreservation Equipment Supply Chain Analysis8.1.2 Covid-19 Impact on Stem Cell Cryopreservation Equipment Supply Chain8.2 Distribution Channels Analysis8.2.1 Stem Cell Cryopreservation Equipment Distribution Channels8.2.2 Covid-19 Impact on Stem Cell Cryopreservation Equipment Distribution Channels8.2.3 Stem Cell Cryopreservation Equipment Distributors8.3 Stem Cell Cryopreservation Equipment Customers

9 Key Findings

10 Appendix10.1 About Us10.2 Disclaimer

About Us:QY Research established in 2007, focus on custom research, management consulting, IPO consulting, industry chain research, data base and seminar services. The company owned a large basic data base (such as National Bureau of statistics database, Customs import and export database, Industry Association Database etc), experts resources (included energy automotive chemical medical ICT consumer goods etc.

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Stem Cell Cryopreservation Equipment Market Size, Growth Trends, Top Players, Application Potential and Forecast to 2026| Chart, Worthington...

SYDNEY, N.S.

Its National Blood Donor Week in Canada, but some Cape Breton Regional Municipality residents are feeling left out.

I was so surprised when the clinic closed here several years ago, said Maggie Lynne MacNeil of Sydney, who made it a priority to donate blood when the service was here. It was a motor vehicle accident involving her husband seven years agothat opened her eyes to the importance of blood donation.

MacNeil said shes grateful Canadian Blood Collection Services provides this important service and has contacted them to find out if a clinic might reopen in the municipality.

Monetary donations are important everywhere and can do a lot of good, she said. Blood can save lives. We lost it, but I think if it was brought back people would support it more than ever. There's no way we can donate blood here. That doesn't even make sense."

Rene Horton, spokesperson for Canadian Blood Services, said they do receive questions, comments and requests to return (the service) to Sydney.

We receive approximately five or so per year, she said.

However, a return to Sydney is not on the horizon, said Peter MacDonald, director of donor relations for the Atlantic region.

MacDonald said as of now, there are enough events in their clinic plan to meet hospital demand.

Id never say never, but theres no immediate plans to reopen the site, he said.

The blood collection clinic which was located on Grand Lake Road in Sydney closed in May 2015.

MacDonald said the decision to close was due to a decline in hospital demand for blood and blood products in the area.

Donations wasnt the issue, he said. We had certainly dedicated donors in Sydney and all the CBRM, too, he said.

There is currently no place in the Cape Breton Regional Municipality to donate blood. The nearest place to make a donation is the mobile clinic that visits Port Hawkesbury four or five times a year. Those two-day visits have a 173-unit target and consistently meets and exceeds the target.

This is National Blood Donor Week and Sunday will mark National Blood Donor Day.

The goal for Nova Scotia is 667 donations and as of Thursday, the Canadian Blood Collection Services needed 204 blood donations for the rest of June to meet the target. Those appointments were already booked to meet that goal.

Less than four per cent of the population of Canada are active donors. There are more than 20,000 active blood donors in Nova Scotia.

One donation of blood could potentially save three lives, as three components are drawn from each:plasma which can be frozen and last for up to a year, red cells which have a 42-day shelf life and platelets which only last for seven days.

There is also always a need for O negative blood, the universal blood type.

MacDonald said there are other ways Cape Bretoners can help including by joining the stem cell registry online at http://www.blood.ca. Participants are sent a kit, swab the inside of their cheek and then return the swab. The results are placed on the stem cell registry.

The need to grow the registry is there, particularly with males 17-35. As well, the registry is in need of ethnic diversity. Currently, the makeup of the registry doesnt match the population in Canada as the patient population continues to become more diverse.

MacDonald said they are grateful to those in Cape Breton who have given blood and continue to do so at mobile clinics.

I would also assure readers of the Cape Breton Post that the fact that we dont collect there doesnt mean that hospitals there dont receive all the help needed, he said.

Theres no risk that hospitals in Cape Breton dont receive blood and blood products when they need it.

Meanwhile, MacDonald said the COVID-19 crisis has impacted the blood service in a few ways. For example, since mid-March, some mobile clinics have been shut down due to the pandemic.

Even with the loss of capacity, we were still able to meet hospital demand, he said, adding demand decreased due to the pandemic as well because elective surgeries were cancelled, there were fewer traumas as less people were moving around and on highways, for example.

As the province begins to reopen and elective surgeries are rescheduled, hospital demand will begin to increase.

Its starting to get back to pre-COVID levels, he said. Fortunately some of those sites not available for mobiles are now reopening.

The full schedule for mobile units can be viewed at http://www.blood.ca and people can download the donate blood app.

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It's National Blood Donor Week, but nowhere to donate in Cape Breton Regional Municipality - The Telegram

LOUISVILLE, Ky. The American Heart Association is investing nearly $17 million to fund a two-year research initiative, ENACT (End Nicotine Addiction in Children and Teens), led by a national network of scientists focusing on the health impacts of e-cigarettes and other nicotine delivery systems on youth and young adults.

As part of this network, researchers at the University of Louisville Christina Lee Brown Envirome Institute will conduct studies to better understand youth vaping. Institute researcher Joy Hart, professor in the UofL Department of Communication, will lead a team engaging local youth to learn about what drives youth to use e-cigarettes, the health effects of this use and how to motivate young people to stop using these products.

This investment is the latest in a multipronged, ongoing commitment announced last fall by the American Heart Association to fight the growing epidemic of youth vaping.

E-cigarettes are being marketed as a healthy option to traditional cigarettes, but no one knows if vaping is safe in the long run because e-cigarettes havent been around long enough to be studied deeply. Some diseases can take years and even decades to develop, so there is more work needed to fully understand all the dangers, said Dr. Robert A. Harrington, AHA president, Arthur L. Bloomfield professor of medicine and chair of the department of medicine at Stanford University. Theres certainly plenty of indication theyre harmful for growing minds and bodies because we know e-cigarettes contain nicotine and we know the harmful effects of nicotine, but its important we grow that overall body of scientific evidence.

Harrington said theres a sense of urgency because, at a time when regular cigarette smoking has reached an all-time low, young people are turning to e-cigarettes at epidemic proportions with nearly one in four high school students reportedly vaping. Thats why these research projects will be high-impact and fast tracked, only two years in length and funded at levels among the highest individual grants awarded in the associations history. The initiative is designed to produce turnkey programs to support youth as well as provide clear evidence to inform policy decisions.

The UofL research project is part of the Rapidly Advancing Discovery to Arrest the Outbreak of Youth Vaping Center (VAPERACE) at Boston University. Hart will spearhead community outreach activities and will work with other center investigators to understand the health impact of vaping on youth and to develop new approaches to vaping cessation. The research team includes Kandi Walker, Rachel Keith and Aruni Bhatnagar, director of the Envirome Institute.

Tobacco use causes health issues for many Kentuckians, and is particularly concerning in young people. We are pleased that researchers in the UofL Christina Lee Brown Envirome Institute will contribute to this significant effort by the American Heart Association and hopefully reduce the negative health effects of tobacco use on the next generation, said Neeli Bendapudi, president of the University of Louisville.

Studying vaping among youth has always been important. It became even more important with widespread lung illness among young people, Bhatnagar said. Now, in context of the COVID-19 pandemic, it appears that those who use nicotine may be at greater risk of serious COVID-19 illness.

In addition to UofL, VAPERACE at Boston University includes projects by Johns Hopkins University and Stanford University. The projects supported by the center include: basic research using human-induced pluripotent stem cell samples to test e-cigarette component toxicity, mobile health technology to measure the physiological cardiovascular impacts of e-cigarettes on youth in real-world settings and a virtual reality and text messaging-delivered e-cigarette cessation program for youth developed by combining social media methods with focus groups.

Two additional projects funded by the AHA as part of the network are based at Ohio State University and Yale University. Overall, network researchers will work to identify the biological impacts of vaping on multiple organ systems (heart, brain, lungs, vascular system, etc.), behavioral factors and specific social influencers of health to reverse these trends.

The rapid pace of e-cigarette products entering the market and targeting our youth requires an ambitious, aggressive approach beyond the incremental pace achieved through traditional research mechanisms. Policymakers, regulators, medical professionals and schools are looking to enact strategies, policies and solutions but theres inadequate evidence to inform these efforts, Harrington said. The American Heart Association is proud to be on the forefront of bringing together some of the best minds in their fields to conduct the research, development and testing to bring bold and innovative results to address the growing epidemic of youth vaping in our commitment of longer, healthier lives for all.

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UofL researchers to study health impacts of vaping, nicotine on youth - The Lane Report

Precision medicine (PM) is an approach to patient care that allows doctors to select treatments that are most likely to help patients based on a genetic understanding of their disease. Personalized nanomedicine involving individualized drug selection and dosage profiling in combination with clinical and molecular biomarkers can ensure the maximal efficacy and safety of the treatment. The major hindrance toward the development of such therapies is the handling of the Big Data, to keep the databases updated. Robust automated data mining tools are being developed to extract information regarding genes, variations, and their association with diseases. Phenotyping, an integral part of PM, is aimed at translating the data generated at cellular and molecular levels into clinically relevant information.Precision Medicine Moves Care from Population-Based Protocols to Truly Individualized Medicine as President of the US announced the Precision Medicine Initiative in his 2015 State of the Union address. Under the initiative, medical care would transition from a one-size-fits-all approach to an individualized approach, in which data on each patients genomic makeup, environment, and lifestyle (the exposome) helps medical professionals tailor treatment and prevention strategies. To achieve the Precision Medicine Initiative mission statement, to enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized care, researchers and clinicians need vast and varied amounts of data and the technology to ensure that data is widely accessible and usable.

Browse Complete Report with TOC https://univdatos.com/report/precision-medicine-market-current-analysis-and-forecast-2020-2026

Insights Presented in the Report

Based on technology type, the market is fragmented into big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics, and others.Recent technological and analytical advances in genomics, have now made it possible to rapidly identify and interpret the genetic variation underlying a single patients disease, thereby providing a window into patient-specific mechanisms that cause or contribute to disease, which could ultimately enable the precise targeting of these mechanisms

Based on the market segment by application type, the market is segmented into oncology, respiratory diseases, central nervous system disorders, immunology, genetic diseases and others. With the advent of precision medicine, cancer treatment is moving from a paradigm in which treatment decision isprimarily based on tumor location and histology followed by molecular information to a new paradigm whereby treatment decisions will be primarily based on molecular information followed by histology and tumor location

Based on the market segment by end-user, the market is fragmented into hospitals & clinics, pharmaceuticals, diagnostic companies, Healthcare-IT firms and others. The precision medicine suppliers that understand technology and the goals of value-based healthcare can create value in the precision medicine value-chain by offering value-based solutions and platforms to interpret and connect data points. There are a number of technology companies who work in the field of precision medicine and more will be founded in the years to come

For better understanding on the market dynamics of Precision Medicine market, detailed analysis was conducted for different countries in the region including North America (United States, Canada, Mexico and Rest of North America), Europe (Germany, UK, France, Italy, Spain and Rest of Europe), Asia-Pacific (China, Japan, Australia, India and Rest of APAC), and Rest of World

Some of the major players operating in the market includeHoffmann-La Roche, Medtronic, Qiagen, Illumina, Abbott Laboratories, GE Healthcare, NanoString Technologies, bioMrieux SA, Danaher Corporation, and AstraZeneca

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The report covers in-depth analysis of prominent industry peers with a primary focus on key business financials, product portfolio, expansion strategies, and recent developments

Detailed examination of drivers, restraints, key trends and opportunities prevailing in the industry.

Examination of industry attractiveness with the help of Porters Five Forces analysis

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Precision Medicine Market Size, Share & Trends Analysis Report by Product Types, And Applications Forecast To 2026 - Cole of Duty

London: Genes may soon reveal who would develop severe COVID-19 symptoms, remain asymptomatic or only have a mild coronavirus attack, say researchers.

Data from popular home genetic-testing kits could help scientists shed light on why some people who catch coronavirus have no symptoms while others become very ill, according to the team from the University of Edinburgh in the UK.

Researchers are now asking people who have used DNA testing services to gain ancestry or health insights to join a study that aims to identify key genes involved in the body's response to the infection.

"Understanding the effect genes have on susceptibility to COVID-19 could aid efforts to tackle the pandemic, and help combat future disease outbreaks," said the researchers.

More than 30 million people worldwide have used genetic testing services. Researchers are now urging them to share their DNA data to help speed up discoveries that could help fight the virus.

"Some people suffer no ill effects from coronavirus infection, yet others require intensive care. We need to identify the genes causing this susceptibility, so we can understand the biology of the virus and hence develop better drugs to fight it," said Jim Wilson, Professor of Human Genetics at the University of Edinburgh.

By providing gene data, volunteers will help the team avoid the costly, time-consuming task of collecting the hundreds of thousands of DNA samples that would otherwise be needed to map the genes involved.

The team aims to identify genes that influence the risk of developing COVID-19 and those that affect disease severity, by comparing volunteers' symptoms -- or lack of them -- with their DNA.

Researchers also aim to analyse the long-term health consequences of infection and self-isolation.

The study is supported by the Medical Research Council, Biotechnology and Biological Science Research Council, Health Data Research UK and Wellcome Trust.

"To identify the genes that explain why some people get very sick from coronavirus and others don't, we need the solidarity of a large proportion of people from different countries who can share their DNA testing results with us. In this case, size really matters," said Albert Tenesa, Professor of Quantitative Genetics at the University of Edinburgh.

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Researchers to identify genes that put some at severe corona risk - ETHealthworld.com

A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

The study, published today inNature Communications, titled, Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots, reveals how the genomic instability induced by the protein APOBEC3A offers a previously unknown vulnerability in cancer cells.

Each day, in human cells, tens of thousands of DNA damage events occur. In cancer cells, the expression of the protein APOBEC3A is one of the most common sources of DNA damage and mutations. While the mutations caused by these particular proteins in cancer cells contribute to tumor evolution, they also cause breaks in the DNA, which offer a vulnerability.

Targeting cancer cells with high levels of APOBEC3A protein activities and disrupting, at the same time, the DNA damage response necessary to repair damages caused by APOBEC3A, could be key to more effective cancer therapies, saidRemi Buisson, PhD, senior investigator and an assistant professor in the Department of Biological Chemistry at the UCI School of Medicine. However, to exploit the vulnerability of the cancer cells, it is critical to first quantitatively measure the proteins activity in tumors.

To understand the role of APOBEC3A in tumor evolution and to target the APOBEC3A -induced vulnerabilities, the researchers developed an assay to measure the RNA-editing activity of APOBEC3A in cancer cells. Because APOBEC3A is difficult to quantify in tumors, developing a highly sensitive assay for measuring activity was critical. Using hotspot RNA mutations, identified from APOBEC3A-positive tumors, the team developed an assay using droplet digital PCR and demonstrated its applicability to clinical samples from cancer patients.

Our study presents a new strategy to follow the dysregulation of APOBEC3A in tumors, providing opportunities to investigate the role of APOBEC3A in tumor evolution and to target the APOBEC3A-induced vulnerability in therapy, said Buisson. We anticipate that the RNA mutation-based APOBEC3A assay will significantly advance our understanding of the function of the protein in tumorigenesis and allow us to more effectively exploit the vulnerabilities it creates in cancer therapy.

This study was funded in part by the National Institutes of Health, a California Breast Cancer Research Program grant and an MPN Research Foundation Challenge grant.

Reference: Jalili, P., Bowen, D., Langenbucher, A. et al. (2020) Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots. Nat Commun. DOI: https://doi.org/10.1038/s41467-020-16802-8

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Exploiting Cancer Cell Vulnerabilities To Create More Effective Cancer Therapies - Technology Networks

Parasite infections are a constant presence for many people who live in tropical regions, particularly in less industrialized areas. These often chronic conditions are at best unpleasant; more seriously, children with parasite diseases that cause diarrhea can die of malnutrition or dehydration.

In Genome Biology, a study led by University of Pennsylvania scientists investigated the links between parasite infection and the gut microbiome. Using genetic methods to characterize the gastrointestinal microbiome of 575 ethnically diverse Cameroonian people representing populations from nine villages with meaningful differences in lifestyle, the researchers discovered that the presence of parasites was strongly associated with the overall composition of the microbiome.

"We found that we could look at someone's microbiome and use it to predict whether someone had a gastrointestinal parasite infection," says Meagan Rubel, who completed her doctorate degree at Penn and is now a postdoc at the University of California, San Diego. "Whether or not it was parasites changing the microbiome or something in the resident microbiota of a person that made them more susceptible to infection, we can't say, but the association was strong."

Rubel led the study in collaboration with Penn's Sarah Tishkoff, a Penn Integrates Knowledge Professor in the Perelman School of Medicine and School of Arts and Sciences, and Frederic Bushman, a microbiologist in the medical school. In addition to the microbiome and parasites, the research also examined markers of immune function, dairy digestion, and pathogen infection, a rich dataset.

The investigation entailed six months of field work, collecting fecal and blood samples from Mbororo Fulani pastoralists, cattle herders with a diet high in meat and dairy; Baka and Bagyeli rainforest hunter-gatherers, who practice a limited amount of farming but also forage for meat and plant-based foods; and Bantu-speaking agropastoralists, who both grow crops and raise livestock. As a comparison group, the study included data from two groups of people living in urban areas of the United States, with a diet heavier in animal fats, proteins, and processed foods.

In the field, the researchers tested for malaria and a number of other pathogens that infect both the blood and gastrointestinal system.

Of the 575 people tested in Cameroon, the researchers found nearly 40% were infected with more than one parasite before receiving an antiparasitic treatment, with hunter-gatherers, on average, most likely to be co-infected with multiple parasites. In particular, the team found that four soil-transmitted gut parasites tended to co-occur at a rate much higher than chance: Ascaris lumbricoides, Necator americanus, Trichuris trichiura, and Strongyloides stercoralis, or ANTS.

"Gut parasites are a global public health concern," says Rubel. "And you tend to see several of these parasites together in resource-poor settings where people may not have access to clinical care, piped water, and soap, so there's more opportunity for them to be transmitted."

Back in the lab at Penn, the researchers used genomic sequencing tools to take a snapshot of the participants' gut microbiomes. The composition of the microbiome, they found, could accurately predict a person's country (U.S. or Cameroon) and lifestyle (urban, pastoralist, agropastoralist, or hunter-gatherer). But after these two variables, the presence of ANTS parasites could be predicted with greater accuracy by the microbiome structure than any other variable the research team studied. Taken together, the microbiome could predict the presence of these four gut parasites with roughly 80% accuracy.

Infection with these parasites also led to upticks in immune system activation, specifically turning on pathways that promote inflammatory responses. Parasite infection was also associated with a greater likelihood of having bacteria from the order Bacteroidales, which are known to play a role in influencing digestion and immune system function.

In a second part of the study, the Penn-led team assessed the relationship between the gut microbiome and milk consumption in the Fulani pastoralist population. Earlier work by Tishkoff and colleagues illuminated how genetic mutations enabling lactose digestion arose in pastoralist communities in Africa, selected through evolution because of the important nutritional benefits of consuming dairy. In looking at the Fulani's microbiomes, they also tended to have an abundance of bacterial genes capable of breaking down galactose, a component of lactose, and fats, compared to other groups. "This enrichment of genes could help you extract more nutrition from the food you eat," Rubel says.

The researchers believe their findings, the largest-ever study on the link between gut microbiome composition and parasite infection from sub-Saharan Africa, can open new possibilities for future work. "The kinds of microbiome markers we found could be useful to predict the type of pathogens you have, or to shed light on the interplay between the microbiome and the immune system," says Rubel.

Eventually, she adds, more research could even illuminate strategies for purposefully modulating the microbiome to reduce the risk of a parasite infection or minimize the harm it causes to the body.

ReferenceRubel, M.A., Abbas, A., Taylor, L.J. et al. Lifestyle and the presence of helminths is associated with gut microbiome composition in Cameroonians. Genome Biol 21, 122 (2020). https://doi.org/10.1186/s13059-020-02020-4.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Investigating the Link between Parasites and the Gut Microbiome - Technology Networks

DALLAS June 14, 2020 Recently developed MRI techniques used to more precisely target a small area in the brain linked to Parkinsons disease and essential tremor may lead to better outcomes without surgery and with less risk of negative effects, a new study led by UT Southwestern researchers suggests.

The study, published today in Brain, describes recently refined MRI methods designed to allow neuroradiologists to zero in on a pea-sized region in the brains thalamus involved in movement. Using the images, doctors then can use high-intensity focused ultrasound (HIFU) to ablate, or burn away, problem tissue, says Bhavya R. Shah, M.D., first author of the study and an assistant professor of radiology and neurological surgery at UT Southwesterns Peter ODonnell Jr. Brain Institute.

The benefit for patients is that we will be better able to target the brain structures that we want, Shah says. And because were not hitting the wrong target, well have fewer adverse effects. The procedures are already Food and Drug Administration-approved for use in patients, and UTSW plans to begin employing them to treat patients when its Neuro High Intensity Focused Ultrasound Program opens this fall.

Adverse effects from imprecise targeting include problems walking or slurring words. While such effects are usually temporary, they can be permanent in 15 to 20 percent of cases, says Dr. Shah.

According to the National Institutes of Health, essential tremor affects up to 10 million Americans and Parkinsons disease impacts more than 1 million. Both are neurologic diseases thought to have genetic links. The first line of treatment for the involuntary trembling or shaking seen with these diseases is medication. However, approximately 30 percent of patients do not respond well to drugs, according to the study.

In the late 1990s, neurosurgeons began using a procedure called deep brain stimulation, opening the skull to permanently implant metal electrodes that could then be stimulated via a battery pack.

About a decade ago, a new MRI-guided procedure emerged that uses high-intensity ultrasound waves to heat and eliminate a small section of the thalamus linked to the disorders. MRI-guided HIFU is currently approved for treatment of essential tremor and tremors seen in Parkinsons disease patients. The outpatient procedure does not require opening the skull, and the patient is awake while it is performed, says Dr. Shah. No cuts. No anesthesia. No implanted devices.

A challenge in both procedures has been locating the precise area inside the brains thalamus to treat the pea-sized ventral intermediate nucleus, says Dr. Shah.

Traditionally, doctors have relied on either landmarks or maps of the brain drawn from cadavers to help them pinpoint the correct location. However, every brain is different, Dr. Shah says, and tiny errors can lead to damage in surrounding tissue, or to missing portions of the correct target.

Three newly refined MRI techniques are better at delineating the target tissue, according to the study.

The most widely studied and perhaps most promising imaging method is called diffusion tractography, says Dr. Shah. It creates precise brain images by taking into account the natural water movement within tissues.

The other methods described are quantitative susceptibility mapping which creates contrast in the image by detecting distortions in the magnetic field caused by substances such as iron or blood and fast gray matter acquisition TI inversion recovery which operates much like a photo negative, turning the brains white matter dark and its gray matter white in order to provide greater detail in the gray matter.

Dr. Shah and his team plan to participate in a multicenter clinical trial with collaborators at the Mayo Clinic in Rochester, Minnesota, testing the diffusion tractography method in patients.

Senior author of the study was Rajiv Chopra, Ph.D., director of image-guided therapy development and associate professor of radiology in the Advanced Imaging Research Center at UTSW. Researchers at the Mayo Clinic also participated in the study.

About UTSouthwestern Medical Center

UTSouthwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 25 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

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Advanced MRI Scans May Improve Treatment of Tremor, Parkinsons Disease - Newswise

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Direct-to-Consumer Genetic Testing market.

Trusted Business Insights presents an updated and Latest Study on Direct-to-Consumer Genetic Testing Market 2019-2026. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Direct-to-Consumer Genetic Testing market during the forecast period (2019-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Abstract, Snapshot, Market Analysis & Market Definition: Direct-to-Consumer Genetic Testing MarketIndustry / Sector Trends

Direct-to-Consumer Genetic Testing Market size was valued at USD 831.5 million in 2018 and is expected to witness 15.2% CAGR from 2019 to 2025.

U.S. DTC Genetic Testing Market Size, By Test Type, 2018 & 2025 (USD Million)

Rising prevalence of genetic diseases such as cystic fibrosis and Alzheimers globally is one of the major factors fostering direct-to-consumer genetic testing market growth. According to Global Genes, over 300 million people globally suffer from rare diseases. Recently developed DTC genetic tests allows consumer to identify probability of acquiring a specific genetic disease. Therefore, increasing adoption of DTC genetic testing for early disease detection and identification of genetic diseases will boost the industry growth over forecast timeline.

Increasing demand for personalized medications to treat genetic diseases will positively impact industry growth in forthcoming years. Individuals genome must be tested to develop personalized medicines. This increases the demand for DTC genetic kits since, it provides detailed information about individuals genetic predisposition. As detailed information regarding genetic makeup of individuals is easily available with the use of DTC genetic kits, researchers can easily design and develop personalized medicine that would help in faster patient recovery. Aforementioned factor is expected to drive the industry growth. However, high cost of DTC genetic testing kits may hamper industry growth to some extent during the forecast period.

Market Segmentation, Outlook & Regional Insights: Direct-to-Consumer Genetic Testing Market

Direct-to-Consumer Genetic Testing Market, By Test Type

Predictive testing segment will experience around 17% growth throughout the analysis period. Considerable segmental growth can be associated with rising prevalence of genetic diseases. Recently developed DTC genetic tests help to identify mutations that increase the chances of acquiring specific disease accurately. Surging awareness regarding benefits of such presymptomatic testing has reduced the mortality rates by enabling effective management of disease. Above mentioned factors have stimulated the segmental growth that is predicted to continue over the forecast timeframe.

Ancestry and relationship testing segment accounted for over 43% revenue share in 2018. Increasing awareness regarding ethnicity tests amongst the American and European population has increased the demand for DTC genetic tests. Accuracy and efficiency possessed by these tests has fostered segmental growth. Moreover, ancestry tests developed by companies such as Ancestry.com are user friendly. Availability of robust DTC ancestry tests providing meaningful clinical, genealogical and even forensic information will positively impact the segment growth.

Direct-to-Consumer Genetic Testing Market, By Technology

Targeted analysis segment was valued over USD 310 million in 2018. Targeted analysis is utilized for determining the defects in genes that are responsible for a particular disorder. Targeted genotyping can accurately measure an individuals gene pool that encodes important information regarding various diseases. Targeted analysis can be conducted at significant low cost compared to other available techniques that should augment its adoption rate over forecast timeframe.

Single nucleotide polymorphism segment will experience around 15% growth throughout the forecast period. Single nucleotide polymorphism chips specifically detect changes in single nucleotide that increases the efficiency of tests. For instance, SNP chips utilized for diagnosing hereditary cancers have detected 1300 mutations in BRCA2 genes. Various companies such as Ancestry.com and Color Genomics utilize SNP arrays that analyse gene sequences at a specific resolution and reveal detailed analysis about the defective genes that may in future cause certain disease. Increasing adoption of such advanced SNP chips in DTC testing kits will trigger the segmental growth.

Germany DTC Genetic Testing Market Size, By Technology, 2018 (USD Million)

Direct-to-Consumer Genetic Testing Market, By Region

North America direct-to-consumer genetic testing market accounted for around 39% regional share in 2018. Regional market growth can be attributed to increasing prevalence of genetic diseases. Rare genetic diseases such as thalassemia, hemophilia and anaemia require continuous and critical monitoring. According to CDC, every year more than 1,000 people are affected by thalassemia. Furthermore, American population has higher literacy rate and also, awareness regarding DTC tests is high amongst the American population that augments demand for DTC genetic tests.

Europe is estimated to experience around 15% growth over the coming years. European direct-to-consumer genetic testing market is highly regulated and for carrying out some of the genetic tests through DTC kits, customers are required to have physicians prescription. However, currently, European regulatory bodies are working on improving regulations set on DTC tests due to improved accuracy and efficiency possessed by them. Thus, improvement in regulatory scenario will positively impact regional market growth.

Latin America DTC Genetic Testing Market Size, By Country, 2025 (USD Million)

Key Players, Recent Developments & Sector Viewpoints: Direct-to-Consumer Genetic Testing Market

Few of the eminent industry players operating in direct-to-consumer genetic testing market are Ancestry, 23andMe, Color, Family Tree DNA, EasyDNA, Helix, Identigene, Full Genomes, Genesis HealthCare, Karmagenes, MyHeritage, MapMyGenome, Living DNA and Pathway Genomics. Chief industry players implement numerous initiatives such as mergers, acquisitions and new product launch to maintain their market position. Receiving approvals from regulatory bodies for new products will also foster companys revenue share. For instance, in October 2018, 23andme received first U.S. FDA approval for de novotechnology utilized in pharmacogenomic tests. This approval will enable company to launch innovative products, thereby fostering companys growth.

Direct-to-Consumer (DTC) Genetic Testing Industry Viewpoint

Direct-to-consumer genetic testing industry can be traced back to early 2000s. Earlier DTC tests were thought to be convenient as they would allow the patients to access their genetic information without involvement of physician. Although, DTC genetic testing kits had several benefits, in the initial days, they were stringently regulated by regulatory bodies. Regulatory scenario has always been stringent since the introduction of DTC genetic kits in European countries. Currently, there has been change in the regulatory scenario and European countries have started receiving approval for DTC genetic kits. Defects in the DTC kits have been reduced and people have started relying on these kits. DTC genetic testing market is sort of matured in North America due to numerous technological advancements and is still in developing phase in Asian countries. With further advancements in technology, DTC genetic tests industry will experience numerous growth opportunitie

Key Insights Covered: Exhaustive Direct-to-Consumer Genetic Testing Market1. Market size (sales, revenue and growth rate) of Direct-to-Consumer Genetic Testing industry.2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Direct-to-Consumer Genetic Testing industry.3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Direct-to-Consumer Genetic Testing industry.4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Direct-to-Consumer Genetic Testing industry.

Research Methodology: Direct-to-Consumer Genetic Testing Market

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THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.

Allogeneic HSCT is a multistep procedure:

For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.

Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.

Presenter:Dr Christina PetersAffiliation:Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract:#S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP

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Stem Cell Assay Market Overview

The Stem Cell Assay market report presents a detailed evaluation of the market. The report focuses on providing a holistic overview with a forecast period of the report extending from 2018 to 2026. The Stem Cell Assay market report includes analysis in terms of both quantitative and qualitative data, taking into factors such as Product pricing, Product penetration, Country GDP, movement of parent market & child markets, End application industries, etc. The report is defined by bifurcating various parts of the market into segments which provide an understanding of different aspects of the market.

The overall report is divided into the following primary sections: segments, market outlook, competitive landscape and company profiles. The segments cover various aspects of the market, from the trends that are affecting the market to major market players, in turn providing a well-rounded assessment of the market. In terms of the market outlook section, the report provides a study of the major market dynamics that are playing a substantial role in the market. The market outlook section is further categorized into sections; drivers, restraints, opportunities and challenges. The drivers and restraints cover the internal factors of the market whereas opportunities and challenges are the external factors that are affecting the market. The market outlook section also comprises Porters Five Forces analysis (which explains buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Stem Cell Assay) in addition to the market dynamics.

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Leading Stem Cell Assay manufacturers/companies operating at both regional and global levels:

Stem Cell Assay Market Scope Of The Report

This report offers past, present as well as future analysis and estimates for the Stem Cell Assay market. The market estimates that are provided in the report are calculated through an exhaustive research methodology. The research methodology that is adopted involves multiple channels of research, chiefly primary interviews, secondary research and subject matter expert advice. The market estimates are calculated on the basis of the degree of impact of the current market dynamics along with various economic, social and political factors on the Stem Cell Assay market. Both positive as well as negative changes to the market are taken into consideration for the market estimates.

Stem Cell Assay Market Competitive Landscape & Company Profiles

The competitive landscape and company profile chapters of the market report are dedicated to the major players in the Stem Cell Assay market. An evaluation of these market players through their product benchmarking, key developments and financial statements sheds a light into the overall market evaluation. The company profile section also includes a SWOT analysis (top three companies) of these players. In addition, the companies that are provided in this section can be customized according to the clients requirements.

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Stem Cell Assay Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the Stem Cell Assay market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of Stem Cell Assay Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Assay Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Assay Market, By Deployment Model

5.1 Overview

6 Stem Cell Assay Market, By Solution

6.1 Overview

7 Stem Cell Assay Market, By Vertical

7.1 Overview

8 Stem Cell Assay Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Assay Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Verified Market Research is a leading Global Research and Consulting firm servicing over 5000+ customers. Verified Market Research provides advanced analytical research solutions while offering information enriched research studies. We offer insight into strategic and growth analyses, Data necessary to achieve corporate goals and critical revenue decisions.

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Stem Cell Therapy Market Overview

The Stem Cell Therapy market report presents a detailed evaluation of the market. The report focuses on providing a holistic overview with a forecast period of the report extending from 2018 to 2026. The Stem Cell Therapy market report includes analysis in terms of both quantitative and qualitative data, taking into factors such as Product pricing, Product penetration, Country GDP, movement of parent market & child markets, End application industries, etc. The report is defined by bifurcating various parts of the market into segments which provide an understanding of different aspects of the market.

The overall report is divided into the following primary sections: segments, market outlook, competitive landscape and company profiles. The segments cover various aspects of the market, from the trends that are affecting the market to major market players, in turn providing a well-rounded assessment of the market. In terms of the market outlook section, the report provides a study of the major market dynamics that are playing a substantial role in the market. The market outlook section is further categorized into sections; drivers, restraints, opportunities and challenges. The drivers and restraints cover the internal factors of the market whereas opportunities and challenges are the external factors that are affecting the market. The market outlook section also comprises Porters Five Forces analysis (which explains buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Stem Cell Therapy) in addition to the market dynamics.

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Leading Stem Cell Therapy manufacturers/companies operating at both regional and global levels:

Stem Cell Therapy Market Scope Of The Report

This report offers past, present as well as future analysis and estimates for the Stem Cell Therapy market. The market estimates that are provided in the report are calculated through an exhaustive research methodology. The research methodology that is adopted involves multiple channels of research, chiefly primary interviews, secondary research and subject matter expert advice. The market estimates are calculated on the basis of the degree of impact of the current market dynamics along with various economic, social and political factors on the Stem Cell Therapy market. Both positive as well as negative changes to the market are taken into consideration for the market estimates.

Stem Cell Therapy Market Competitive Landscape & Company Profiles

The competitive landscape and company profile chapters of the market report are dedicated to the major players in the Stem Cell Therapy market. An evaluation of these market players through their product benchmarking, key developments and financial statements sheds a light into the overall market evaluation. The company profile section also includes a SWOT analysis (top three companies) of these players. In addition, the companies that are provided in this section can be customized according to the clients requirements.

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Stem Cell Therapy Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the Stem Cell Therapy market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of Stem Cell Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Therapy Market, By Deployment Model

5.1 Overview

6 Stem Cell Therapy Market, By Solution

6.1 Overview

7 Stem Cell Therapy Market, By Vertical

7.1 Overview

8 Stem Cell Therapy Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Contact us:

Mr. Edwyne Fernandes

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Stem Cell Alopecia Treatment Market Overview

The Stem Cell Alopecia Treatment market report presents a detailed evaluation of the market. The report focuses on providing a holistic overview with a forecast period of the report extending from 2018 to 2026. The Stem Cell Alopecia Treatment market report includes analysis in terms of both quantitative and qualitative data, taking into factors such as Product pricing, Product penetration, Country GDP, movement of parent market & child markets, End application industries, etc. The report is defined by bifurcating various parts of the market into segments which provide an understanding of different aspects of the market.

The overall report is divided into the following primary sections: segments, market outlook, competitive landscape and company profiles. The segments cover various aspects of the market, from the trends that are affecting the market to major market players, in turn providing a well-rounded assessment of the market. In terms of the market outlook section, the report provides a study of the major market dynamics that are playing a substantial role in the market. The market outlook section is further categorized into sections; drivers, restraints, opportunities and challenges. The drivers and restraints cover the internal factors of the market whereas opportunities and challenges are the external factors that are affecting the market. The market outlook section also comprises Porters Five Forces analysis (which explains buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Stem Cell Alopecia Treatment) in addition to the market dynamics.

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Leading Stem Cell Alopecia Treatment manufacturers/companies operating at both regional and global levels:

Stem Cell Alopecia Treatment Market Scope Of The Report

This report offers past, present as well as future analysis and estimates for the Stem Cell Alopecia Treatment market. The market estimates that are provided in the report are calculated through an exhaustive research methodology. The research methodology that is adopted involves multiple channels of research, chiefly primary interviews, secondary research and subject matter expert advice. The market estimates are calculated on the basis of the degree of impact of the current market dynamics along with various economic, social and political factors on the Stem Cell Alopecia Treatment market. Both positive as well as negative changes to the market are taken into consideration for the market estimates.

Stem Cell Alopecia Treatment Market Competitive Landscape & Company Profiles

The competitive landscape and company profile chapters of the market report are dedicated to the major players in the Stem Cell Alopecia Treatment market. An evaluation of these market players through their product benchmarking, key developments and financial statements sheds a light into the overall market evaluation. The company profile section also includes a SWOT analysis (top three companies) of these players. In addition, the companies that are provided in this section can be customized according to the clients requirements.

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Stem Cell Alopecia Treatment Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the Stem Cell Alopecia Treatment market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of Stem Cell Alopecia Treatment Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Alopecia Treatment Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Alopecia Treatment Market, By Deployment Model

5.1 Overview

6 Stem Cell Alopecia Treatment Market, By Solution

6.1 Overview

7 Stem Cell Alopecia Treatment Market, By Vertical

7.1 Overview

8 Stem Cell Alopecia Treatment Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Alopecia Treatment Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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AUTO1 continued favorable safety profile and high level of clinical activity, pivotal Phase 1b/2 AUTO1-AL1 program in adult ALL initiated and enrolling patients

- AUTO3 profile potentially supports use in a broader outpatient setting

Conference Call and Webcast to be held Friday, June 12, 2020at 7:30 am EDT / 12:30 pm BST

LONDON, June 12, 2020 --Autolus Therapeutics plc(Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced new data highlighting progress on its AUTO1 program, the companys CAR T cell therapy being investigated in the ongoing ALLCAR Phase 1 study of relapsed / refractory adult B-Acute Lymphocytic Leukemia (ALL), at the European Hematology Association EHA25 Virtual Congress beginning June 11.

AUTO1 in ALL

As of the data cut-off date ofMay 13, 2020, 19 patients had received AUTO1.AUTO1 was well tolerated, with no patients experiencing Grade 3 CRS. Three patients (16%) with high leukemia burden (>50% blasts) experienced Grade 3 neurotoxicity that resolved swiftly with the application of steroids. Of the 19 patients, 16 (84%) achieved MRD-negative CR.Two out of 16 patients received a transplant while in remission and CD19-negative relapse occurred in 3 (16%) patients. Durability of remissions is encouraging. Event Free Survival (EFS) and Overall Survival (OS) at 6 months are 62% and 72% respectively in all patients, and 76% and 92% respectively in the 13 patients treated with the closed (commercial) process. Median EFS and OS has not been reached, at a median follow up of 12.2 months (range up to 24.4 months).

I am very encouraged by the tolerable safety profile and high level of sustained CRs we have observed with AUTO1 in the ALLCAR19 study that was achieved without subsequent stem cell transplant, said Dr. Claire Roddie,Consultant Hematologist,UCL Cancer Institute and University College London Hospital.

Approximately 60% of adult ALL patients relapse or are refractory to first line therapy and there continues to exist a high unmet need, said Dr. Michael Bishop, MD, Professor of Medicine and Director of the Cellular Therapy Program atUniversity of Chicago Medicine.AUTO1 is a novel CD19 CAR T candidate with a compelling activity and safety profile and has the potential to change standard of care as a curative therapy for r/r ALL.

The data update on AUTO1 presented at this years EHA meeting show an encouraging durability of response without subsequent stem cell transplant and confirm the positive safety profile. said Dr.Christian Itin, chairman and chief executive officer of Autolus.We have started enrolment of patients with r/r aALL in our pivotal Phase 1b/2 AUTO1-AL1 study.

AUTO3 in DLBCL

Dr. Wendy Osborne presented ALEXANDER Phase 1/2 clinical trial data for AUTO3. This data is consistent with our update on May 29, 2020, with a data cut-off date of April 27, 2020.

These data are very encouraging, in terms of safety and tolerability, with a high level of clinical activity, said Dr. Wendy Osborne,Consultant Hematologist,Freeman Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust. We are looking forward to enrolling additional patients in the outpatient cohort.

Dr. Wendy Osborne of Newcastle upon Tyne Hospitals NHS Foundation Trust also discusses AUTO3 data during the American Society of Clinical Oncology (ASCO) Annual Meeting in this video courtesy of the Lymphoma Hub.

Investor call on Friday June 12, 2020

Management will host a conference call and webcast at7:30 am EDT/12:30 pm BST to discuss the EHA data. To listen to the webcast and view the accompanying slide presentation, please go to:https://www.autolus.com/investor-relations/news-and-events/events.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 4838626. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 4838626.

About Autolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

About AUTO1

AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety - while maintaining similar levels of efficacy - compared to current CD19 CAR T cell therapies.Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL. The company has also now progressed the program to a potential pivotal study, AUTO1-AL1.

About AUTO1-AL1 pivotal study

The AUTO1-AL1 study will enroll patients with relapsed / refractory ALL. The study will have a short Phase1b component prior to proceeding to a single arm Phase 2 study. The primary end point is overall response rate and the key secondary end points include duration of response MRD negative CR rate and safety. The study will enroll approximately 100 patients across 30 of the leading academic and non-academic centers in the US,UKandEurope.

About AUTO3

AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in diffuse large B cell lymphoma in the ALEXANDER clinical trial, with a 20-patient cohort that was initiated in Q2 2020 to assess feasibility of treatment in an outpatient setting.

Visit link:
Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress Pivotal AUTO1 study in adult ALL patients enrolling -...

NantKwest, Inc. (Nasdaq: NK), a clinical-stage, natural killer cell-based therapeutics company, today announced the publication of two peer-reviewed manuscripts in the Journal of Immunotherapy of Cancer. These invitro and in-vivo studies, conducted in collaboration with the National Cancer Institute pursuant to a Cooperative Research and Development Agreement, support the mechanism and functionality of NantKwests clinical-stage engineered natural killer (NK) cell lines, haNK and first-in-class PD-L1 t-haNKTM, as effecting anti-tumor activity in treatment-refractory cancer types even in the hypoxemic setting of the solid tumor microenvironment.

"These published studies provide important insight and validation for the mechanism and activity of our novel first-in-class engineered NK cells for use in notoriously difficult solid tumor types," said Patrick Soon-Shiong, M.D., Chairman and Chief Executive Officer of NantKwest. "NK cells have the potential to kill tumor cells; however, the hypoxic nature of the suppressive tumor environment has been shown to curb primary NK cell function. These published data indicate that our engineered haNK cells remain active in hypoxic conditions, which may be an important new mechanism of its anti-tumor activity. In addition, our NK cells appear to be resistant even to acute hypoxia and are capable of maintaining tumor killing activity in conditions comparable to a suppressive tumor microenvironment."

Dr. Soon-Shiong continued, "In addition, we are encouraged to observe anti-tumor activity in every cancer cell line tested by the investigators at NCI. The positive data in the in-vivo models of solid tumors with PD-L1 t-haNK, our engineered haNK cell line that also expresses a PD-L1 CAR, provides a novel approach to target tumors expressing PD-L1. This highly targeted NK cell therapy has the potential to address the evolution of tumors as they become resistant to chemotherapy, antibody therapy and, ultimately, checkpoint immunotherapy. We have hypothesized that cancer undergoes a quantum change and adapts to the therapy administered, resulting in the selection of resistant, cancer stem-like cells. It is at this stage of evolution where intractable tumors such as in patients with metastatic pancreatic cancer and triple negative breast cancer, are deemed incurable. It is our belief that these cancer "stem" cells, which do not divide and hence are untouchable by chemotherapy, become resistant and render checkpoint therapy futile by not expressing t-cell receptor ligands. In the face of this immunosuppressive milieu, our PD-L1 t-haNK cells can act to kill these otherwise highly resistant cancer cells, as demonstrated by these two important reports by our colleagues at the NCI. Our clinical results in the first patient with advanced metastatic pancreatic cancer to have received PD-L1 t-haNK demonstrated a durable complete response."

Study highlights from the publication titled "Overcoming hypoxia-induced functional suppression of NK cells" include:

Study highlights from the article titled "PD-L1-targeting high-affinity NK cells (PD-L1 t-haNK) induce direct antitumor effects and target suppressive MDSC populations" include:

The articles may be found on the Publications section of the NantKwest corporate website here: https://nantkwest.com/peer-reviewed-publications/

About NantKwestNantKwest (NASDAQ: NK) is an innovative, clinical-stage immunotherapy company focused on harnessing the power of the innate immune system to treat cancer and virally-induced infectious diseases. NantKwest is the leading producer of clinical dose forms of off-the-shelf natural killer (NK) cell therapies. The activated NK cell platform is designed to destroy cancer and virally-infected cells. The safety of these optimized activated NK cellsas well as their activity against a broad range of cancershas been tested in Phase I clinical trials in Canada and Europe, as well as in multiple Phase I and II clinical trials in the United States. By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs, NantKwests goal is to transform medicine by delivering off-the-shelf, novel NK cell-based therapies to routine clinical care. NantKwest is a member of the NantWorks ecosystem of companies. For more information, please visit http://www.nantkwest.com.

Story continues

haNK is a registered trademark of NantKwest, Inc.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that NantKwest will be successful in improving the treatment of cancer or other critical illnesses, including COVID-19. Risks and uncertainties related to these endeavors include, but are not limited to, obtaining FDA approval of NantKwests NK cells as well as other therapeutics and manufacturing challenges.

Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements.

These and other risks regarding NantKwests business are described in detail in its Securities and Exchange Commission filings, including in NantKwests Quarterly Report on Form 10-Q for the quarter ended March 31, 2020. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200610005251/en/

Contacts

Jen Hodson NANT Jen@nant.com

See the original post:
NantKwest Announces Studies in Collaboration with the National Cancer Institute Demonstrating Enhanced Killing Activity of First-in-Class PD-L1 t-haNK...

Three people with the inherited blood diseases sickle cell and beta thalassemia remain free of burdensome blood transfusions and their worst symptoms, months after receiving an infusion of genetically modified stem cells.

One of the three, a young woman with a severe form of beta thalassemia, has now been followed for over a year since she was treated, while the second, a woman in her 30s with sickle cell disease, is more than nine months removed from her infusion. They are the first two patients in pioneering studies of a therapy, developed by CRISPR Therapeutics and Vertex, that's based on the gene editing technology known as CRISPR.

Both patients continue to respond to treatment, bolstering evidence of genetic medicine's potential to permanently alter the course of devastating hereditary conditions like sickle cell and transfusion-dependent beta thalassemia. A gene therapy developed by Bluebird Bio has shown similar potential.

First results from the two studies, disclosed last November, were "taking the promise of CRISPR and turning that into a reality," said Samarth Kulkarni, CRISPR Therapeutics' CEO, in an interview. The additional data and follow-up now available "show these effects can be long-lasting and durable."

And in beta thalassemia, the first patient's experience is now supported by results from another patient who was treated about five months ago. This individual has also been able to stop receiving blood transfusions.

Taken together, the two patients responses are "proof of concept," CRISPR Therapeutics and Vertex claim, that their approach to treating beta thalassemia has the potential to be curative.

In sickle cell, the companies are also hopeful. The one patient for whom they have data has not had a vaso-occlusive crisis, a painful episode caused by the disease's characteristic sickling of red blood cells, since her treatment.

"The clinical manifestation of the disease is different, but we see consistent outcomes across both diseases," said Bastiano Sanna, Vertex's head of cell and genetic therapies, in an interview.

Three other beta thalassemia patients and one other sickle cell disease patient have been treated in the two studies of CRISPR Therapeutics and Vertex's therapy, dubbed CTX001. If results continue to look positive, CTX001 could be another powerful way to help people for whom treatment options have long been limited.

CRISPR, an easy-to-use method of genetic surgery that's derived from a bacterial defense system, has become a mainstay in labs across the world for all types of experiments. Its potential use as a human therapeutic has drawn closer as companies harnessing the technology CRISPR Therapeutics, Editas Medicine and Intellia Therapeutics have advanced their research. CRISPR Therapeutics is the first of the three to deliver results from a clinical trial.

CRISPR and Vertex unveiled their updated results at the European Hematology Association's virtual meeting on Friday. Also being presented were the latest data from Bluebird's gene therapy, known as LentiGlobin.

Bluebird is much further along, having treated 60 patients with beta thalassemia and 37 with sickle cell disease across six different studies.

Updated results from three of those studies showed 23 of 27 evaluable patients with beta thalassemia were transfusion independent for at least a year following treatment. And in sickle cell, no serious vaso-occlusive crises were observed in the 18 patients who had at least six months of follow-up. An episode was previously reported in one patient several months after LentiGlobin treatment, but was judged to be non-serious.

One sickle cell patient died suddenly 20 months following infusion with LentiGlobin, Bluebird reported Friday. Both the treating physician and an independent study committee concluded the death, ruled to be cardiovascular in nature, was unlikely to be related to the gene therapy.

Both beta thalassemia and sickle cell are diseases caused by mutations in the beta globin gene, faulty DNA that results in either absent or warped hemoglobin. Without enough hemoglobin, patients' red blood cells can't carry needed oxygen throughout the body. And those with sickle cell have abnormal hemoglobin that makes red blood cells fragile and stiff, causing them to stick in blood vessels.

Both diseases require chronic blood transfusions, and can lead to organ damage and reduced lifespans. Treatment options are limited, although that's now changing. The Food and Drug Administration, over the past few years, has approved Reblozyl, for beta thalassemia, and Oxbryta and Adakveo, for sickle cell.

Adakveo reduces the frequency of vaso-occlusive crises, while Reblozyl and Oxbryta are chronic medicines meant to boost patients' hemoglobin levels.

CRISPR Therapeutics and Vertex, along with Bluebird, are trying to accomplish the same goal but in more dramatic fashion: raising hemoglobin levels high enough so patients can stop blood transfusions and, in sickle cell, avoid pain crises altogether.

CRISPR and Vertex use CRISPR/cas9 gene editing to modify the DNA of stem cells extracted from a patient's bone marrow. The cells are engineered to produce a type of hemoglobin that's present at birth but normally replaced soon after. Once returned to the body and engrafted in the bone marrow, these CRISPR'd cells substitute this so-called fetal hemoglobin for the missing adult hemoglobin.

In the three patients treated so far, that appears to be what's happened. Both beta thalassemia patients are producing hemoglobin at levels considered normal. The sickle cell patient now has enough fetal hemoglobin to dilute the effects of sickled hemoglobin, potentially helping to preserve red blood cells.

Crucially, CRISPR and Vertex shared data for the first time indicating a high percentage of edited cells are present in each patient's bone marrow, supporting their confidence that the effects of treatment might last.

Bluebird, by contrast, doesn't edit the DNA of extracted stem cells, but rather inserts a modified gene into those cells. Once infused and engrafted in a patient, the cells can produce gene therapy-derived hemoglobin.

In most beta thalassemia and sickle cell patients treated with Bluebird's LentiGlobin, hemoglobin levels rose to normal or near-normal levels.

LentiGlobin is already approved for certain beta thalassemia patients in Europe as Zynteglo. In the U.S., Bluebird has hit delays and pushed back when it expects to submit an application to the middle of next year. A filing for an accelerated approval in sickle cell would likely follow sometime in the second half of 2021.

CRISPR and Vertex, meanwhile, plan to enroll more patients into their two studies, which they hope could serve as sufficient for an approval application if positive, Kulkarni said.

Read the original here:
New CRISPR, gene therapy results strengthen potential for treatment of blood diseases - BioPharma Dive

Finding Provides New Potential Approaches to Treating Fatal Disease

Researchers at the University of Maryland School of Medicine (UMSOM) have identified how certain gene mutations cause amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease. The pathway identified by the researchers may also be responsible for a certain form of dementia related to ALS. The finding could offer potential new approaches for treating this devastating condition, which causes progressive, fatal paralysis and sometimes mental deterioration similar to Alzheimers disease. Their discovery was published this week in theProceedings of the National Academy of Sciences (PNAS)and included collaborators from Harvard University, University of Auckland, Kings College London, and Northwestern University.

More than 5,000 Americans are diagnosed with ALS every year, a condition that is usually fatal and has no cure. Patients with ALS slowly lose the ability to move their muscles, leading to problems with basic functions such as breathing and swallowing. About half of ALS patients also develop dementia. Genetic studies of families with a predisposition to develop ALS have shown that the condition can be associated with certain gene mutations. Some of these mutations involve the gene UBQLN2 which regulates the disposal of misfolded garbage from the bodys cells. Until now, researchers did not fully understand how UBQLN2 mutations interfere with this pathway and cause ALS.

Mervyn Monteiro, PhDWe mapped out the process by which ubiquilin-2 (UBQLN2) gene mutations disrupt an important recycling pathway that cells use to get rid of their trash, saidMervyn Monteiro, PhD, Professor of Anatomy and Neurobiology, who is affiliated with the UMSOMs Center for Biomedical Engineering and Technology (BioMET) at UMSOM. Without this recycling, misfolded proteins build up in the nerve cell and become toxic, eventually destroying the cell. This destruction could lead to neurodegenerative disorders like ALS.

To investigate how UBQLN2 mutations cause ALS, Dr. Monteiros group used both human cells and UBQLN2-mutant mouse models for their investigations. The mouse models, which they described in a 2016PNAS publication, mimic the progression of the disease in people who inherit these gene mutations.

Dr. Monteiros group first removed the UBQLN2 gene from human cells and found it completely stalled the recycling pathway. They then reintroduced either the normal gene or one of five gene mutations into the cells. They found that reintroduction of normal UBQLN2 restored the recycling pathway while all five of the gene mutations failed to restart the pathway.

Using the mouse model, Dr. Monteiro and his colleagues outlined the reason for the pathway disruption in the presence of gene mutations. They found that the mice with the gene mutations had reduced levels of a certain protein called ATP6v1g1, which is an essential part of a pump that acidifies the cells trash container in order to initiate the breakdown and recycling process.

Our new findings are exciting because similar acidification defects have been found in Alzheimers, Parkinsons and Down syndrome, Dr. Monteiro said. This suggests that restoration of the defect could have broad implications for not only treating ALS, but possibly other neurodegenerative diseases as well.

The research study was supported by grants from the Packard Center for ALS Research at Johns Hopkins, the ALS Association, and the National Institutes of Health (grant number: R01-NS100008).

The BioMET research team led by Dr. Monteiro continues to make important advances in understanding the mechanisms that give rise to ALS, saidDean E. Albert Reece, MD, PhD, MBA, who is also Executive Vice President for Medical Affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor, University of Maryland School of Medicine. Future treatments and preventive measures for this devastating disease would not be possible without this foundational work.

Read more:
UM School of Medicine Researchers Identify New Genetic Defect Linked to ALS - BioSpace

June 10, 2020 In a new study, Johns Hopkins researchers found that testing people for SARS-CoV-2 (COVID-19) too early in the course of infection is likely to result in a false negative test, even though they may eventually test positive for the virus.[1] This is important to understand since many hospitals are using these COVID tests to screen patients before imaging exams, diagnostic testing or procedures.

The report found even a week after infection, one in five people who had the virus had a negative test result. The findings was published in the May 13 issue of Annals of Internal Medicine.

A negative test, whether or not a person has symptoms, doesnt guarantee that they arent infected by the virus, said Lauren Kucirka, M.D., Ph.D., M.Sc., obstetrics and gynecology resident at Johns Hopkins Medicine. How we respond to, and interpret, a negative test is very important because we place others at risk when we assume the test is perfect. However, those infected with the virus are still able to potentially spread the virus.

Kucirka said patients who have a high-risk exposure should be treated as if they are infected, particularly if they have symptoms consistent with COVID-19. This means communicating with patients about the tests shortcomings. One of several ways to assess for the presence of SARS-CoV-2 infection is a method called reverse transcriptase polymerase chain reaction (RT-PCR). These tests rapidly make copies of and detect the viruss genetic material. However, as shown in tests for other viruses such as influenza, if a swab misses collecting cells infected with the virus, or if virus levels are very low early during the infection, some RT-PCR tests can produce negative results. Since the tests return relatively rapid results, they have been widely used among high-risk populations such as nursing home residents, hospitalized patients and healthcare workers. Previous studies have shown or suggested false negatives in these populations.

For the new analysis, Johns Hopkins Medicine researchers reviewed RT-PCR test data from seven prior studies, including two preprints and five peer-reviewed articles. The studies covered a combined total of 1,330 respiratory swab samples from a variety of subjects including hospitalized patients and those identified via contact tracing in an outpatient setting.

Using RT-PCR test results, along with reported time of exposure to the virus or time of onset of measurable symptoms such as fever, cough and breathing problems, the researchers calculated the probability that someone infected with SARS-CoV-2 would have a negative test result when they had the virus infection. In the published studies, healthcare providers collected nasal and throat samples from patients and noted the time of virus exposure or symptom onset and sample collection.

From this data, the Johns Hopkins researchers calculated daily false-negative rates, and have made their statistical code and data publicly available so results can be updated as more data are published.

The researchers estimated that those tested with SARS-CoV-2 in the four days after infection were 67 percent more likely to test negative, even if they had the virus. When the average patient began displaying symptoms of the virus, the false-negative rate was 38 percent. The test performed best eight days after infection (on average, three days after symptom onset), but even then had a false negative rate of 20 percent, meaning one in five people who had the virus had a negative test result.

We are using these tests to rule out COVID-19, and basing decisions about what steps we take to prevent onward transmission, such as selection of personal protective equipment for healthcare workers, Kucirka explained. As we develop strategies to reopen services, businesses and other venues that rely on testing and contact tracing, it is important to understand the limitations of these tests.

Ongoing efforts to improve tests and better understand their performance in a variety of contexts will be critical as more people are infected with the virus and more testing is required. The sooner people can be accurately tested and isolated from others, the better we can control the spread of the virus, the researchers said.

Another John Hopkins study in March found the average incubation period for COVID-19 was approximately five days.[2] This was originally used as a guide by some in developing quarantine guidelines. However, this new study shows that test COVID PCR test results are not reliable for a firm diagnosis until well after a week of infection.

Additional authors include Denali Boon, Stephen Lauer, Oliver Layendecker and Justin Lessler and of Johns Hopkins.

Funding for the study was provided by the National Institute of Allergy and Infectious Diseases (R01AI135115 and T32DA007292), the Johns Hopkins Health System and the U.S. Centers for Disease Control and Prevention (NU2GGH002000).

Reference:

1. Lauren M. Kucirka, Stephen A. Lauer, Oliver Laeyendecker, et al. Variation in False-Negative Rate of Reverse Transcriptase Polymerase Chain ReactionBased SARS-CoV-2 Tests by Time Since Exposure. Annuals of Internal Medicine. May 13, 2020. doi.org/10.7326/M20-1495.

2. Stephen A. Lauer, Kyra H. Grantz, Qifang Bi, et al. The Incubation Period of Coronavirus Disease 2019 (COVID-19) From Publicly Reported Confirmed Cases: Estimation and Application. Ann Intern Med. 2020 Mar 10 : M20-0504. Published online 2020 Mar 10. doi: 10.7326/M20-0504.

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COVID-19 Genetic PCR Tests Give False Negative Results if Used Too Early - Imaging Technology News

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Biorepository project initiated early in pandemic to streamline coronavirus research efforts

Philip Mudd, MD, PhD, picks up research samples at the BJC Institute of Health at Washington University School of Medicine in St. Louis. Mudd, an emergency medicine physician, and his colleague, Jane O'Halloran, MD, PhD, an infectious diseases specialist, have led efforts to create a repository for storing and managing specimens collected from patients with COVID-19. The samples, including blood, urine and saliva specimens, are being distributed to investigators conducting COVID-19 research across the university.

In the weeks before the St. Louis region saw its first patients with COVID-19, physician-scientists at Washington University School of Medicine began planning and preparing how best to collect blood and other biological samples from such patients so specimens could be quickly disseminated to researchers seeking strategies to treat, prevent and contain the novel coronavirus.

With financial support from The Foundation for Barnes-Jewish Hospital, Siteman Cancer Center and Washington Universitys Institute of Clinical and Translational Sciences (ICTS), as well as input from the Community Advisory Board of Washington Universitys Institute for Public Health and ICTS, the School of Medicine created a repository to store and manage specimens collected from adult and pediatric patients who have tested positive for SARS-CoV-2 the virus that causes COVID-19. The sample-collection efforts are led by Philip Mudd, MD, PhD, an assistant professor of emergency medicine, and Jane OHalloran, MD, PhD, an assistant professor of medicine.

To date, more than 350 patients have donated samples for the research effort, and over 7,000 samples have been distributed to more than 20 labs on the School of Medicine and Danforth campuses to help understand the basic biology of the infection and seek ways to prevent or treat it.

The biorepository is an important resource for scientists working in many different areas of COVID-19 research, Mudd said. We are hopeful that the analysis of these samples will speed the development of treatments and new diagnostics that will help patients in St. Louis and around the world. Were grateful for the support of the foundations donors and for the generosity of our patients and are optimistic that this work will lead to new and useful breakthroughs in the treatment of COVID-19.

According to the investigators, collecting such samples including blood, urine, stool and nasal swabs via one centralized process speeds research, prevents scientists from duplicating work already underway and relieves patients of the burden of being asked to participate in multiple studies.

The project includes research teams that span the entire university, including investigators who are internationally recognized for their research into other viruses, such as Ebola, SARS, West Nile, Zika and emerging strains of influenza.

Studies underway include research to:

To receive samples for research, investigators must submit an application to a committee that is part of Washington Universitys ICTS, led by William G. Powderly, MD, the J. William Campbell Professor of Medicine and co-director of the universitys Division of Infectious Diseases. The ICTS committee managing patient specimens led by Christina A. Gurnett, MD, PhD, the A. Ernest and Jane G. Stein Professor of Developmental Neurology and director of the Division of Pediatric and Developmental Neurology is fielding many requests and encouraging a team science approach to enhance collaboration and reduce duplication of efforts. The biorepository has already led to new collaborations for researchers with complementary skills.

Its important to streamline this work from a research perspective, but its equally important to ease the process of participation for our patients, OHalloran said. Ordinarily, individual researchers design studies and approach patients separately. Knowing we would have many scientists studying COVID-19, it was critical for us to find a way to facilitate the process of obtaining patient samples. Were grateful to our patients for providing such a valuable resource to help us understand and combat this new virus.

Because some of the research may involve sequencing the DNA of patients with COVID-19 to understand each persons susceptibility to the illness, the researchers consulted the Community Advisory Board for guidance on how to address treatable genetic conditions independent of COVID-19 that might be revealed during the course of the research. The board, made up of 12 people with diverse leadership experience in local health-care and community organizations, provides advice to investigators on the conduct of clinical research involving the local community.

Anytime an individual undergoes whole genome sequencing, even people who may appear perfectly healthy, there is the potential to reveal genetic mutations that indicate a very high risk of developing a disease. Because some genetic conditions have treatments or prevention strategies available, doctors conducting COVID-19 research would like to have the ability to return this important information to patients, with patient permission, in a way that will allow patients and family members to make informed decisions about their health.

As part of studying the genetics of COVID-19, the researchers may learn information about a research participants risk of developing any one of 59 hereditary conditions that lead to very high risk of developing life-threatening but preventable or treatable diseases such as breast cancer, heart arrhythmias or blood-clotting disorders, said advisory board member Doug Lindsay, a personal medical consultant who advises patients with rare diseases or complex conditions. How do you best provide this kind of information if at all to patients or family members who are totally preoccupied with the immediate crisis of COVID-19 illness?

The overall thrust of our advice is, Give patients the choice to know or not know, Lindsay said. We also feel it is best to deliver this information at a later time, to give the patients, or family members if the patient has passed away, a chance to make an appointment to go over follow-up questions about the COVID-19 research study that they or their family members were enrolled in. The genetic studies of COVID-19 can reveal hereditary conditions, so the information is relevant for family members, too. We feel it is important to give people the mental space to deal with major medical decisions independent of the immediate pandemic.

The committee also has recommended that genetic counselors be made available to patients who are found to have any of these 59 conditions and choose to learn their genetic status. The committee also wants to ensure that patients and families know the kinds of medical specialists they should follow up with based on their genetic information.

These questions are about what a healthy relationship between participants and investigators looks like, Lindsay said. Participants who donate their data to these studies are real people. The information that the doctors are identifying may shape lives over more than one generation. We hope this kind of advice can help researchers be good stewards of the information put in their care.

Added Gurnett, The guidance provided by the Community Advisory Board has been critical to helping our investigators and clinicians provide the best possible care and conduct clinical research in the most ethical and respectful ways possible. We thank the patients and their families for their central contributions to the work of understanding this virus, as their participation is key to reducing its impact on our communities.

The Institute of Clinical and Translational Sciences is supported by the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH), grant number UL1 TR002345.

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, ranking among the top 10 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.

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Patients with COVID-19 donate specimens to advance research efforts - Washington University School of Medicine in St. Louis

Newswise Irvine, CA June 12, 2020 A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

The study, published today in Nature Communications, titled, Quantification of ongoing APOBEC3A activity in tumor cells by monitoring RNA editing at hotspots, reveals how the genomic instability induced by the protein APOBEC3A offers a previously unknown vulnerability in cancer cells.

Each day, in human cells, tens of thousands of DNA damage events occur. In cancer cells, the expression of the protein APOBEC3A is one of the most common sources of DNA damage and mutations. While the mutations caused by these particular proteins in cancer cells contribute to tumor evolution, they also cause breaks in the DNA, which offer a vulnerability.

Targeting cancer cells with high levels of APOBEC3A protein activities and disrupting, at the same time, the DNA damage response necessary to repair damages caused by APOBEC3A, could be key to more effective cancer therapies, said Remi Buisson, PhD, senior investigator and an assistant professor in the Department of Biological Chemistry at the UCI School of Medicine. However, to exploit the vulnerability of the cancer cells, it is critical to first quantitatively measure the proteins activity in tumors.

To understand the role of APOBEC3A in tumor evolution and to target the APOBEC3A -induced vulnerabilities, the researchers developed an assay to measure the RNA-editing activity of APOBEC3A in cancer cells. Because APOBEC3A is difficult to quantify in tumors, developing a highly sensitive assay for measuring activity was critical. Using hotspot RNA mutations, identified from APOBEC3A-positive tumors, the team developed an assay using droplet digital PCR and demonstrated its applicability to clinical samples from cancer patients.

Our study presents a new strategy to follow the dysregulation of APOBEC3A in tumors, providing opportunities to investigate the role of APOBEC3A in tumor evolution and to target the APOBEC3A-induced vulnerability in therapy, said Buisson. We anticipate that the RNA mutation-based APOBEC3A assay will significantly advance our understanding of the function of the protein in tumorigenesis and allow us to more effectively exploit the vulnerabilities it creates in cancer therapy.

This study was funded in part by the National Institutes of Health, a California Breast Cancer Research Program grant and an MPN Research Foundation Challenge grant.

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI Researchers Uncover Cancer Cell Vulnerabilities; May Lead to Better Cancer Therapies - Newswise