One of the biggest barriers to applying precision medicine to clinical trials and patient engagement is getting different digital health systems to talk to each other.

Seqster is one company tackling such issues of interoperability, and its CEO and co-founder Ardy Arianpour joined the pharmaphorum podcast to discuss how it applies a person-centric approach to empowering patients.

The San Diego-based tech start-ups research portal gathers patient data, such as electronic health records, genetic information and fitness results from wearables, to improve the way health data can be shared.

Its an aim thats high on the agenda of bodies such as the Centers for Medicare and and Medicaid Services (CMS) and the office of the National Coordinator for Health Information Technology (ONC) in the US, whose new interoperability rules have just been finalised.

Seqsters research portal currently connects users to over 3,000 healthcare providers and more than 100,000 hospitals and clinics in the US, giving patients a way to manage all their health data from one mobile app.

The firm recently secured strategic investment from Takeda Pharmaceuticals, via its Takeda Digital Ventures arm. The pharma company has said it wants Seqster to be a cornerstone of its digital health strategy, and Ardy also talked about what the deal involves.

A biotech and digital health entrepreneur, heplayed an instrumental role in expanding genetic testing access with the launch of BRCA testing and was a key player in the 2013 landmark SCOTUS decision scrapping gene patents.

Prior to starting Seqster,Ardylaunched several clinical and consumer-based genetic tests as CCO of Pathway Genomics and served as SVP of Ambry Genetics, which was sold to Konica in 2017 for $1 billion.

You can listen to episode 18 of thepharmaphorum podcastin the player below, download the episode to your computer or find it and subscribe to the rest of the series in iTunes, Spotify, acast and Stitcher.

Digital health and interoperability: the pharmaphorum podcast

 

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Digital health and interoperability: the pharmaphorum podcast - - pharmaphorum

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Last week, the global number of confirmed cases of novel coronavirus or COVID-19 crossed 110,000 in over 100 countries, with a significant number of new cases emerging in South Korea, Iran and Europe. Concern over the extent of the pandemic has already affected both the worlds financial markets as well as the economy. Although India has been relatively less affected so far with only 43 confirmed cases as of 9 March it is too early in the international disease cycle to relax. China managed to contain the spread by enacting a cordon sanitaire around lakhs of people, using authoritarian measures that might be hard to implement elsewhere in the world. So, the global outbreak could get a lot worse, and if it does, we will certainly be affected.

That is why Indiaand the Narendra Modi governmentmust take a national approach tomanagethe risks of a COVID-19 epidemic in India. Such an approach would not only pull together all government departments and institutions, but also involve the private sector and civil society. (Disclosure: A member of my immediate family works for a private genetic testing firm.)

It is important to say this because thus far, the task of addressing theCOVID-19 has been delegated exclusively to the government. Almost all activities, from airlifting Indian nationals, screening arrivals at airports, testing samples, quarantining and treatment are carried out by the government. While this will be effective if the number of cases remainsinthe current order of magnitude, the governments facilities alone will not be sufficient if the number of cases rises 100timesor more.

The good news is that India has a private healthcare sector and R&D capability that can be used in the fight. The bad news is that were not letting them.

If weevenhave a fewlakh suspectedcases, the governments resources will fall short of what is required. The right time to think about the capacity required to handle such a massive crisis is now. The single most important thing for India to have a national response as opposed to a government response is to enable the private healthcare ecosystem to play an appropriate role to complement the governments efforts wherever possible.

Also read: Improve surveillance, screen pneumonia cases how experts want India to fight coronavirus

One of the most critical areas where private capability should be harnessed is in screening and testing. The greater our success in detectingCOVID-19 cases early, the greater our ability to contain the outbreak. Faster testing means fewer people in unnecessary quarantine, lower load on hospitals and faster contact tracing.

Currently, there are52governmentlaboratoriesin ICMRs Virus Research & Diagnostic Laboratories (VRDLs) that are equipped to carry outCOVID-19tests. Another 57 VRDL network labs are authorised to collect samples through state government health authorities. All of these operate under the aegis of the National Institute of Virology (NIV), Pune. As of6March, a total of 4058 samples from 3404 individuals have been tested by the network. The ICMR-NIV-VRDL network has the capacity to handle perhaps a 10X increase in the number of samples and is bound to have procured test kits to handle such a load.

Currently,private laboratories are not permitted to test forCOVID-19. In anticipation of a greater surge, theModigovernment must enable the most reliable private players to conduct suchtests as well. There are 53 private laboratories in India accredited by the College of American Pathologists (CAP) and hundreds more by the National Accreditation Board for Testing and Calibration Laboratories (NABL). Some of Indias best private laboratories can be carefully selected by the respective state governmentsbased on their technical capabilities, biosafety levels, quality standards and track records.

We can double the number of test facilities in a few precious weeks, and increase it even more over a longer period. Many private laboratories already offer commercial testing for other viral diseases, so with incremental effort,they can test forCOVID-19 too. This will also create incentives for private Indian companies with R&D capabilities to consider developing test kits for COVID-19,which currentlyhave to be imported from China and Europe. It is in Indias strategic interest to be able to develop probes, reagents and test kits for the current and future viral epidemics.

Also read: With centre-state political standoff, can coronavirus outbreak be effectively fought?

In addition to mandating standards and safety levels, ICMR must insist on compulsory and real-time reporting of samples and test results so that the government authorities have a single database to workwith. There should be no expectation of government funding or subsidy, just as there is no case forthegovernment to intervene in the pricing of these tests.

As the number of cases crossed 400 last week, the United States not only permitted private laboratories to performCOVID-19 tests, but alsochanged policyto enable laboratories to use tests they develop faster in order to achieve more rapid testing capacity in the United States. Canada has distinguished itself byanopen approach decentralising and opening up testing from the very outset. These are prudent and responsible responses to a feared escalation ofapublic health emergency. India with greater vulnerabilities and weaker public health systems should take a similar approach.

The author is the director of the Takshashila Institution, an independent centre for research and education in public policy. Views are personal

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India can fight COVID-19, but only if the private sector is allowed to step in quickly - ThePrint

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New Jersey, United States,- The Preimplantation Genetic Testing Market is well prepared, focusing on the competitive landscape, geographic growth, segmentation and market dynamics, including drivers, constraints and opportunities. It highlights key production, sales and consumption trends so players can improve their sales and growth in the Preimplantation Genetic Testing Market. It offers a detailed analysis of the competition and the leading companies in the Preimplantation Genetic Testing Market. Here it focuses on the latest developments, sales, market values, production, gross margin and other important factors in the business of top players operating in the Preimplantation Genetic Testing Market.

Global Preimplantation Genetic Testing Market was valued at USD 306.16 million in 2016 and is projected to reach USD 701.49 million by 2025, growing at a CAGR of 9.65% from 2017 to 2025.

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The various contributors to the value chain in the Preimplantation Genetic Testing Market include manufacturers:

With a comprehensive quantitative and qualitative analysis, the report offers an encyclopedic and accurate research study on important aspects of the Preimplantation Genetic Testing Market. It shows key factors that influence the growth of various segments and regions in the Preimplantation Genetic Testing Market. It also offers SWOT, Porters Five Forces and PESTLE analyzes to thoroughly examine the Preimplantation Genetic Testing Market. It contains a detailed study of manufacturing costs, upstream and downstream buyers, dealers, marketing strategies and development trends for marketing channels in the Preimplantation Genetic Testing Market. It also provides strategic advice and recommendations for players to ensure success in the Preimplantation Genetic Testing Market.

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Table of Contents:

Overview: The report begins with an overview of the Preimplantation Genetic Testing Market, in which the authors discuss the scope of the products, type and application segments as well as the regional markets. This section also contains highlights of the market size analysis.

Competition by manufacturers: Here, the analysts give the production share, the share of sales and the average price of the manufacturers for the reporting period 2014-2019. Readers are also provided with details on products, areas served and production facilities by manufacturers. This section contains another chapter that highlights various competitive situations and trends.

Share of production by region: This section shows the gross margin, price, production and revenue of all regional markets examined in the report.

Key players: Each player profiled in the report is rated for market growth based on served markets, core business, price, sales, gross margin, production, manufacturing locations, served areas and other factors.

Production cost analysis: It includes the analysis of the main raw materials, the analysis of the production cost structure, the analysis of the manufacturing process and the analysis of the industrial chain.

Market forecast: It includes the price and trend forecast, the sales and growth rate forecast and the forecast of the production growth rate of the global and regional markets for the forecast period 2019-2026.

Finally, the Preimplantation Genetic Testing Market offers a general conclusion of research and the feasibility of investing in new projects is assessed. The Preimplantation Genetic Testing Market is a valuable guide for individuals and companies interested in selling the market.

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TAGS: Preimplantation Genetic Testing Market Size, Preimplantation Genetic Testing Market Growth, Preimplantation Genetic Testing Market Forecast, Preimplantation Genetic Testing Market Analysis, Preimplantation Genetic Testing Market Trends, Preimplantation Genetic Testing Market

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Preimplantation Genetic Testing Market to See Major Growth By 2026 - Monroe Scoop

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Not everyone diagnosed with long-QT syndrome (LQTS) needs to take beta blockers to cut their risk for sudden death, despite guidelines that recommend all such patients be given the drugs, say researchers based on their retrospective cohort study.

Such patients who are judged to be at exceptionally low risk from their LQTS and who want to avoid the often oppressive side effects of beta blockers can safely follow an "intentional nontherapy" strategy, the authors conclude.

The approach avoids the use of beta blockers, which the guidelines call for even in those with genetically confirmed LQTS but who are asymptomatic and whose electrocardiograms are normal.

The nontherapy also excludes implantable cardioverter defibrillators (ICDs) and denervation surgery for LQTS but maintains standard precautions, such as avoidance of QT-interval-prolonging medications.

"We are certainly overtreating this entity overall. Way too many ICDs are being put into too many long-QT patients whose risk is nominally low," Michael J. Ackerman, MD, PhD, Mayo Clinic, Rochester, Minnesota, told theheart.org | Medscape Cardiology.

"Maybe some of our long-QT patients don't need any intentional therapy," he said. "If you have somebody who's at extraordinarily low risk, and you're confident in your low-risk forecast, maybe they don't need a beta blocker."

Ackerman is senior author on the report, which was published February 24 in Heart Rhythm. The lead author is Ciorsti J. MacIntyre MD, Dalhousie University, Halifax, Canada.

"Patients themselves should not make the leap to think that they can come off beta blockers. They really have to talk to a physician who knows and is comfortable dealing with long-QT-syndrome patients," Mark S. Link, MD, told theheart.org | Medscape Cardiology.

"The thing to get across from the study, and to make sure everyone knows, is that this is a very highly selected group of long-QT patients that came off beta blockers because of perceived low-risk status," said Link, of the University of Texas Southwestern Medical Center, Dallas, who isn't associated with the current report.

Importantly, Ackerman and the report emphasize, a decision to follow the intentional nontherapy strategy calls for shared decision making after "detailed risk assessment and patient education."

But whether to take beta blockers can be a quality-of-life dilemma for many patients who have indications, Ackerman observed. Given that that the drugs are recommended universally for patients who are identified as having LQTS, "You can take a very low-risk person who never had a disease symptom and now give them a new disease called, 'I feel lousy every single day of my life.' "

In the current analysis, which Ackerman likens to a "progress report" on the strategy, 55 such patients with LQTS but no history of symptoms were intentionally not treated with beta blockers, device therapy, or surgery.

They represented 8.3% of a total of 661 patients with clinically or genetically diagnosed LQTS seen over a 17-year period at a major center. In that subgroup, there were no LQTS-triggered cardiac events or cardiac deaths over a mean follow-up of 7.5 years, the investigators reports. Even among the remaining 606 patients, there was only one death.

Among the intentionally nontreated patients, QTc intervals averaged 448 ms. On genetic testing, 85.5% of these patients tested positive for a form of LQT, and 73% had a family history of LQTS.

None in the nontreated group had symptoms; by comparison, 32% of those who took beta blockers had symptoms. For the nontreated group, resting QTc intervals averaged 448 ms, vs 469 ms for the treated group (P < .001).

Most of the nontreated patients in the analysis had started on beta blockers, "hated it, then moved to intentional nontherapy," Ackerman said.

But these days, "if we've sized up their risk forecast to be incredibly low, then we are slowly becoming supportive of not even starting beta-blocker therapy," he said.

Link pointed to limitations of the analysis. For example, there were only 55 patients in the beta-blocker nontherapy group, "and when you consider the risk of a long-QT patient having an event, it's pretty low over a 2- or 3- or 4-year time period, even over a lifetime if they are a low-risk LQTS patient," he said.

"It's a small number of patients and relatively small follow-up compared to a lifetime risk," Link said. A longer follow-up might have shown more events in the beta-blocker nontherapy group, he said, so "to say there's no risk I think would be stretching it based on the data from this study."

Ackerman said his center's LQTS program has now had over 1300 patients, compared to fewer than 700 covered by the analysis. About 10% of them were selected for intentional nontherapy after discussion with the patient and family, he said. For perspective, "I think a similar percentage of patients probably deserve aggressive ICD therapy."

The hypothetical patient he would be "most confident about moving to nontreatment," Ackerman said, might be an asymptomatic man with type-1 LQTS who is found to be genotype positive by cascade screening after age 40 and whose QTc interval is <440 ms. "That man, by everything we know about the disease, is as close to a zero-risk host that we know of."

Ackerman has consulted for Audentes Therapeutics, Biotronik, Boston Scientific, Daiichi Sankyo, Gilead Sciences, Invitae, Medtronic, MyoKardia, and St. Jude Medical and, along with his institution, has a licensing agreement with AliveCor. MacIntyre has received honoraria or speaker fees from Abbott and Medtronic. The other authors and Link have disclosed no relevant financial relationships.

Heart Rhythm. Published online February 20, 2020. Abstract

Follow Steve Stiles on Twitter: @SteveStiles2. For more from theheart.org | Medscape Cardiology, follow us on Twitter and Facebook.

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Going Off Beta Blockers as an Option in Selected Low-Risk LQTS - Medscape

Recommendation and review posted by Bethany Smith

First, by their nature, pluripotent stem cells can potentially be used to create any cell or tissue the body might need to counter a wide range of diseases, from diabetes to spinal cord injury, to childhood leukemia, to heart disease.

Second, pluripotent stem cells can potentially be customized to provide a perfect genetic match for any patient. This means that patients could receive transplants of tissue and cells without tissue matching and tissue rejection problems, and without the need to take powerful immune-suppressing drugs for the rest of their lives. Although this vision hasnt yet been achieved, researchers at Boston Childrens Hospital have successfully treated mouse models of human disease using this strategy and hope that the same can be done with patients.

Disease in a dish:Third, pluripotent stem cells make excellent laboratory models for studying how a disease unfolds, which helps scientists pinpoint and track the very earliest disease-causing events in cells. Immune deficiencies, Type 1 diabetes, muscular dystrophy, and myriad other disorders are rooted in fetal development. In the lab, researchers can recapture these early originsobserving where the first muscle cell comes from, or the first blood cell, and how this differs when the patient has a genetic disease. Using this information, doctors may be able to intervene and correct the genetic defect before the disease advances.

Unique applications:Each type of pluripotent stem cell has different characteristics that make it useful in different ways, and each has different lessons to teach.

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Why are Pluripotent Stem Cells Important? Boston ...

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Keeping the Upper Hunter's Liddell power station open for an extra three years would have disastrous environmental and human health consequences, a national environment advocacy group has warned. It comes as Energy Minister Angus Taylor refused to rule out the possibility that the federal government could spend $300 million to keep the power station open past 2023 or even purchase it directly. Using the results of a 2018 study by Newcastle-based epidemiologist Dr Ben Ewald, Environmental Justice Australia calculated air pollution from Liddell Power station would cause an additional 41 premature deaths if it remained open for an extra three years. The extension would also cause an additional 33 low-birthweight babies and 51 additional cases of type 2 diabetes from exposure to pollutants such as sulfur dioxide and nitrogen dioxide. "Saving taxpayers $300 million is an obvious reason for shredding this ridiculous plan but preventing more people from dying prematurely from serious illnesses like stroke, cancer and heart disease, is a compelling enough reason on its own," Director of Research and Advocacy at Environmental Justice Australia Nicola Rivers said. "The health bill from air pollution costs Australians more than 4000 lives and $24 billion a year. Energy Minister Angus Taylor should stop trying to find ways to keep this ageing and inefficient power station open and put the health of the community first." Liddell's owner, AGL Energy , gave seven years notice that it planned to close the 48-year-old plant by 2023. But Mr Taylor lobbied to keep it open until 2026 and set up the Liddell Taskforce to investigate extending its operations. Department officials have played down reports that the government was about to purchase the power station outright from AGL or provide the $300 million required to keep it operating beyond its forecast closure date. The work of the Liddell taskforce had not yet completed and that the report to be delivered to energy minister Angus Taylor was still being finalised, a budget estimates hearing heard on Tuesday. Mr Taylor also told ABC's 7.30 Report on Monday that a decision on the government's response to the looming closure of the Liddell power station would be made after the taskforce had delivered its recommendations. "I haven't received the final report yet but what I will say is that it is crucial that as... these power stations get older, we have either life extension or replacement and that is to ensure that we have downward pressure on prices," Mr Taylor said. It is estimated that the state's five power stations collectively emit enough uncontrolled air pollution to cause 279 deaths each year. While you're with us, did you know the Newcastle Herald offers breaking news alerts, daily email newsletters and more? Keep up to date with all the local news - sign up here IN NEWS TODAY

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Keeping the Upper Hunter's Liddell power station open for an extra three years would have disastrous environmental and human health consequences, a national environment advocacy group has warned.

It comes as Energy Minister Angus Taylor refused to rule out the possibility that the federal government could spend $300 million to keep the power station open past 2023 or even purchase it directly.

The extension would also cause an additional 33 low-birthweight babies and 51 additional cases of type 2 diabetes from exposure to pollutants such as sulfur dioxide and nitrogen dioxide.

"Saving taxpayers $300 million is an obvious reason for shredding this ridiculous plan but preventing more people from dying prematurely from serious illnesses like stroke, cancer and heart disease, is a compelling enough reason on its own," Director of Research and Advocacy at Environmental Justice Australia Nicola Rivers said.

"The health bill from air pollution costs Australians more than 4000 lives and $24 billion a year. Energy Minister Angus Taylor should stop trying to find ways to keep this ageing and inefficient power station open and put the health of the community first."

Liddell's owner, AGL Energy , gave seven years notice that it planned to close the 48-year-old plant by 2023. But Mr Taylor lobbied to keep it open until 2026 and set up the Liddell Taskforce to investigate extending its operations.

The work of the Liddell taskforce had not yet completed and that the report to be delivered to energy minister Angus Taylor was still being finalised, a budget estimates hearing heard on Tuesday.

Mr Taylor also told ABC's 7.30 Report on Monday that a decision on the government's response to the looming closure of the Liddell power station would be made after the taskforce had delivered its recommendations.

"I haven't received the final report yet but what I will say is that it is crucial that as... these power stations get older, we have either life extension or replacement and that is to ensure that we have downward pressure on prices," Mr Taylor said.

It is estimated that the state's five power stations collectively emit enough uncontrolled air pollution to cause 279 deaths each year.

While you're with us, did you know the Newcastle Herald offers breaking news alerts, daily email newsletters and more? Keep up to date with all the local news - sign up here

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Why do some people feel like they need three cups of coffee just to get through the day when others are happy with only one? Why do some people abstain entirely? New research suggests that our intake of coffee the most popular beverage in America, above bottled water, sodas, tea, and beer is affected by a positive feedback loop between genetics and the environment.

This phenomenon, known as quantile-specific heritability, is also associated with cholesterol levels and body weight, and is thought to play a role in other human physiological and behavioral traits that defy simple explanation.

It appears that environmental factors sort of set the groundwork in which your genes start to have an effect, said Paul Williams, a statistician at Lawrence Berkeley National Laboratory (Berkeley Lab). So, if your surroundings predispose you to drinking more coffee like your coworkers or spouse drink a lot, or you live in an area with a lot of cafes then the genes you possess that predispose you to like coffee will have a bigger impact. These two effects are synergistic.

Williams findings, published in the journal Behavioral Genetics, came from an analysis of 4,788 child-parent pairs and 2,380 siblings from the Framingham Study a famous, ongoing study launched by the National Institutes of Health in 1948 to investigate how lifestyle and genetics affect rates of cardiovascular disease. Participants, who are all related to an original group from Framingham, Massachusetts, submit detailed information about diet, exercise, medication use, and medical history every three to five years. Data from the study have been used in thousands of investigations into many facets of human health.

Paul T. Williams. (Credit: Roy Kaldschmidt/Berkeley Lab)

Williams used a statistical approach called quantile regression to calculate what proportion of participants coffee drinking could be explained by genetics as the study follows families and what must be influenced by external factors. Past research shows that the most significant environmental factors influencing coffee drinking are culture and geographic location, age, sex, and whether or not one smokes tobacco; with older male smokers of European ancestry drinking the most, overall.

The analysis indicated that between 36% and 58% of coffee intake is genetically determined (although the exact causative genes remain unknown). However, confirming Williams hypothesis that coffee drinking is a quantile-specific trait, the correlation between a parents coffee drinking and an offsprings coffee drinking got increasingly stronger for each offsprings coffee consumption quantile, or bracket (for example, zero cups per day, one to two cups, two to four cups, and five or more cups).

When we started to decode the human genome, we thought wed be able to read the DNA and understand how genes translate into behavior, medical conditions, and such. But thats not the way its worked out, said Williams, who is a staff scientist in Berkeley Labs Molecular Biophysics & Integrated Bioimaging (MBIB) Division. For many traits, like coffee drinking, we know that they have a strong genetic component weve known coffee drinking runs in families since the 1960s. But, when we actually start looking at the DNA itself, we usually find a very small percentage of the traits variation can be attributed to genes alone.

The traditional assumption in genetic research has been that ones surroundings and lifestyle alter gene expression levels in consistent and measurable ways, ultimately creating the outward manifestation called a phenotype of a trait. Williams statistics work shows that the situation is more complex, which helps explain the diversity of traits we see in the real world.

MBIB Division Director Paul Adams commented, Pauls statistical studies complement the genomics research that Berkeley Lab bioscientists conduct to learn more about the relationship between genes and the environment.

Next, Williams plans to assess whether quantile-specific heritability plays a role in alcohol consumption and pulmonary function. This is a whole new area of exploration that is just now opening up, he said. I think it will change, in a very fundamental way, how we think genes influence a persons traits.

This research was funded by a grant from the National Institute of Environmental Health Sciences and a gift from HOKA ONE ONE. The Framingham Study Data were made available through the Biologic Specimen and Data Repository Information Coordinating Center of the National Heart, Lung, and Blood Institute.

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Complex Biology Behind Your Love (or Hatred) of Coffee - Mirage News

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For plenty of canine house owners, thunderstorms are a supply of angst, a stroll to the canine park could be a fraught enjoy, and New 12 monthss celebrations are in particular worrying. In step with a brand new learn about of hundreds of puppy canine, anxiousness and fear-related habits issues are popular. Sure breeds are in particular delicate to loud noises or being left on my own. Different breeds might have interaction in compulsive behaviors reminiscent of biting themselves or urinating, suggesting a genetic part to the job.

James Serpell, an ethologist on the College of Pennsylvania, who used to be now not concerned within the learn about, says that the issue stems from house owners failing to correctly socialize their canine. Many dogs rescued from shelters will have been inadequately skilled once they had been younger, and the issue is compounded when new house owners are overly wary with them. Its a type of helicopter-parenting idea implemented to canine, he says. Animals arent getting sufficient publicity to standard social interactions, play habits and roughhousing with different canine. Thats requesting bother.

Within the learn about, Hannes Lohi, a geneticist on the College of Helsinki, and his colleagues surveyed Finnish house owners of 13,715 puppy canineor just about 2 % of the overall inhabitants of the animals in Finland. The canine house owners answered to questions concerning the canine age, socialization,and behaviour round people and unfamiliar canine and in new environments. The researchers printed their effects on Thursday in Medical Reviews. About 72 % of the canine exhibited problematic behaviors reminiscent of aggression or fearfulness. In the meantime 32 % of them had been petrified of noises, which used to be the commonest type of anxiousness, and about one quarter had been afraid of fireworks particularly. Sensitivity to loud noises greater with age. More youthful canine tended to break assets or urinate when left on my own extra continuously than older animals did. And male canine had been extra hyperactive and competitive than feminine ones.

Lohi additionally discovered that canine breeds had very other behavioral profiles.Romagnolos had been possibly to be petrified of thunder, and Labrador retrievers had been least more likely to be. Extra miniature schnauzers, and less Labradors, had been competitive towards strangers than different canine. Blended breeds had the best ranges of inattentiveness, a trait proven in canine regarded as onerous to coach. The breed specificity of those characteristics means that genetics performs a job of their building, Lohi says.

In a learn about he co-authored in January 2019 in Translational Psychiatry, Lohi and his colleagues discovered a gene in German shepherds connected to age-dependent listening to defects and anxiousness. However its not actually recognized whether or not this can be a [physical] or psychiatric factor, he says.

The brand new learn about additionally tested comorbidities, or other stipulations found in the similar animal. Concern and noise sensitivity had been commonplace comorbidities, even supposing this may increasingly were since the pattern incorporated such a lot of canine that exhibited each and every trait. And separation-related anxiousness habits used to be extra commonplace amongst canine that had been delicate to noise. Serpell is skeptical about calling those observations comorbidities, on the other hand. The time period has a tendency to suggest {that a} pathology is concerned, however those are standard canine behaviors, he explains. As an alternative, Serpell says, I might be expecting there to be an affiliation between worry and aggression. Numerous aggression in canine is induced through worry.

Nicholas Dodman, a veterinary behaviorist at Tufts College and leader scientist on the Heart for Dog Habits Research, who used to be now not inquisitive about Lohis research, printed a identical paper on behavioral issues within the July-August 2019 factor of theMagazine of Veterinary Habits. There are some inherent pitfalls to the questionnaire-based way, which practice to our learn about as neatly, Dodman says. Surveying house owners about their canine habits assumes theyre vigilant witnesses, he says. And seeing a definite habits in a dog, such because the animal scratching itself, does now not give an explanation for why it shows that habits. The statement does now not permit an inference to be made, as an example, that the canine has advanced a compulsive dysfunction.

Human variety for characteristics reminiscent of herding or guarding will have predisposed some breeds to interact in compulsive behaviors, Lohi says. Within the new learn about, border collies, that have been bred to herd cattle, had been extra vulnerable to chasing lighting and shadows, while Staffordshire bull terriers had been the possibly to chase their very own tails, an impulsive habits that means a genetic defect has been enriched in that breed, he provides.

Breeding systems can step by step do away with such characteristics through averting canine with behavioral issues that experience a genetic part, Lohi says. However selective breeding for any trait has dangers, says Anindita Bhadra, a behavioral biologist on the Indian Institute of Science Schooling and Analysis, who additionally used to be now not a part of the brand new learn about. Making an attempt to reproduce anxiety-free canine may just result in different issues, she says. We all know that the majority advanced characteristics are multigenic, and synthetic variety continuously ends up in inadvertent adjustments whilst deciding on for a collection of characteristics, Bhadra provides.

Lohi says the next move in figuring out those behavioral problems is to tease aside what environmental, way of life and genetic possibility components predispose canine to them. The overarching objective of the analysis is in the long run to know the way helpful canine will also be to type human anxiousness and what sort of they proportion identical possibility or protecting components, he says. Ultimately, this might lend a hand to advance the well being and welfare throughout species.

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3 Fourths of Canines Are Angst-Ridden-and Homeowners Might Be Partially to Blame - periodical360

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When Emma Hodcroft read that, seemingly out of nowhere, a rash of cases of the novel coronavirus had popped up in Britain in late January, she started collecting media reports on them, searching the articles for clues as to how it had moved to the island nation. Early reports suggested that a lone traveler from Singapore, who was unaware he was infected with virus, had visited a French chalet for a few days and had spread the virus to others at the ski resort. This intrigued Hodcroft, who is half British and a postdoctoral researcher in evolutionary biologist Richard Nehers lab at the University of Basel in Switzerland, where she uses genetics to study and track diseases. She took notes on the cases that were associated with the infected traveler. At first, there wasnt that much information and the story was simple, she tells The Scientist. But more and more cases kept appearing, and she found it hard to keep track of who had traveled to which country and when they were diagnosed.

Hodcroft decided to generate an infographic showing the connections between the traveler from Singapore and the other coronavirus cases emerging in Europe. I thought, Ill make an image and see if anyone else finds this useful, she says. She posted the image on Twitter, and somewhat unexpectedly, it got a lot of attention, she says. People were definitely really, really interested in this. So I kept that image updated over the next week or so. As she updated it, the graphic showed that at least 21 people were exposed to the virus at the ski resort the traveler from Singapore visited; 13 of those people ended up developing COVID-19, the disease caused by the virus. After shed finished the preliminary work, a colleague of Hodcroft saw it and suggested she write it up for publication. She posted the paper on February 26; the next day it appeared in Swiss Medical Weekly.

Hodcroft talked with The Scientist about the work, how its conclusions have been supported by genetic testing of viral strains from patients, and what it tells us about the spread of the virus, SARS-CoV-2, in other countries.

Emma Hodcroft: Firstly, that it seems like so many people [at least 13] could be infected by a single person. It seems like they were infected by the man who traveled from Singapore. So thats quite a lot of forward transmission on his part in a fairly short time period; he was only in France for about four days. Of course, this could be some unusual event that doesnt normally happen, but it lets us put an outer bound on what is possible even if it is not common.

The other thing thats surprising is that, according to the patient statement that he released, the focal patient never had any symptoms. In his own words, he never felt sick. So he did all of this transmission without ever having any indication that he was unwell or that he should be taking any precautions to modify his behavior. It tells us that some infections might be from people who never even know that theyre sick.

Text continues below infographic

Contact tracing showing the spread of SARS-CoV-2 in a particular cluster of patients in Europe.

EH: As far as we can tell, no one from this cluster had severe symptoms. It seems like some people did have some symptoms, but they were never serious. And thats also interesting because it shows that if we didn't know about this outbreak, its pretty likely that these people would have kind of written this off as a bad cold or the flu. None of them would have ended up going to hospital or significantly changing their behavior. And again, this indicates that it might be quite hard, and it is becoming quite hard, to contain this virus because some people don't feel very unwell, such that they would change their behavior or go for testing.

EH: In the US, from the information available, it still doesnt seem like the US has really ramped up testing. We dont know the number of tests that have been performed because its come down off of the CDC website, which is a little concerning. But at least the last reports that were given to us show the US was really lagging behind most countries in the number of tests that it had done.

A few days ago, the research group called the Seattle Flu Study, which is designed to take community samples from random people who have any kind of cough, runny nose, or cold-like symptoms and look for the fluthey pivoted and started testing some of the samples for coronavirus. They found a case in the Seattle area and sequenced the viral genome of the infected person [posted on NextStrain] and showed it links very closely with another case in the Seattle area thats from mid-January. And so this strongly suggests (though we dont yet know for certain) that there has been ongoing undetected transmission in Seattle since mid-January and wasnt picked up because we werent looking for it. This has become clearer in the last few days, as more cases and even deaths have been reported in Washington State. That tells us the virus hasnt just appeared in the last few days in the area.

Text continues below graphic

The viral genome of the first case in Washington (USA/WA1/2020) is identical to Fujian/8/2020. The genome of the virus from a second case in Washington (USA/WA2/2020) is identical to the first Washington case, except it has three additional mutations. This suggests WA1 was a traveler from China bringing the virus to Snohomish County, Washington in mid-January, where the virus circulated undetected for about five weeks, a timespan that explains why WA2 is so similar genetically, with a few mutations. The graphic shows the connection to the other cases with viral sequences now available.

EH: This virus causes respiratory illness, which can make you feel unwell for a few days and then you get better or it can progress. If the illness progresses it can cause lung damage that makes the person more susceptible to other illnesses, such as bacterial infection. This can be treated too and for many people that treatment turns the course of the infection, but some dont and the effort can essentially delay their death. So the infection may have occurred weeks [before a person dies]. This is not something intrinsic to this virus, however. With respiratory illness, its usually something that takes a substantial amount of infection and lung damage before you succumb to it.

EH: Sequencing can tell us a lot about what is happening with the virus right now. The Washington samples are a perfect example. . . . Without having these genomes, we never would have seen this signal of ongoing transmission, which we saw just before the case explosion in Washington. And on the flip side we can tell when cases are coming in from other countries. We have another genome from Washington State thats grouping with genomes that we know have a travel history to Italyso it seems like this could be a case where [an infected person] came back from Italy.

When you have a very small number of cases of a disease, you can do this just through epidemiological contact tracing: you can go to everyone and ask questions and find out the connections between the cases. As the case numbers scale up, this becomes very hard to do. With genetic sequencing, we can do this without having to go and try and figure out where everyone was at the time of infection. Weve had an influx of sequences from Brazil, Switzerland, Mexico, Scotland, Germany. These have clustered with sequences from Italy and have a travel history from Italy and so from that we can show that Italy really is now exporting cases around the world to multiple countries.

EH:Theres been a lot of modeling, not only with genetics but epidemiologically in the last few weeks, and we had pretty strong indications that circulation was wider than publicly thought. At the time, we did try to some extent to get this message out to government health agencies and the public in general. I do think that in the future, incorporating a little bit more of that scientific expertise perhaps into the public dialogue and government decision-making could make a big difference. The earlier that you can act in an epidemic, you have more effect you can have, because one person goes on to infect a few more people who go on to infect a few more people. Its much harder once that has gone up to 10 [infected] people, than if you can stop with person one.

One thing I would note is that studies have shown that limiting transportation really doesnt make much of an impact for outbreaks. Quarantining particular cities, if they seem to be epicenters, can work as a preventive measure, but as the epidemic scales up, you move past being able to contain it in this sense, [and] what you end up doing is just disrupting supply routes, interrupting business, making all of these things much harder.

Editors note: This interview has been edited for brevity.

Ashley Yeager is an associate editor atThe Scientist. Email her at ayeager@the-scientist.com. Follow her on Twitter @AshleyJYeager.

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Coronaviruss Genetics Hint at its Cryptic Spread in Communities - The Scientist

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What would the implications be if decoding your genes cost less than a pair of designer jeans? We might soon find out after a Chinese company claimed it can sequence the human genome for $100.

The speed at which the price of genetic sequencing has fallen has been astonishing, from $50,000 a decade ago to roughly $600 today. For a long time, the industry saw the $1,000 genome as the inflection point at which we would enter the genomic agewhere getting a read out of your DNA would be within reach for huge swathes of the population.

That milestone has come and gone, but progress hasnt stopped. And now Chinese firm BGI says it has created a system that can sequence a full genome for just $100. If the claims hold up, thats a roughly six times improvement over state-of-the-art technology.

The key to the breakthrough is a significant increase in the size of the chip that is used to analyze genetic data, so twice as many genomes can be processed at once. Their machine also uses a robotic arm to dunk the chip into baths of the chemicals used to carry out the sequencing process, which allows them to be reused multiple times.

The company says the system, which will be made available to customers late this year, is aimed at large-scale genomics projects and could make it possible to decode the DNA of 100,000 people a year.

The breakthrough could spur further price falls as well by breaking the stranglehold that industry leader Illumina has had on the market. Dennis Grishin, co-founder of startup Nebula Genomics, told MIT Tech Review that he believed the reason the price of genetic sequencing had remained stuck around $1,000 in recent years was due to Illuminas near monopoly.

A $100 genome could significantly broaden the scope of what we can do with genetic data. The growing field of population genetics promises to uncover the genetic quirks that set different groups of people apart, which can prove vital for developing new medicines and understanding the susceptibility of different groups to certain conditions.

While some ambitious projects, such as the UK Biobank project aimed at collating genetic data on 500,000 people, are already underway, the cost of sequencing has so far limited the scope of these projects. A dramatically cheaper system could see these kinds of initiatives become far more commonplace, greatly expanding our understanding of genetic diversity among humans.

By bringing the cost of full genome sequencing within reach of everyday people, the approach could also dramatically expand the scope of personalized medicine. While services like 23andMe have seen a huge expansion in consumer genetic testing, these services only decode a small fraction of the genome that isnt particularly useful for medical purposes.

DNA sequencing is already used to tailor cancer treatment by determining how peoples genetics are likely to influence their response to certain treatments, but it is still far from standard practice. At $100 the practice could become far more common and also be expanded to predict responses to a host of other treatments, ushering in a new era of personalized medicine.

Theres also hope that it would enable new tests that could provide early warning of susceptibility to a host of genetic diseases, or even sequence the DNA of patients microbiomes to detect imbalances in their gut flora that might be responsible for certain conditions or impact their responses to certain treatments.

Rade Drmanac, chief scientific officer of Complete Genomics, a division of BGI, told MIT Tech Review that at $100 it could soon be common to sequence the DNA of every child at birth. This could provide unprecedented early-warning for a host of diseases, but would also open up a Pandoras box of ethical concerns.

The movie Gattaca already explored the potential for discrimination when genetic testing becomes trivially easy, particularly when paired with increasingly powerful genetic engineering that is bringing the potential for designer babies ever closer.

Perhaps more importantly though, our understanding of how our genetics impact our lives is still very hazy. While we have identified some genes that strongly influence propensity for certain diseases, most human characteristics are governed by complex interactions between multiple genes whose activity can vary throughout our lives in response to environmental pressures.

Our ability to read our DNA is far ahead of our ability to understand it, which could lead to all sorts of problemsfrom creating a new class of worried well flagged as at risk of certain conditions that never come to be, to unnecessarily medicalizing or stigmatizing patients in ways that alter the trajectories of their lives.

With a $100 genome now within reach, we will have to tackle these issues with urgency to make sure the genomic age is one to look forward to rather than one to fear.

Image Credit: Pete Linforth from Pixabay

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$100 Genome Sequencing Will Yield a Treasure Trove of Genetic Data - Singularity Hub

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THE LOST FAMILYHow DNA Testing Is Upending Who We AreBy Libby Copeland

DNA is not love, notes a woman in The Lost Family, a new book about consumer genetic testing. Disappointed to learn that a man she thought was her half brother wasnt after all, she was underscoring that reality as revealed by DNA may not match reality as actually experienced in families.

This idea surfaces repeatedly in Libby Copelands well-researched exploration of the consequences intended and otherwise of Americans increasingly common practice of sending saliva samples to companies like 23andMe and AncestryDNA. The growing and assertively marketed industry then gives customers information about their background and allows them to find relatives by comparing their DNA with that of others in the companies databases.

For many, the results help flesh out family trees they already recognize. Others, including people adopted as children or conceived with donor sperm, can resolve longstanding questions about their birth parents. But some receive shocking surprises, like an NPE, shorthand for non-paternity event or not the parent expected revelations that ones mother or father is not who the person thought they were. The Lost Family explains the rise of the consumer genetics industry and how search angels citizen-scientist genetic genealogy experts devote hours to helping seekers solve DNA mysteries.

Copeland reiterates a few central questions throughout the book: How much of your sense of yourself should scientists and algorithms be allowed to dictate?; What makes us who we are?; and Is it always better to know the truth? Of course, the answers are up to the individual reader and will vary based on things like family dynamics and how earthshaking the consequences of knowing might be. Copelands balanced treatment of the subject allows readers to reach their own conclusions and shows them many of the factors they might consider as they do.

Theres a section on the pros and cons of companies ability to share consumer genetic information with researchers and pharmaceutical companies which could lead to better medical treatments, but also to a host of privacy issues. For instance, genetic ancestry databases to solve cold criminal cases, a practice thats taken off since it helped identify the notorious Golden State Killer.

Copeland also discusses the slipperiness of genetic ethnicity estimates, which are much less precise than the matching of individual family members. A test result might categorize someone as 37 percent Scandinavian or Irish, but as companies reference databases expand and their algorithms sharpen, such estimates could shrink or grow. The databases are also lopsided, containing more samples from people with European backgrounds than those with African or Latin American ancestry.

The book describes the controversial history, and future potential, for genetic information to be misguidedly conflated with racial and ethnic categories, even though science has documented more genetic differences within distinct population groups than between them. Attempts to twist genetic information to draw boundaries between groups have seeded societal toxicities like eugenics and white nationalism. And even people lacking nefarious motivation can blunder by linking genes and heritage too closely, Copeland writes. She cites the criticism of Elizabeth Warrens assertion that her 2018 DNA test suggesting she likely had at least one Native American ancestor six to 10 generations ago supported Warrens longstanding belief, based on her familys stories, that she was part Cherokee and Delaware Indian.

The Lost Family intersperses expository sections with an intermittent narrative of the step-by-step journey of Alice Collins Plebuch, a woman who is thrown for a loop when AncestryDNA results contradict her impression that her forebears were Irish, English and Scottish. Readers might find the unfurling of Plebuchs story a bit too attenuated, but her eventual discovery illustrates the hidden history that genetic testing can uncover.

Copeland drops in stories of other people who ferreted out family secrets. For some, like the man who learned his grandfather was African-American, not Italian, the information adds richness and context to their lives. For others, like the woman who received a letter from a lawyer asking her to stop contacting her biological father, or the woman whose half siblings wanted nothing to do with her, the denouement can be painful.

[ As Dani Shapiro recounts in her latest memoir, Inheritance, a few years ago she took a DNA test and discovered that she was only half Jewish and unrelated to the woman she had always thought was her half sister. ]

DNA testing reveals a truth that is black andwhite, even if the circumstances are gray, and then, everybody has to grapple with it, Copeland writes. Still, she says that anecdotes suggest most people are glad they unearthed the facts, even if the consequences were difficult. Hopefully, thats true because, the book contends, so many Americans are sending spit samples to DNA databanks that many genetic nuggets will inevitably be surfaced whether people want to keep them buried or not.

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Before You Spit in That Vial, Read This Book - The New York Times

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In the day and age of genetic testing, people are able to trace their roots back generations. Kate Middleton apparently has many distant celebrity relatives, including her husband Prince William and comedian Ellen Degeneres. But many may not know that sibling actresses Dakota and Elle Fanning are also related to the Duchess of Cambridge, and they share a very interesting ancestor.

Prince William and Kate Middleton had a fairytale romance, and the world has adoringly watched her rise to royalty. Born to parents Michael and Carole Middleton, Kate comes from an upper-middle-class upbringing with no direct royal ties. But when her marriage to Prince William was confirmed, genealogists began digging through her ancestry.

As reported by Channel 4 News Patrick Cracroft-Brennan, Kate was technically a commoner before she married William, but her bloodline can be traced back to King Edward III.

Her great-great-grandmother, Frances Elizabeth Greenhow, was the 10 x great-granddaughter of Sir William Gascoigne, a Yorkshire knight who died in 1487, said Cracroft-Brennan. He married Lady Margaret Percy, 4th and youngest daughter of Henry Percy, Earl of Northumberland. The Earl descended from King Edward III through both his parents. Prince Charles and the late Princess Diana both descend from Sir William Gascoigne and his wife Lady Margaret.

With this lineage, Cracroft confirmed that Kate and William are fourteenth cousins once removed through his mother and fifteenth cousins through his father.

From George Washington to Jane Austen, it turns out Kate Middleton has plenty of noteworthy relatives. But two of her most famous celebrity relatives in modern times are sibling actors Elle and Dakota Fanning.

According to People, after digging through seven centuries of historical records (including birth and death certificates, census records, and burial and baptism records), researchers at Ancenstry.com found that the Fanning sisters are relatives of King Edward III through their mom, Heather Arrington.

The historians discovered that Elle and Dakota are 22nd great-grandaughters of the noted King who ruled England in the 1300s. Generation after generation, the lines we looked at pieced back directly to King Edward III proving that Elle is a direct descendant of royalty, a historian for Ancestry.com, told People. You can consider her a long-lost princess. This connection is so unique and rare.

With King Edward III in common, Kate and the Fanning sisters are distant cousins. In an interview E!, Elle talked about how wild the whole thing is. Its so weird, she told the outlet. I saw it the other day I looked online and like King Edward III is my great-great-great-grandfather. I was like, Is this real?'

When Tatler asked Elle what she thought of Kate, she was all praise. But she also admitted she wouldnt be able to handle the scrutiny that comes with being a royal. Kate and Meghan are doing royal femininity very differently, said the younger Fanning sister. I cant imagine being in the public eye like that. Every single move you make is critiqued.

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Is Kate Middleton Related to Dakota and Elle Fanning? - Showbiz Cheat Sheet

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Bone remodeling and regeneration are dependent on resident stem/progenitor cells with the capability to replenish mature osteoblasts and repair the skeleton.

Until now, it has been thought that stem cells for bone lie within the bone marrow and the outer surface of the bone. Many studies have described the existence of a network of vascular channels that helped distribute blood cells out of the bone marrow. However, none of the studies had proved the existence of cells within these channels.

A new study by the scientists from the UConn School of Dental Medicine has discovered the population of stem cells that reside along the vascular channels within the cortical bone and have the ability to generate new bone. These stem cells stretch across the bone and connect the inner and outer parts of the bone.

Lead investigator Dr. Ivo Kalajzic, professor of reconstructive sciences, said, This is a discovery of perivascular cells residing within the bone itself that can generate new bone-forming cells. These cells likely regulate bone formation or participate in bone mass maintenance and repair.

This is the first study that reports the existence of these progenitor cells within the cortical bone that can generate new bone-forming cellsosteoblaststhat can be used to help remodel a bone.

To reach this conclusion, the scientists observed the stem cells within an ex vivo bone transplantation model. These cells migrated out of the transplant and started to reconstruct the bone marrow cavity and form new bone.

However, further study is required to determine the cells potential to regulate bone formation and resorption.

The study is presented in the journal Stem Cells.

Read more:
These new stem cells have the ability to generate new bone - Tech Explorist

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10 things to know about stem cell therapy

New Delhi, March 3 (IANSlife) The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions. Stem cell treatments for brain or neural diseases like Parkinson''s and Alzheimer''s disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way. Vipul Jain, CEO of Advancells and also a Serial entrepreneur, explains in detail the treatment, its uses, cost and effectiveness.

Q: What are stem cells?

Undifferentiated cells that are able to differentiate and transform into any type of cells of the body when and where needed. They have an enormous potential to repair, heal and regenerate. Stem cells come from blood, bone marrow, umbilical cord blood and adipose tissue.

Types of stem cell therapy

Autologous stem cell therapy: Patient receives stem cells from his/her own body

Allogeneic stem cell therapy: Patient receives the stem cells donated by another individual

Autologous stem cell therapy is better than allogeneic stem cell therapy as chances of mismatching are not there and they pose the minimum risk of immune rejection. Also, no side effects or adverse effects are seen as a person''s own blood cells are used. They start the healing process immediately in a natural way.

What is stem cell therapy?

The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. Stem cells can be obtained from the bone marrow, adipose tissues etc. Due to their tremendous potential to prevent and to treat various health conditions and to repair the injured tissues global research investigation is continuously being done as to explore the maximum advantage of these cell lines.

The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions. Stem cell treatments for brain or neural diseases like Parkinson''s and Alzheimer''s disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way.

What are the sources of stem cell?

Depending upon the disease, different stem cell source can be used in a specific condition. The procedure may involve the extraction of stem cells from adipose tissue-derived stem cells with the combination of PRP (Platelet-rich plasma) or can be obtained from bone marrow that can differentiate into progenitor cells that differentiate into various other tissues which can help in the therapy.

Procedure of stem cell therapy

The stem cells are isolated from the bone marrow or adipose tissues followed by their processing and enrichment under sterile conditions. These activated stem cells are placed back into the patient''s body at the target site for repairing the damaged tissue. It is necessary that the stem cells are injected in the specific area of injury as only then the desired results will be achieved.

Adipose stem cells are preferred over bone marrow stem cells as they are easy to isolate and contain a higher number of stem cells.

Stem cells injection

The stem cells injections are gaining much interest because it is devoid of the painful procedure, takes less time in comparison to a surgery, there are no host and recipient rejections as stem cells are harvested from the patient''s body itself and a targeted delivery system is available.

The stem cells obtained are processed in a sophisticated stem cell lab and after activation are inserted back into the host with the help of intravenous, intramuscular, intra-arterial, intradermal and intrathecal injections as per the requirement of the treatment process.

What is the use of anesthetics and why? Usually, local anesthetics are used during a stem cell procedure to numb the area but sometimes general anesthesia is also given while extracting the stem cells from bone marrow. But it is necessary to find out what anesthetic your doctor uses during orthopedic stem cell treatments.

A number of anesthetics have been found to kill the stem cells thus; the treatment''s end result will greatly depend on the use of anesthetics. Some anesthetics very well sync with the stem cell and hence, aid in the treatment.

How good are the processing techniques in the onsite labs?

Stem cells are to be extracted and processed in a clean room, under aseptic conditions maintaining a controlled environment. The doctor should explain the entire process and the number of viable stem cells infused into the patient during the process. Also, the precision of the injections to provide good quality of stem cells at the site of injury will help in better and faster recovery of the patient''s damaged area.

Duration and cost of the therapy

Cost of the treatment and its duration varies from one patient to another. The disease which needs to be cured, the severity, age factor, health condition, etc, define the duration of the therapy. One may respond during the treatment phase itself while the other may show results after a few sessions or weeks. Depending upon the disease diagnosed, the stem cells extracted, duration of the therapy, other adjuvants used in the process, the cost of the stem cell therapy can vary.

Follow-up visits

It is essential that after the stem cell therapy the patient should visit the stem cell doctor for recuperation therapies. The primary goals of such therapy is the prevention of secondary complications, analysis of recovery of motor, sensory and all the bodily functioning, psychological support/counseling for depression, mood swings or anxiety etc. and reintegration into the community.

There can be different sets of precautions which need to be followed at various steps for the recovery of the damaged tissues. The treatment and post treatment conditions may vary from person to person depending upon the disease and the severity.

Success rate of stem cell therapy

Stem cell therapy has shown results in treating serious ailments like leukemia, grafting tissues, autism, orthopedic conditions and skin problems etc. Stem Cell Therapy has been successfully used in the treatment of around 80 serious disorders.

Survival rates among patients who received stem cell treatment are significantly high, whether the cell donors are related or unrelated to them. With the ongoing research around the world, scientists are exploring new possibilities in which a number of life threatening diseases can be prevented and cured hence, the stem cells have proved to be promising in the near future as many aspects are yet to be revealed.

--IANS

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Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

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10 things to know about stem cell therapy - Outlook India

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CAPE CANAVERAL, Fla. SpaceX is preparing for its fifth launch of the year: a resupply mission to the International Space Station (ISS). The mission, which is scheduled to launch Friday (March 6) at 11:50 p.m. EST (0450 GMT on March 7), will bring a bevy of science material to the astronauts living and working in the orbiting laboratory.

This flight, dubbed CRS-20, marks the 20th and final mission for SpaceX under the company's first commercial cargo resupply services contract with NASA. Perched atop a Falcon 9 rocket will sit a cargo Dragon capsule filled with more than 4,300 lbs. (1,950 kilograms) of supplies, including more than 2,100 lbs. (950 kg) of science equipment.

The scientific cargo will support a host of experiments across Expeditions 62 and 63, focusing on a range of topics, from biological sciences (growing human heart cells in space), to water conservation methods, to particle-foam manufacturing and the addition of a new research platform on the ISS.

You can watch SpaceX's Dragon launch livehere on Space.com, courtesy of SpaceX, beginning at about 11:30 p.m. EST (0430 GMT), courtesy of NASA TV. You can alsowatch the launch directly from SpaceX here, beginning at 11:35 p.m. EST (0435 GMT).

Video: What's flying to the space station on SpaceX's CRS-20 mission?Related: SpaceX Dragon cargo ship launching tonight. How to watch live.

In its never-ending quest to create the best athletic shoe, Adidas has turned its sights to the International Space Station. The sportswear company has developed a performance midsole an additional shoe layer between the insole (next to your feet) and the sole (what touches the ground) that will enhance comfort.

To create its midsole, Adidas uses a process called particle foam molding, in which thousands of small pellets are blasted into a mold so they fuse together. To streamline the process and create the best shoe it can, Adidas is going to try this process in microgravity. The experiment, dubbed Adidas BOOST (Boost Orbital Operations on Spheroid Tessellation), will look at how the particles fuse together in space.

By removing gravity from the process, the team can take a closer look at individual pellet motion and location. The results of this investigation could show that the space station is a good platform for testing out new manufacturing methods and could lead to more-efficient means of packing and cushioning materials.

Related: Adidas launching new sneakers inspired by historic NASA spacesuits

Delta Faucet Co., a manufacturer of shower heads and other bathroom hardware, is launching a payload on CRS-20 that will seek to better understand how water droplets form. The company will use that knowledge to build a better shower head that lines up with Delta's ultimate goal: creating the sensation of increased pressure while using less water.

Conserving water is incredibly important, but one of the biggest drawbacks is that eco-friendly, low-flow shower heads do not perform as well as their less environmentally friendly counterparts. Users complain that the water pressure feels so low it's difficult to rinse off properly, which can result in longer showers and, ultimately, more water usage.

To help mitigate this issue, Delta has created a unique shower head, called the H2Okinetic, that controls the size and the speed of the water droplets with the help of an oscillating chip. That chip creates a better shower experience by breaking up the water flow into bigger droplets and shooting them out faster, giving the illusion of more water.

Related: Showering in space: Astronaut home video shows off 'hygiene corner'

"Water is a precious commodity," Garry Marty, principal engineer at Delta Faucet, said during a prelaunch briefing on Thursday (March 5). "We are trying to create a shower head to keep our customers happy while using less water."

He went on to explain that once the water leaves the pipes, it essentially doesn't have any pressure. What you're feeling are the droplets. With this new shower head, Delta Faucet is able to control the size and speed on each drop, revolutionizing the way a shower device delivers a shower.

"Lower-flow showers aren't really great to be under," Marty said. "But the more we understand, the more we can improve."

Marty added that, someday, humanity will be living on the moon or Mars and will need a way to take a shower. The lessons learned from this research go beyond conserving water and user experience, he said; it has implications for the space industry as well. But for now, the bigger concern is to better understand the fundamentals of water droplet formation.

Heart disease is the No. 1 cause of death in the U.S. A team of researchers from Emory University in Atlanta, led by Chunhui Xu, are sending an experiment up to the space station to explore how effectively stem cells can be turned into heart muscle cells.

The data collected could lead to new therapies and even speed up the development of new drugs that can better treat heart disease.

The microgravity environment found on the space station is known to have a profound effect on cell growth. Through this research, the team aims to understand the impact microgravity has on cardiac precursors (cardiac cells created from stem cells) and how effectively they produce cardiac muscle cells, called cardiomyocytes.

Related: Heart cells beat differently in microgravity, may benefit astronauts

Ground-based research shows that when cells are grown under simulated microgravity conditions, the production rate of cardiomyocytes is greater than if they were grown under the effects of gravity. By sending the experiment to the space station, Xu and her team will be able to determine if their results are accurate.

"Our goal is to help make stem cell-based therapy more readily available," Xu said during the briefing. "If successful, the demand for it will be tremendous, because heart disease is the No. 1 killer in America."

In order to have a successful therapy, Xu said that the team will need to produce a large number of high-quality cardiomyocytes. To do that, the researchers need to first understand the mechanisms behind cell transformation.

Bartolomeo is a new research platform that will be installed on the exterior of the space station. Placed outside the European Columbus module, this science balcony will host as many as 12 research experiments at one time.

Built by Airbus, the platform will enable researchers to conduct more experiments on the station's exterior. During a prelaunch briefing, NASA and Airbus explained that Bartolomeos potential uses include Earth observation, robotics, materials science and astrophysics.

"All of your [research] dreams can come true with Bartolomeo," said Andreas Schuette, program manager of Bartolomeo at Airbus.

And parking spots on the washing machine-sized platform are all-inclusive, which means that researchers can pay one price to launch, install, operate and even return to Earth. By working directly with agencies like NASA, ESA, and SpaceX, Airbus is able to offer a cost-effective means of conducting research on the space station.

The company is also working with the United Nations in an effort to entice those who wouldn't otherwise be able to afford to send payloads into space, Schuette told Space.com. The duo have teamed up with the United Nations Office for Outer Space (UNOOSA) to make that happen. (The agency works to make space more accessible.)

If all goes as scheduled, the Dragon will arrive at the International Space Station on Monday (March 9) at approximately 6 a.m. EDT (1000 GMT). From there, NASA astronauts Jessica Meir and Drew Morgan will use the station's Canadarm2 robotic arm to capture and attach the spacecraft, before beginning the unloading process.

Follow Amy Thompson on Twitter @astrogingersnap. Follow us on Twitter @Spacedotcom or Facebook.

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SpaceX Dragon to launch heart cell experiment and more to space station tonight - Space.com

Recommendation and review posted by Bethany Smith

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Recommendation and review posted by Bethany Smith

As with the early medical cannabis plays, a cluster of ASX-listed stocks has wasted little time attaching itself to the c word. Were talking of course about the coronavirus COVID-19 but sadly not another c word: cure.

Or not yet.

According to broker Morgans daily tally, the virulent bug has so far infected 95,332 people, with 38,564 current cases (6,883 of them critical).

Of the remaining 56,768 cases with an outcome, 53,483 recovered and 6,883 achieved a definitive performance indicator.They died.

Okay, a circa 7 per cent mortality rate or even a 1 or 2 per cent rate is nothing to sneeze at, so to speak. But we do wish breathless TV reporters would cease referring to it as the deadly virus, but that would be like asking them to stop referring to a horror smash rather than a sad everyday road accident.

While were on it, we also implore folk to stop hoarding toilet paper: after all, its the coronavirus, not the Caroma-virus.

Named after its crown-like shape but not the Royal Family per se, the common coronavirus is responsible for past pestilences including Severe Acute Respiratory Syndrome (SARS) and Middle Eastern Respiratory Syndrome (MERS).

The virus may indeed fizzle out, as the earlier SARS plague did.

But for the time being, we need the best and brightest minds in the labs to come up with a treatment or more likely a vaccine.

There are some promising developments overseas, which your columnist will return to if he hasnt succumbed as well (he did shake hands with someone who went to a Chinese restaurant a couple of weeks back).

Among the local biotechs and we use the term loosely theres been no lack of endeavour in linking their efforts to the virus.

But to be fair, in some cases investors did it for them.

Take Biotron (ASX:BIT), which was an obvious subject of attention given the company is focused on developing antiviral drugs for HIV and hepatitis.

Biotron also has a program for pan respiratory viruses and mentioned corona in a June 2019 presentation. Some punters latched on to the fact that it wasnt referring to a 1970s Toyota or Mexican beer and the Hot Copper pundits were off and running.

Biotron CEO Dr Michelle Miller has been more circumspect.

Yes, she says, the company has some good advanced compounds to work on, but the reality is that theres nothing that would be ready to fight the current outbreak.

Dr Miller says while the companys work on pan respiratory viruses continues, theres not much to add at this stage.

LISTEN TO: Health Kick Podcast: Coronavirus, HIV and hepatitis are in the sights of Aussie biotech Biotron

Uscom (ASX:UCM) shares went on a run after the company reported increased orders for its haemodynamic monitoring devices in China.

Uscom stands for Ultra-Sonic Cardiac Output Monitors.

The Uscom 1A device is a non-invasive diagnostic that monitors cardiovascular functions, using Doppler ultrasound to detect abnormalities.

Chinese health authorities have recommended Uscom 1A as a monitoring device for severe coronavirus cases, while international guidelines also suggest using the device for paediatric sepsis.

Uscom reported that in the first five weeks of 2019, Chinese sales orders rose 124 per cent, from 17 units to 38 units.

Uscom chief Professor Rob Phillips says the company is well positioned with the virus, but notes that Uscom is not a coronavirus story as such: fatalities from cardiovascular pulmonary failure result from conditions such as pneumonia.

Happily for Uscom, the outbreak comes as the company hones-in on the Chinese market with a new direct sales model.

READ: Dr Borehams Crucible: Uscom gets an A+ for everything but its share price

The molecular diagnostics house has a suite of approved tests that cover gastro-enteric strains, flavivirus/alphavirus, sexually-transmitted diseases and drum roll respiratory pathogens.

Genetic Signatures (ASX:GSS) Easyscreen tests cover pan coronaviruses, which until now has not been able to distinguish COVID-19 from, say, SARS.

But thats all changed, with the company introducing a supplementary test that does just that. Management is fast-tracking a validation program to obtain the data required for international regulatory approvals as rapidly as possible.

However, Genetic Signatures cant be accused of beating up its prospects: management says while the bug presents significant opportunities, the outcome of the emerging pandemic is uncertain.

While the early-stage coronavirus is detected by a blood test, chest x-rays are then used to gauge the severity of the illness and assess fluid in the lungs.

Micro-X (ASX:MX1) is all about developing lightweight and portable x-ray machines for medical applications, as well as other purposes such as defence and airports.

The companys first product, Carestream DRX Revolution Nano is approved in the US and Europe.

In mid-February the company said it had procured orders for $780,000 of machines from governments of two Asian countries, in response to the coronavirus threat. This week, another $1m of orders, all marked for urgent delivery, flooded in.

While these are terrible circumstances with the coronavirus spreading so quickly, we are pleased that our equipment will soon be able to assist medical teams with their responses in affected countries, Micro-X CEO Peter Rowland says.

Why waste a crisis? No fewer than four ASX stocks are capitalising on demand for hand and surface sanitisers to halt the bug in the first place.

Antimicrobial solutions house Zoono Group (ASX:ZNO) proclaims that its impressively-monikered Z-71 Microbe Shield, as used in its hand sanitisers, kills COVID-19 99.99 percent of the time.

Zoono is selling into China via a tie up with Eagle Health (ASX:EHH), which manufactures and distributes product into 26 provinces.

READ: Health: Zoono is not one to waste a crisis as epidemics beef up revenue

Aeris Environmental (ASX:AEI) goes one step better, claiming its Aeris Active product kills influenza and noroviruses in 99.999 percent of cases.

For those remaining 0.001 percent, bad luck and dont buy a lottery ticket.

Interestingly, that announcement did not refer specifically to the coronavirus. But earlier, Aeris announced the Singapore National Environment Agency had listed Aeris Active as one of the general disinfectants effective against the virus.

Meanwhile, fruit juice maker Food Revolution Group (ASX:FOD) has turned from filling its bottles with squeezed oranges to stuffing them with alcohol-based hand sanitiser under the Sanicare brand.

Who would have thought? The swift repositioning results from a 1,260sqm upgrade at the companys plant at Mill Park in outer Melbourne, which enables all sorts of gels, powders, oils and cosmetics to be bottled.

Mainstream sanitiser products such as Dettol and Lysol (made by multinational Reckitt and Benckiser) are flying off the shelves.

But is a good scrub with soap and water just as effective? Australian National University microbiologist Professor Peter Collignon opines theres little difference between hand washing and the alcohol-based sanitisers.

One is just more convenient than the other and contains alcohol, he says. You can put it in your pocket and dont have to be near a sink or basin to use it.

So whos actually tackling the disease? Offshore, theres a conga line of developers having a crack at a vaccine.

In Israel, scientists at the Galilee Research Institute claim to be on the cusp of finalising a product that is capable of getting regulatory assent within 90 days.

Thats what you call fast-track approval.

According to the Jerusalem Post, the same team of scientists has been developing a prophylactic against infectious bronchitis virus, which affects poultry.

The effectiveness of the vaccine has been proven in pre-clinical trials carried out at the countrys Veterinary Institute.

In the US, Gilead Sciences plans to recruit 1,000 patients with coronavirus for a clinical trial to test its experimental anti-viral drug remdesivir (as used to tackle Ebola virus).

With the backing of the World Health Organisation, the drug is also being trialed in China.

Maryland-based, Nasdaq-listed Novavax says it is cloning the coronavirus to develop a vaccine, in the same way it developed one for MERS in 2013.

Novavax is looking at several vaccine candidates for animals and hopes to find one for human testing by the end of May.

Our previous experience working with other coronaviruses, including both MERS and SARS, allowed us to mobilise quickly, Novavax CEO Stanley Eck said.

Fellow Nasdaq minnow Moderna has shipped an experimental vaccine to the National Institute of Allergy and Infectious Diseases for testing.

Backed by billionaire hedge fund founder Jim Simons, Long Island-based private outfit Codagenixexpects to have a vaccine ready for animal testing in four to six weeks, with one suitable for testing about six weeks later.

The Codagenix know-how is based on recoding the genomes of viruses to render them harmless. The technique is not exactly unknown, as its been used to eradicate polio and small pox.

And who can forget Australias very own Relenza anti-influenza Biota, which became Alpharetta Georgias Nabi, changed its name to Aviragen and then was subsumed as a sub-division of San Franciscos Vaxart, popping its head above the parapet to also claim an anti-viral program for COVID-19.

READ: Biotech big guns are buying up the minnows

The South China Morning Post reports that a 65-year-old woman on her COVID-19 deathbed walked out of Chinas Kunming Hospital after being given a stiff shot of mesenchymal stem cells (MSCs).

Two trials are also underway to test the therapy against pneumonia, at a Beijing Military Hospital and Zhongnan Hospital of Wuhan University (yep, in the coronavirus capital).

Could the excitement rub-off on our ASX-listed plays Mesoblast (ASX:MSB), Cynata Therapeutics (ASX:CYP), Orthocell (ASX:OCC) and Regeneus (ASX:RGS)?

Cynatas Dr Ross Macdonald says the reports look authentic; and he believes that MSCs could be an effective adjunct in managing patients with serious issues pertaining to COVID-19.

This is not because MSCs are inherently anti-viral or can act as a vaccine, but more because they have shown benefit in major pathologies associated with infection, he says.

Cynata, we stress, has not mentioned coronavirus in its dispatches and nor has any of the other non-China MSC plays or not yet anyway.

But still, what decent CEO would not give his company a plug?

The clear advantage of (Cynatas) Cymerus technology (is) the ability to make large quantities of consistent, robust MSCs without having to find gazillions of donors, Dr Macdonald says.

Your columnist stresses that the coronavirus influence on the sector is not all positive, with some biotechs likely to be affected by supply or other disruptions.

In mid-February, Cochlear (ASX:COH) quickly stepped off the mark by announcing its earnings for the 2019-20 year were likely to come in at $270-290m, compared with the previously guided $290-300m.

The reason is that hospitals in China and Hong Kong have delayed cochlear implant procedures to avoid the risk of infection.

The aforementioned Uscom notes that with labs preoccupied with the virus, short-term revenues are less predictable. In other words, the coronavirus is a distraction as well as an opportunity.

IDT Australias (ASX:IDT)Dr David Sparling told Biotech Daily that his company had no direct supply chain exposure to China at all, and was doubtful that even the companys gowns and protective gear had much to do with the Middle Kingdom.

If you throw enough money and resources at tackling a disease you will get a result, right?

Er, not quite: cures for well-researched ailments such as Alzheimers disease, multiple sclerosis and an array of cancers remain elusive.

But when youve got an ailment that is crippling the global economy, the imperative to find a solution is somewhat more intensive.

Our best guess is that like SARS and MERS, COVID-19 will hang around for years to come, but the ill-effects will be made more tolerable with an effective vaccine and/or improved immunity over time.

In other words, it will become just another disease in the pantheon of maladies blighting humanity.

In the race for a cure, Gileads Remdesivir looks interesting, given it has been used before.

As for the opportunists in the sanitiser game, the surge in demand means tangible revenue gains and good on them.

But lets be clear: theyre hardly breaking new ground technology-wise and their gains will only be short term as other suppliers enter the market.

As for a cure, or lack of one, we suggest that investors hedge their bets with an exposure to the funeral stocks Invocare (ASX:IVC)and Propel Funeral Partners (ASX:PFP).

After all, theyre the last people to let you down.

Disclosure: Dr Boreham is not a qualified medical practitioner and does not possess a doctorate of any sort. If he doesnt shake hands with you or spare you a square of toilet paper, dont be offended.

This column first appeared in Biotech Daily.

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Dr Borehams Crucible: The small cap biotechs trying to make a buck from coronavirus - Stockhead

Recommendation and review posted by Bethany Smith

Two bills that would affect transgender Idahoans are moving through the Idaho Legislature. KPVI spoke with two transgender women, and a doctor specializing in transgender patients, about the legislation.

Meet Katie Burdick.

She works at a local call center and moved to Pocatello at 18 years-old. Burdick says she first knew she was a girl at four-years-old.

I had this epiphany, says Burdick. I believeclose to my exact words were: Mom. Im a girl.

But while Burdicks mom is super accepting today, at the time, she wasnt.

She wasnt too keen on it then, I was young and it was a very conservative time, says Burdick.

I had two parents who love me, but they didnt understand.

20 year-old Rosetta Roberts had a similar experience.

I dont really talk to my family anymore because I dont have that much acceptance from them, says Roberts. They still mis-gender me. My mom tries to gender me correctly, which I greatly appreciate, but it still hurts and I avoid her.

Roberts is an Idaho State University student double-majoring in computer science and applied mathematics. She hopes to one day be a professor.

She says she didnt realize she was transgender until 17-years-old, when she moved out of her parents home.

Id heard about it [transgender people], but I had the misconception that they were just men wearing dresses at the time.

Even though she didnt know the word for it, she knew something was wrong.

I started having dysphoria around when I started to hit puberty and things just didnt feel right, says Roberts.

I felt like my body was fighting me and that it wasn't going the direction that I wanted.

House Bill 500

January 19, 2015 and April 23, 2018 are significant dates for Burdick and Roberts.

Roberts says April 23 is more important to her than her birthday.

"That's when my life turned around, that's when I started feeling happy," says Roberts.

Thats the date she started hormone treatments to become a woman.

Very quickly I felt a lot better in the head, says Roberts. Its also had visual effects on my appearance, which Ive grown to love. I love my body now. I still sometimes struggle with not loving my body but Im usually pretty confident now.

For Burdick, January 19 was the start of what would become a long medical journey.

Ive always wanted to be as womanly as I can, so at 24, 25, 26, the hormones werent really taking and so I was kind of still mildly depressed, says Burdick. But about a year ago they started to take effect. From an emotional standpoint I was so much happier.

Both women are now living their most authentic lives, but are worried about legislation like House Bill 500 which would ban transgender women and girls from female sports.

"I think it's archaic and almost draconian," says Burdick.

Representative Barbara Ehardt of Idaho Falls is the bill's sponsor.

She says the legislation is about protecting women and girls.

"It's been disheartening to watch through the years this slow but fast infiltration of biological boys and men participating in women's sports," says Ehardt.

Ehardt cant name an active Idaho case of a transgender woman or girl taking advantage of her strength in female sports, but says its only a matter of time before it happens.

She points to an active lawsuit in Connecticut in which three girls are suing two transgender sprinters who have frequently outperformed their cisgender competitors, according to NBCs Connecticut affiliate.

(You can read more about that case here: https://www.nbcconnecticut.com/news/local/girls-sue-to-block-participation-of-transgender-athletes/2222777/)

The NCAA and the Idaho High School Activities Association lets transgender girls and women to play on girls or womens teams if theyve been on testosterone suppression drugs for at least a year.

Ehardt says thats not enough. House Bill 500 sites a 2019 study from the Karolinska Institute which found that "Muscle strength, size and composition following 12 months of gender-affirming treatment in transgender individuals: retained advantage for the transwomen.

Doctor Neil Ragan has specialized in transgender care for seven years, and he now sees around 143 transgender patients from across southeastern Idaho. He pushes back on the Karolinska study sited in House Bill 500, saying thisissue is more nuanced thanhow it's being approached.

In my opinion they're picking and choosing, says Dr. Ragan. And you can find a study that will support any position that you want to take."

What I have observed with my own transgender women on estrogen is that their body strength clearly declines, says Dr. Ragan.

He also points out that theres a broad range of natural strength in both the male and female sex.

Burdick can relate to this.

Even though I was born with a male chromosome the hormones can work wonders, says Burdick. There are cis-gender women who are stronger than me and Im stronger than some cis-gender women.

The bill also includes a section about establishing an athlete's sex if its called into question.

The bill text says:

A student may establish sex by presenting assigned physician's statement that shall indicate the student's sex based solely on:

Dr. Ragan and other transgender advocates worry these examinations could be invasive.

Ehardt calls this a false narrative and misguided. She claims the test would be no more invasive than a routine physical, a cheek swab or a urine test.

But Dr. Ragan says it isnt always that simple.

These exams may not prove anything. There is such a thing as an intersex condition, disorders of sexual development, babies are born and you cant tell initially what sex or what gender they are. Sometimes that is an adrenal issuethere are people who have chromosomal abnormalities, says Dr. Ragan.

"The diversity in human biology is astonishing and I think that when we start trying to legislate who fits into what category, we're going to have difficulty because there will be people who dont fit neatly into either categories."

Ehardt argues that sex cannot be fluid. We are not taking away your ability to identify. You can still identify however you want. But you will have to compete under the category of your biological sex.

Roberts says it's about letting people pursue their dreams.

"I dont want these people to be blocked from their dreams. I myself have dreams if I was blocked from my dreams I would be really really hurt. I don't think transgender people in sports should be blocked."

House Bill 509

Burdick destroyed her childhood photos.

"At first it was like almost anger but then it very quickly went into like a peace and serenity of 'this is gone now'," she says.

Burdicks parents wouldnt allow her to transition, so she was forced to go through male puberty.

"It was just a part of me that I'm like 'I don't even want to look."

Roberts was also forced to grow up in a boy's body.

It made me depressed and I felt more like my body was a vehicle for my head than like a part of me. It felt like puberty was destroying my body.

Roberts can't change her past, but after a 2018 court ruling, she could change the sex on her birth certificate, which saysprotects her frombeing outed.

"One of the big problems with being trans, is that if people find out, a lot of people often become discriminatory," says Roberts.

Blackfoot Rep. Julianne Young sponsored House Bill 509, which would ban changes to sex on birth certificate after a year.

She says this would protect the state's population records.

In a statement Young says:

"Safeguarding the accuracy of our vital records is a vital part of protecting the public health and safety. In addition, many public and private policies and contracts rely on a biologically informed definition of sex.

Dr. Ragan doesnt agree with that argument.

"The sheer number of transgender patients...the number is so small it's not going to significantly skew the demographics."

Many transgender people change theirsex on other documents like drivers licenses or social security documents.

I haveall my insurance cards changed. I have legal name changed. An affidavit from Dr. Ragan saying Im legally female, says Burdick.

Dr. Ragan believes its vital that transgender people have consistent documentation.

"In order for an individual to comfortably navigate this complicated world we live in it's really important that all of their documents align."

Living comfortably sounds good to Roberts.

"I just don't want them to bother us. I want to not have to worry about things like this."

In an email to Rep. Young, Roberts asked her to stop sponsoring the bill. She never heard back.

KPVI also reached out to Rep. Young for further comment, and never got a response.

Looking Forward

Both Roberts and Rosetta say they want to move out of Idaho.

Maybe even the U.S. eventually, says Roberts.

I dont like the atmosphere that the government has towards transgender people.

Roberts has a message for Rep. Young andEhardt:

The bills that theyre sponsoring are very very hurtful to the trans community and I would ask them to stop sponsoring these bills, and to try to help the trans community as much as possible.

Burdick says she wants to live in a bigger city because she feels people are ignorant, especially at the highest levels of government.

Because they have that power, they dont really get stuff. I think they put aside the hoi polloi, the common people.

Like Roberts, Burdick hopes Idaho politicians will start focusing on legislation to help transgender people, like creating more safe spaces for transgender youth.

Try to have more empathy try to listen and understand, she says.

When asked what shes done with support the trans community, Ehardt says shes been working on curbing LGBTQ youth suicide.

Burdick hopes to educate people as much as possible.

I think educating people about transgender issues and rights is a good thing. Yelling back doesnt work, says Burdick.

She wants people to know that Were not cross dressers. Were not drag queens. Were not putting on a show.

Both House Bill 500 and 509 have passed the Idaho House of Representatives.

500 will be discussed in the State Affairs committee Friday. House Bill 509 is also set to be heard in the same committee.

Link:
Transgender in Idaho: a conversation with two trans women on House Bills 500 and 509 - KPVI News 6

Recommendation and review posted by Bethany Smith

All federal limits on testing will be lifted, Pence says.

Vice President Mike Pence said the Centers for Disease Control and Prevention was lifting all restrictions on testing for coronavirus, and would be releasing new guidelines to fast-track testing for people who fear they have the virus, even if they are displaying mild symptoms.

Today we will issue new guidance from the C.D.C. that will make it clear that any American can be tested, no restrictions, subject to doctors orders, Mr. Pence told reporters at the White House.

The federal government has promised to significantly ramp up testing, after drawing criticism for strictly limiting testing in the first weeks of the outbreak. But health care supply companies and public health officials have cast doubt on the governments assurances, as complaints continue that the need for testing remains far greater than the capacity.

The estimates were getting from industry right now by the end of this week, close to a million tests will be able to be performed, the head of the Food and Drug Administration, Dr. Stephen Hahn, said at a White House briefing on Monday.

But some companies developing tests say their products are still weeks away from approval.

And even if a million test kits were available, public health laboratories say they would not be able to process nearly that many within a week. A spokesman for the Department of Health and Human Services said on Monday that public health labs currently can test 15,000 people daily, though that figure is expected to grow.

The F.D.A. said that Dr. Hahn was taking into account the anticipated increased production of test kits by an outside manufacturer, Integrated DNA Technologies, which is now selling kits to the federal government and other buyers.

The C.D.C. botched the first attempt to mass produce a diagnostic kit, a discovery made only after hundreds of kits had been shipped to state laboratories. A promised replacement took several weeks, and still did not permit state and local laboratories to make final diagnoses.

Right now, Id say wed need more capacity, Dr. Hahn said at the White House briefing.

The Department of Veterans Affairs confirmed on Tuesday that a veteran had tested positive for the virus and was transferred to the V.A. hospital in Palo Alto, Calif., presenting the federal government with a new challenge a coronavirus infection in its sprawling veterans health care system.

The patient was diagnosed on Monday and is being cared for in isolation, a V.A. spokeswoman said on Tuesday. It was not clear whether local public health officials had already announced the case; Santa Clara County, Calif., has had several previous coronavirus cases, including two announced on Tuesday without any specifics.

The veterans health system, responsible for caring for more than nine million former service members, has been struggling with an overhaul of its $16 billion medical records system, which has been delayed amid technical and training glitches.

Last week, the U.S. military announced its first active-duty case, an American soldier stationed in South Korea, the country with the second-largest coronavirus outbreak after China. In response, Army officials closed the office buildings the infected soldier had used, called off social events on the base and sent clean teams to disinfect anywhere the soldier had been. A civilian employee at another American military base in South Korea has also tested positive for the virus.

[Read: China pushes back as the coronavirus crisis damages its image.]

President Trump said on Tuesday that he may further tighten limits on international travel in hopes of blocking the arrival of more visitors infected by coronavirus, but he ruled out for now any restrictions on domestic travel within the United States.

Were not looking at that at all. Theres only one hot spot, he told reporters. But were looking at other countries and were being very stringent.

The Trump administration has already imposed limits on travel from China, barred all travel to Iran and issued warnings to Americans not to travel to parts of Italy and South Korea.

Later, at the National Institutes of Health, Mr. Trump added that he was not actively considering restrictions on travel to Mexico but expressed concern about Japan, which is hosting the Summer Olympics in Tokyo and has just constructed a new stadium for the competition.

I dont know what theyre going to do, he said. They have this fabulous facility.

As for Mexico, he played down the prospect of travel limits. Were not looking at it very strongly, he said. Were not seeing a lot of evidence in that area.

Two people who died last week in the Seattle area were infected with coronavirus, officials said on Tuesday, suggesting that the virus had spread in that region days earlier than health officials had previously known.

That brought the death toll in Washington State, and in the United States, to nine. So far, those deaths have all been in the Seattle area.

The confirmation of additional deaths adds to an escalating emergency in a region that has rapidly emerged as a focal point for the virus in the United States, where there have now been at least 120 cases of coronavirus in more than a dozen states, as local health authorities from coast to coast raced to assess the risk to schools, medical centers and businesses.

The other deaths, all announced over the last few days, included residents of a nursing care facility in Kirkland, a Seattle suburb.

Health officials in North Carolina announced that states first case of coronavirus on Tuesday afternoon. They said the patient there had traveled to Washington and been exposed at a long-term care facility where there was an outbreak, an apparent reference to the Life Care nursing center in Kirkland, Wash.

The North Carolina patient was said to be doing well and isolated at home in Wake County.

Officials in Washington State were rushing to take steps to contain the spread. Health officials were asking the State Legislature for an additional $100 million in funding to help respond to the virus. Some leaders were weighing more widespread closings of events, and around Seattle, immediate steps were being taken.

In King County, officials were in the process of purchasing a motel in the region that could house people needing isolation. They were also working to repurpose modular homes that had been originally meant to be used by homeless people.

When the coronavirus first broke out in January, its impact on the travel and hospitality businesses appeared limited to China. But the outbreak has now spread to every continent except Antarctica, and the cities and industries that rely heavily on travel and tourism are bracing for pain.

One city that is anxiously watching the spread of the virus is Las Vegas, which had more than 42.5 million visitors last year, 6.6 million of them to attend business conferences, according to the citys Convention and Visitors Authority.

Some big upcoming conferences have already been canceled, including an annual Adobe Systems event with 20,000 attendees and celebrity speakers like Gwyneth Paltrow and Tom Brady. Adobe said it planned to host portions of the event online.

Major companies like Facebook, Twitter and Intel have said their employees will not attend the popular South by Southwest festival in Austin, Tex., which starts next week, but organizers insisted on Tuesday that the festival would go on.

The New York subway will be disinfected every 72 hours, officials say.

If theres one place where being packed in close quarters with crowds of strangers is unavoidable, it is the New York City subway system well known not just to the locals, but to millions of tourists and anyone who has ever watched a television show or movie set in the city. And that makes it a focus of public worry about contagion.

Officials said on Monday that industrial-grade disinfectants would be used to clean everything from train cars to MetroCard machines every 72 hours. The safety of our customers and employees is our first priority as we continue to monitor the coronavirus, said Patrick T. Warren, chief safety officer at the Metropolitan Transportation Authority.

From Monday evening to midday Tuesday, transit workers disinfected nearly all of the systems 472 subway stations, more than 1,900 subway cars and nearly 2,000 buses, officials said. Transit officials in neighboring New Jersey, which has its own vast commuter rail and bus network, have also ramped up its cleaning regimen.

The Centers for Disease Control and Prevention recommends keeping essential services like public transit in operation, in part so that health care workers and other emergency responders can get to work.

Other major cities experiencing outbreaks have announced similar precautions. In Tehran, public health officials have said they are disinfecting buses at least four times a day and cleaning trains after each trip. In Italy, buses, trains and ferries are also being disinfected regularly.

Although research on the coronavirus is still in the early stages, a 2011 study on a possible influenza outbreak in New York City found that only 4 percent of infections would occur on the subway.

Iran frees 54,000 inmates to avoid spread in prisons.

The number of people in Iran infected by the coronavirus surged past 2,300, the death toll rose to 77, and the countrys head of Parliament said that nearly two dozen lawmakers had tested positive and should avoid meeting with members of the public, the authorities announced on Tuesday.

The new tallies were reported as the judiciary, apparently hoping to minimize the risk of contagion in Irans penal system, said it had temporarily freed more than 54,000 prisoners considered to be symptom-free. But it was unclear from the announcement how many of the prisoners had actually been tested, given the shortage of testing equipment in Iran.

Gholamhossein Esmaili, a judiciary spokesman, who announced the prisoner releases, did not specify whether any of the reported cases or deaths so far have included prisoners.

Iran now has 2,336 reported cases, more than any country other than China and South Korea, and 11 more people have died, according to figures cited by the deputy health minister, Alireza Raisi, on state TV.

State news media reported on Tuesday that Ahmad Toysarkani, an adviser to the judiciary, was among those who had died. The virus has been felt at the highest levels of Iranian society, with Masoumeh Ebtekar, the presidents deputy for womens affairs and the highest-ranking woman in the government, among those who have become infected.

Ayatollah Ali Khamenei, the supreme leader, was pictured in state news media wearing plastic gloves, and he told the Iranian people to follow medical advice to help combat the outbreak, saying anything that facilitates its spread is a sin.

The death toll in Italy surges while the pope has a common cold.

The death toll in Italy jumped to 79 on Tuesday, an increase of 27 deaths in one day, Italian officials said.

Those who had died in the previous 24 hours ranged in age from 55 to 101, said Angelo Borrelli, the head of Italys Civil Protection Agency who is coordinating the countrys response to the crisis.

Most were over 70, and some had pre-existing conditions. But for the others, we still arent sure, he said at a news conference in Rome.

Of the 2,502 infections so far up from the 1,835 announced on Monday 90 percent were located in the northern Italian regions of Lombardy, Veneto and Emilia Romagna, Mr. Borrelli said. Italy has had by far the largest outbreak outside of Asia.

Meanwhile, the Vatican said Tuesday that Pope Francis was ailing with a common cold, and did not have symptoms that could be related to other pathologies.

On Tuesday, France, which has the second highest number of cases in Europe, announced 21 new cases of coronavirus on its soil, raising the total number of cases to 212. A top official at Frances health ministry also announced one new fatality from the virus, a 92-year-old man in the western Morbihan region, bringing the death toll up to four.

Gov. Andrew M. Cuomo announced on Tuesday morning a second confirmed case of the coronavirus in New York, saying that a man in his 50s in Westchester County, just outside of New York City, had tested positive.

The man initially went to a hospital in Westchester about four or five days ago, before it was confirmed he had the virus, the authorities said, acknowledging that he may have exposed doctors, nurses and others to the infection.

On Tuesday, health officials told the mans synagogue, Young Israel of New Rochelle, to call off its religious services for the foreseeable future. Citing guidance from state officials, the county also told congregants who attended Shabbat services on Feb. 22 or went to a funeral or a bat mitzvah there on Feb. 23 to quarantine themselves; the county said it would order quarantines if the affected people did not do so voluntarily.

The patient, whose test was confirmed overnight in New York City, is a lawyer who works in Manhattan and lives in New Rochelle. Mr. Cuomo said the new patient had an underlying respiratory illness and is now at NewYork-Presbyterian Hospital in the city. The mayors office said he was in serious condition.

Though he traveled recently to Miami, he had not traveled to any of the areas that are known hubs of transmission for the virus. For New York, this means the new coronavirus can no longer be thought of as an external threat that has yet to arrive.

Health authorities in New York are now scrambling to trace the chain of transmission in both directions: who infected him, and who he may have infected.

Two of the mans children have links to New York City. One child attends a Jewish high school in Riverdale, which was closed on Tuesday as a precaution. The other, a college student in the city who officials believed lives on campus, has exhibited symptoms associated with the illness.

Both of them have been quarantined and are being tested for coronavirus, according to Mr. de Blasio.

On Sunday, officials announced the states first case, a 39-year-old woman in Manhattan who had been visiting Iran, one of the epicenters of the viruss rapid worldwide spread.

Symptoms of the infection include fever, cough, difficulty breathing or shortness of breath, and gastrointestinal problems or diarrhea.

Serious cases cause lung lesions and pneumonia. But milder cases may resemble the flu or a cold, making detection of the more deadly coronavirus difficult.

Current estimates suggest that symptoms may appear in as few as two days or as many as 14 days after exposure.

If you think youre sick, stay home, except to get medical care. If you have symptoms, the C.D.C recommends that you call a medical professional if you have recently been in an area with a coronavirus outbreak, or have had close contact with someone who has been to such an area.

Fed rate cut fails to quell investor fears of a coronavirus hit to the economy.

The Federal Reserve delivered the emergency rate cut investors had been clamoring for at 10 a.m. on Tuesday. The market rally that followed lasted about 15 minutes.

By the end of trading on Tuesday, stocks were sharply lower and bond yields had plummeted to previously unthinkable lows, suggesting that investors think theres little the Federal Reserve can do to keep the coronavirus from hammering the U.S. economy.

The S&P 500 fell about 2.8 percent, undoing much of Mondays 4.6 percent surge. The yield on 10-year Treasury notes dropped below 1 percent.

Both moves suggest investors see growing threats to the outlook for economic growth and corporate profits over the next 10 months.

Economists around the globe sharply downgraded their economic growth expectations for the year. JPMorgan Chase economists and market analysts estimated that investors are pricing in a 90 percent chance of recession, according to a research note published Tuesday afternoon.

India curbs medicine exports, affecting supplies worldwide.

In response to the epidemic, the government of India on Tuesday curbed exports of 26 drugs and drug ingredients, including a wide range of antibiotics and certain vitamins, meaning that the new coronavirus could soon have ripple effects on people worldwide with other ailments.

The world relies heavily on Indias huge pharmaceutical industry for generic drugs, but Indian officials are concerned about safeguarding the supply for their own country.

Indian drug makers depend on Chinese factories for key drug ingredients, particularly for antibiotics and vitamins. The Covid-19 outbreak in China and quarantine of Hubei province, where much of the production is centered, has severely hampered Chinas ability to produce those ingredients.

Although Indian companies had stockpiled extra ingredients ahead of the annual Lunar New Year holiday, those reserves are now running low.

Its unclear whether Indias exports of the specified drugs will be completely stopped. The governments order said that exports of the drugs are restricted, which means a government permit is required before they can be shipped out of the country.

The antibiotics facing export limits are tinidazole, metronidazole, chloramphenicol, erythromycin salts, neomycin, clindamycin salts and ornidazole.

Other drugs on the restricted list include the painkiller acetaminophen, the hormone progesterone, the antiviral drug aciclovir, and the vitamins B1, B6 and B12.

Cruise ship passengers in the U.S. being released from quarantine.

Many of the more than 120 American evacuees from the Diamond Princess cruise ship in Japan were set to be released from quarantine on Tuesday from a military base in Texas, after state and local officials said the C.D.C. modified its release protocol in response to their concerns.

The former passengers have been at Lackland Air Force Base in San Antonio for roughly two weeks since being evacuated from the cruise ship. Those being released have tested negative for the virus and have not shown symptoms while in quarantine.

They were scheduled to go home on Monday, but their release was delayed at the urging of San Antonio and Texas state officials. The city tried and failed to obtain a federal court order barring the C.D.C. from releasing more people.

None of those released on Tuesday will stay at local hotels, city officials said, but instead will be taken on buses from the air base to the airport.

Quarantined people who have been infected must be symptom-free and have two negative test results within 24 hours are eligible for release.

Reporting and research were contributed by Jennifer Steinhauer, Katie Rogers, Christina Goldbaum, Reed Abelson, Sarah Kliff, Azi Paybarah, Jesse McKinley, Katie Thomas, Knvul Sheikh, Choe Sang-Hun, Peter Baker, Benjamin Mueller, Marc Santora, Joseph Goldstein, Michael Gold, Luis Ferre-Sadurni, Eric Schmitt, Helene Cooper, Roni Caryn Rabin, Russell Goldman, Paul Mozur, Raymond Zhong, Noah Weiland, Emily Cochrane, Aaron Krolik, Claire Fu, Elaine Yu, Elisabetta Povoledo, Vindu Goel, Manny Fernandez, Mitch Smith, Patrick J. Lyons, Richard Prez-Pea, Dagny Salas, Iliana Magra and Constant Meheut.

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U.S. Will Drop Limits on Virus Testing, Pence Says - The New York Times

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CRANBURY, N.J. and PHILADELPHIA, March 05, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics, Inc. (Nasdaq: FOLD) today announced the official opening of the companys Global Research and Gene Therapy Center of Excellence in uCity SquareinPhiladelphia to advance its industry leading portfolio of rare disease gene therapy programs. In 2019, Amicus and the University of Pennsylvania (Penn) announced a major expansion of their Gene Therapy Collaboration which provides Amicus with disease-specific worldwide rights to Penns Next Generation Gene Therapy Technologies from the Wilson Lab for the majority of lysosomal storage disorders, as well as twelve additional more prevalent rare diseases including Rett Syndrome, Angelman Syndrome and select other muscular dystrophies.

John F. Crowley, Chairman and Chief Executive Officer ofAmicus Therapeutics, Inc., stated, This is a remarkable advancement in the history of Amicus and further strengthens our great collaboration with Dr. Jim Wilson and the Gene Therapy Center at Penn. Philadelphia is a magnet for talent in gene therapy and an engine for innovation. This new global research center located in the cradle of liberty will become part of the cradle of cures as we move many gene therapy programs forward toward patients in need. With exclusive global rights to 50 rare diseases in collaboration with Dr. Wilsons team we hope to be able to alleviate an enormous amount of human suffering with the great science work that will be done in this new facility.

The 75,000 sq. ft. Center is located on the top three floors of the new building at 3675 Market Street and consists of office and state-of-the-art laboratories. It will ultimately house approximately 200 researchers and drug developers focused exclusively on gene therapies.

A by invitation only ribbon cutting event takes place today to celebrate the opening with special guests to include Dr. Jim Wilson, government officials and patients living with rare diseases and their families.

About Amicus TherapeuticsAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com and follow on Twitter and LinkedIn.

CONTACTS:

Media:Christopher ByrneExecutive Director, Corporate Communicationscbyrne@amicusrx.com(609) 662-2798

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Amicus Opens New Global Research and Gene Therapy Center of Excellence in Philadelphia - BioSpace

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Scientists at the Casey Eye Institute, in Portland, Ore., have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness. KTSDesign/Science Photo Library/Getty Images hide caption

Scientists at the Casey Eye Institute, in Portland, Ore., have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness.

For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body.

The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored.

"We're really excited about this," says Dr. Eric Pierce, a professor of ophthalmology at Harvard Medical School and director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear. Pierce is leading a study that the procedure launched.

"We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine," Pierce tells NPR.

The CRISPR gene-editing technique has been revolutionizing scientific research by making it much easier to rewrite the genetic code. It's also raising high hopes of curing many diseases.

Before this step, doctors had only used CRISPR to try to treat a small number of patients who have cancer, or the rare blood disorders sickle cell anemia or beta-thalassemia. While some of the initial results have been promising, it's still too soon to know whether the strategy is working.

In those other cases, doctors removed cells from patients' bodies, edited genes in the cells with CRISPR in the lab and then infused the modified cells back into the volunteers' bodies to either attack their cancer or produce a protein their bodies are missing.

In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery.

Beginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement.

"The majority of people affected by this disease have the most severe end of the spectrum, in terms of how poor their vision is," Pierce says. "They're functionally blind."

The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells enabling patients to regain at least some vision.

"It's the first time the CRISPR gene-editing is used directly in a patient," Pierce says. "We're really optimistic that this has a good chance of being effective."

The study is being sponsored by Editas Medicine, of Cambridge, Mass., and Allergan, based in Dublin. It will eventually involve a total of 18 patients, including some as young as ages 3 to 17, who will receive three different doses.

"We're very excited about this. This is the first time we're doing editing inside the body," says Charles Albright, the chief scientific officer at Editas.

"We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way," Albright says.

Francis Collins, director of the National Institutes of Health, calls the advance "a significant moment."

"All of us dream that a time might be coming where we could apply this approach for thousands of diseases," Collins tells NPR. "This is the first time that's being tried in a human being. And it gives us hope that we could extend that to lots of other diseases if it works and if it's safe."

Pierce, Albright and others stressed that only one patient has been treated so far and that the study, still at a very early stage, is designed primarily to determine whether injecting the gene-editing tool directly into the eye is safe.

To that end, the researchers are starting with lowest dose and the oldest patients, who have already suffered extensive damage to their vision. And doctors are only treating one eye in each patient. All of those steps are being taken in case the treatment somehow backfires, causing more damage instead of being helpful.

"CRISPR has never been used directly inside a patient before," Pierce says. "We want to make sure we're doing it right."

Still, he says, if the underlying defect can be repaired in this patient and others with advanced damage, "we have the potential to restore vision to people who never had normal vision before. It would indeed be amazing."

The study involves a form of Leber congenital amaurosis known as Type 10, which is caused by a defect in the CEP290 gene.

If the approach appears to be safe and effective, the researchers will start treating younger patients.

"We believe children have the potential to have the most benefit from their therapy, because we know their visual pathways are still intact," Albright explains.

The procedure, which takes about an hour to perform, involves making tiny incisions that enable access to the back of the eye. That allows a surgeon to inject three droplets of fluid containing billions of copies of the virus that has been engineered to carry the CRISPR gene-editing instructions under the retina.

The idea is that once there, the CRISPR editing elements would snip out the mutation that causes a defect in CEP290. The hope is that this would be a one-time treatment that would correct vision for a lifetime.

If it works, the volunteers in the study might be able to have the procedure repeated on the other eye later.

"If we can do this safely, that opens the possibility to treat many other diseases where it's not possible to remove the cells from the body and do the treatment outside," Pierce says.

The list of such conditions might include some brain disorders, such Huntington's disease and inherited forms of dementia, as well as muscle diseases, such as muscular dystrophy and myotonic dystrophy, according to Pierce and Albright.

"Inherited retinal diseases are a good choice in terms of gene-based therapies," says Artur Cideciyan, a professor of ophthalmology at the University of Pennsylvania, given that the retina is easily accessible.

But Cideciyan cautions that other approaches for these conditions are also showing promise, and it remains unclear which will turn out to be the best.

"The gene-editing approach is hypothesized to be a 'forever fix,' " he says. "However, that's not known. And the data will have to be evaluated to see the durability of that. We'll have to see what happens."

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CRISPR Used To Edit Genes Inside A Patient With A Rare Form Of Blindness : Shots - Health News - NPR

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GRENOBLE, France and RESEARCH TRIANGLE PARK, N.C., March 02, 2020 (GLOBE NEWSWIRE) -- CYTOO, a leading drug discovery company on muscle disorders, today announced that it has entered into a research collaboration aimed at selecting a gene therapy candidate for a rare muscle disorder withAsklepios BioPharmaceutical, Inc.(AskBio), a clinical-stage, fully integrated adeno-associated virus (AAV) gene therapy company. Under the terms of the agreement, AskBio and CYTOO will work together to develop an AAV-based screening platform derived from patient cells. The goal of the collaboration is to screen and select a preclinical candidate capable of restoring a healthy phenotype from patient cells culturedin vitro.

CYTOO has developed a muscle-on-a-plate platform using patients primary cells, called MyoScreen. MyoScreen is anin vitrosystem in which skeletal muscle cells mimic the physiology, contractile and metabolic functions of human musclein vivoand allow infection by AAV-based gene therapy vectors targeting muscle.

Dr. Philippe Moullier, Chief Scientific Officer, AskBio Europe, said, As a leader in the gene therapy space, the ability to quickly and efficiently screen potential therapeutic candidates will be invaluable. The expertise provided by CYTOO and the MyoScreen platform will potentially give us a better understanding of how those candidates perform in patient cells and improve efficiency throughout the R&D process.

Luc Selig, CYTOOs CEO, added, Gene therapy for muscle disorders is becoming a reality for patients and their families, and we are proud that AskBio has chosen our expertise to investigate a potential new treatment. We have developed MyoScreen as a laboratory model of patient-derived muscle that can be used to screen gene therapy candidates and QC clinical and commercial batches.

The financial terms of the agreement were not disclosed.

About CYTOOCYTOO is a preclinical-stage drug discovery company addressing muscular disorders (NMDs, muscle waste, muscle disuse, metabolic ageing). The company has developed MyoScreen, a versatile and high-throughput muscle-on-a-plate R&D platform, from patient-derived myotubes, that can be used to model any muscle disorder and screen any type of therapeutic candidate. The platform is open to partnering with biotech and pharmaceutical companies and has been the starting point of CYTOOs internal drug discovery program on Duchenne Muscular Dystrophy. Among partners of CYTOO: Daiichi Sankyo, Pfizer, Axcella. CYTOO has offices in Grenoble, France and Bethesda, MD, USA.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy type 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more atwww.askbio.comor follow us onLinkedIn.

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Gene therapy has been used to treat a person with haemophilia for the first time in Ireland, a patient group has announced.

The Irish Haemophilia Society (IHS) confirmed on Thursday morning that the person received gene therapy as part of a clinical trial. IHS chief executive Brian OMahony said the treatment is a momentous occasion for the haemophilia community in Ireland.

The general term haemophilia describes a group of inherited blood disorders in which there is a life-long defect in the clotting mechanism of the blood.

Since the 1970s, haemophilia has been treated by the administration of intravenous infusions of the missing clotting factor. However, work done by companies and academic institutions has given new hope that an effective treatment could be based on gene therapy, the IHS said.

The clinical trial uses a viral vector to deliver gene therapy to the persons liver intravenously. In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood. It is ironic that a virus could now be the delivery system which offers the best hope of a practical cure for severe haemophilia, Mr OMahony said.

It is hoped that the effect of the gene therapy infusion will last for many years and possibly for a lifetime.

The principal investigator on the trial in Ireland is Dr Niamh OConnell of the National Coagulation Centre in St. Jamess Hospital. She said the gene therapy was ground breaking.

The opportunity to participate in clinical trials is part of the commitment of the National Haemophilia Service to personalise treatment and to improve the quality of life and outcomes for people with haemophilia.

The study, which is being run by drug manufacturer UniQure, involves three Irish patients among a total of 60 around the world. There will be an intensive period of monitoring of effectiveness at first, followed by a longer term evaluation over five years. Only one treatment is administered to trial patients.

The particular gene therapy is focused on patients who are missing clotting factor IX, the second most-common type of haemophilia. Earlier results show that the level of clotting factor increased from 1 per cent - generally seen as severe haemophilia - to between 33 and 51 per cent in a small number of individuals treated, levels seen in mild cases or even amongst the non-haemophiliac population.

Professor Martina Hennessy of the Wellcome HRB Clinicial Research facility in St Jamess, where the gene therapy was infused, said that access to high quality research is an integral part of good healthcare because it raises standards and pushes the boundaries of what can be achieved.

Delivering gene therapy requires specialised training and equipment, we have been preparing with Dr OConnell and her team for over a year to undertake this exciting work, in partnership with the Irish Haemophilia Society. Other trials are planned, we hope this expertise leads other Irish patient groups also being able to access potentially life changing treatments in the future, she said.

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Gene therapy used in clinical trial for person with haemophilia - The Irish Times

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An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winning journalist imagined 2020 would look like when she reported on the Human Genome Project back in 1996.

The Human Genome Project was an international scientific collaboration that successfully mapped, sequenced and made publicly available the genetic content of human chromosomes or all human DNA. Taking place between 1990 and 2003, the project caused many to speculate about the future of medicine.

In 1996, Walter Gilbert, a Nobel laureate, said, The results of the Human Genome Project will produce a tremendous shift in the way we can do medicine and attack problems of human disease. In 2000, Francis Collins, then head of the HGP at the National Institutes of Health, predicted, Perhaps in another 15 or 20 years, you will see a complete transformation in therapeutic medicine. The same year, President Bill Clinton stated the Human Genome Project would revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.

It is now 2020 and no one carries a genome card. Physicians typically do not examine your DNA to diagnose or treat you. Why not? As I explain in a recent article in the Journal of Neurogenetics, the causes of common debilitating diseases are complex, so they typically are not amenable to simple genetic treatments, despite the hope and hype to the contrary.

The idea that a single gene can cause common diseases has been around for several decades. In the late 1980s and early 1990s, high-profile scientific journals, including Nature and JAMA, announced single-gene causation of bipolar disorder, schizophrenia and alcoholism, among other conditions and behaviors. These articles drew massive attention in the popular media, but were soon retracted or failed attempts at replication. These reevaluations completely undermined the initial conclusions, which often had relied on misguided statistical tests. Biologists were generally aware of these developments, though the follow-up studies received little attention in popular media.

There are indeed individual gene mutations that cause devastating disorders, such as Huntingtons disease. But most common debilitating diseases are not caused by a mutation of a single gene. This is because people who have a debilitating genetic disease, on average, do not survive long enough to have numerous healthy children. In other words, there is strong evolutionary pressure against such mutations. Huntingtons disease is an exception that endures because it typically does not produce symptoms until a patient is beyond their reproductive years. Although new mutations for many other disabling conditions occur by chance, they dont become frequent in the population.

Instead, most common debilitating diseases are caused by combinations of mutations in many genes, each having a very small effect. They interact with one another and with environmental factors, modifying the production of proteins from genes. The many kinds of microbes that live within the human body can play a role, too.

Since common serious diseases are rarely caused by single-gene mutations, they cannot be cured by replacing the mutated gene with a normal copy, the premise for gene therapy. Gene therapy has gradually progressed in research along a very bumpy path, which has included accidentally causing leukemia and at least one death, but doctors recently have been successful treating some rare diseases in which a single-gene mutation has had a large effect. Gene therapy for rare single-gene disorders is likely to succeed, but must be tailored to each individual condition. The enormous cost and the relatively small number of patients who can be helped by such a treatment may create insurmountable financial barriers in these cases. For many diseases, gene therapy may never be useful.

The Human Genome Project has had an enormous impact on almost every field of biological research, by spurring technical advances that facilitate fast, precise and relatively inexpensive sequencing and manipulation of DNA. But these advances in research methods have not led to dramatic improvements in treatment of common debilitating diseases.

Although you cannot bring your genome card to your next doctors appointment, perhaps you can bring a more nuanced understanding of the relationship between genes and disease. A more accurate understanding of disease causation may insulate patients against unrealistic stories and false promises.This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Why Sequencing the Human Genome Failed to Produce Big Breakthroughs in Disease - Discover Magazine

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Lets take inventory on the therapies that are being developed for the coronavirus epidemic. Here is a very thorough listof at Biocentury, and I should note that (like Stat and several other organizations) theyre making all their Covid-19 content free to all readers during this crisis. Id like to zoom in today on the potential small-molecule therapies, since some of these have the most immediate prospects for use in the real world.

The ones at the front of the line are repurposed drugs that are already approved for human use, for a lot of obvious reasons. The Biocentury list doesnt cover these, but heres an article at Nature Biotechnology that goes into detail. Clinical trials are a huge time sink they sort of have to be, in most cases, if theyre going to be any good and if youve already done all that stuff its a huge leg up, even if the drug itself is not exactly a perfect fit for the disease. So what do we have? The compound that is most advanced is probably remdesivir from Gilead, at right. This has been in development for a few years as an RNA virus therapy it was originally developed for Ebola, and has been tried out against a whole list of single-strand RNA viruses. That includes the related coronaviruses SARS and MERS, so Covid-19 was an obvious fit.

The compound is a prodrug that phosphoramide gets cleaved off completely, leaving the active 5-OH compound GS-44-1524. It mechanism of action is to get incorporated into viral RNA, since its taken up by RNA polymerase and it largely seems to evade proofreading. This causes RNA termination trouble later on, since that alpha-nitrile C-nucleoside is not exactly what the virus is expecting in its genome at that point, and thus viral replication is inhibited.

There are five clinical trials underway (heres an overview at Biocentury). The NIH has an adaptive-design Phase II trial that has already started in Nebraska, with doses to be changed according to Bayesian readouts along the way. There are two Phase III trials underway at China-Japan Friendship Hospital in Hubei, double-blinded and placebo-controlled (since placebo is, as far as drug therapy goes, the current standard of care). And Gilead themselves are starting two open-label trials, one with no control arm and one with an (unblinded) standard-of-care comparison arm. Those might read out first, depending on when they get off the ground, but will be only rough readouts due to the fast-and-loose trial design. The two Hubei trials and the NIH one will add some rigor to the process, but Im not sure when theyre going to report. My personal opinion is that I like the chances of this drug more than anything else on this list, but its still unlikely to be a game-changer.

Theres an RNA polymerase inhibitor (favipiravir) from Toyama, at right, thats in a trial in China. Its a thought a broad-spectrum agent of this sort would be the sort of thing to try. But unfortunately, from what I can see, it has already turned up as ineffective in in vitro tests. The human trial thats underway is honestly the sort of thing that would only happen under circumstances like the present: a developing epidemic with a new pathogen and no real standard of care. I hold out little hope for this one, but given that theres nothing else at present, it probably should be tried. As youll see, this is far from the only situation like this.

One of the screens of known drugs in China that also flagged remdesivir noted that the old antimalarial drug chloroquine seemed to be effective in vitro. It had been reported some years back as a possible antiviral, working through more than one mechanism, probably both at viral entry and intracellularly thereafter. That part shouldnt be surprising chloroquines actual mode(s) of action against malaria parasites are still not completely worked out, either, and some of what people thought they knew about it has turned out to be wrong. There are several trials underway with it at Chinese facilities, some in combination with other agents like remdesivir. Chloroquine has of course been taken for many decades as an antimalarial, but it has a number of liabilities, including seizures, hearing damage, retinopathy and sudden effects on blood glucose. So its going to be important to establish just how effective it is and what doses will be needed. Just as with vaccine candidates, its possible to do more harm with a rushed treatment than the disease is doing itself

There are several other known antiviral drugs are being tried in China, but I dont have too much hope for those, either. The neuraminidase inhibitors such as oseltamivir (better known as Tamiflu) were tried against SARS and were ineffective; there is no reason to expect anything versus Covid-19 although these drugs are a component of some drug cocktail trials. The HIV protease therapies such as darunavir and the combination therapy Kaletra are in trials, but thats also a rather desperate long shot, since theres no particular reason to think that they will have any such protease inhibition against what this new virus has to offer (and indeed, such agents werent much help against SARS in the end, either). The classic interferon/ribavirin combination seems to have had some activity against SARS and MERS, and is in two trialsfrom what I can see. Thats not an awful idea by any means, but its not a great one, either: if your viral disease has interferon/ribavirin as a front line therapy, it generally means that theres nothing really good available. No, unless we get really lucky none of these ideas are going to slow the disease down much.

There are a few other repurposed-protease-inhibitors ideas out there, such as this one. (Edit: I had seen this paper but couldnt track it down, so thanks to those who sent it along). This paper suggests that the TMPRSS2 protease is important for viral entry on the human-cell-side of the process, a pathway that has been noted for other coronaviruses. And it points out that there is a an approved inhibitor (in Japan) for this enzyme (camostat), so that would definitely seem to be worth a trial, probably in combination with remdesivir.

Thats about it for the existing small molecules, from what I can see. What about new ones? Dont hold your breath, is all I can say. A drug discovery program from scratch against a new pathogen is, as many readers here well know, not a trivial exercise. As this Bloomberg article details, many such efforts in the past (small molecules and vaccines alike) have come to grief because by the time they had anything to deliver the epidemic itself had passed. Indeed, Gileads remdesivir had already been dropped as a potential Ebola therapy.

You will either need to have a target in mind up front or go phenotypic. For the former, what youd see are better characterizations of the viral protease and more extensive screens against it. Two other big target areas are viral entry (which involves the spike proteins on the virus surface and the ACE2 protein on human cells) and viral replication. To the former, its worth quickly noting that ACE2 is so much unlike the more familiar ACE protein that none of the cardiovascular ACE inhibitors do anything to it at all. And targeting the latter mechanisms is how remdesivir was developed as a possible Ebola agent, but as you can see, that took time, too. Phenotypic screens are perfectly reasonable against viral pathogens as well, but youll need to put time and effort into that assay up front, just as with any phenotypic effort, because as anyone who does that sort of work will tell you, a bad phenotypic screen is a complete waste of everyones time.

One of the key steps for either route is identifying an animal model. While animal models of infectious disease can be extremely well translated to human therapy, that doesnt happen by accident: you need to choose the right animal. Viruses in general (and coronaviruses are no exception) vary widely in their effects in different species, and not just across the gaps of bird/reptile/human and the like. No, youll run into things where even the usual set of small mammals are acting differently from each other, with some of them not even getting sick at all. This current virus may well have gone through a couple of other mammalian species before landing on us, but youll note that dogs (to pick one) dont seem to have any problem with it.

All this means that any new-target new-chemical-matter effort against Covid-19 (or any new pathogen) is going to take years, and there is just no way around that. This puts small molecules in a very bimodal distribution: you have the existing drugs that might be repurposed, and are presumably available right now. Nothing else is! At the other end, for completely new therapies you have the usual prospects of drug discovery: years from now, lots of money, low success rate, good luck to all of us. The gap between these two could in theory be filled by vaccines and antibody therapies (if everything goes really, really well) but those are very much their own area and will be dealt with in a separate post.

Either way, the odds are that we (and I mean we as a species here) are going to be fighting this epidemic without any particularly amazing pharmacological weapons. Eventually well have some, but I would advise people, pundits, and politicians not to get all excited about the prospects for some new therapies to come riding up over the hill to help us out. The odds of that happening in time to do anything about the current outbreak are very small. We will be going for months, years, with the therapeutic options we have right now. Look around you: what we have today is what we have to work with.

See the original post here:
Covid-19 Small Molecule Therapies Reviewed - Science Magazine

Recommendation and review posted by Bethany Smith