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‘Origami Organs’ Can Potentially Regenerate Tissues – Technology Networks

Northwestern Medicine scientists and engineers have invented a range of bioactive tissue papers made of materials derived from organs that are thin and flexible enough to even fold into an origami bird. The new biomaterials can potentially be used to support natural hormone production in young cancer patients and aid wound healing.

The tissue papers are made from structural proteins excreted by cells that give organs their form and structure. The proteins are combined with a polymer to make the material pliable.

In the study, individual types of tissue papers were made from ovarian, uterine, kidney, liver, muscle or heart proteins obtained by processing pig and cow organs. Each tissue paper had specific cellular properties of the organ from which it was made.

This new class of biomaterials has potential for tissue engineering and regenerative medicine as well as drug discovery and therapeutics, corresponding author Ramille Shah said. Its versatile and surgically friendly.

Shah is an assistant professor of surgery at the Feinberg School of Medicine and an assistant professor of materials science and engineering at McCormick School of Engineering. She also is a member of the Simpson Querrey Institute for BioNanotechnology.

For wound healing, Shah thinks the tissue paper could provide support and the cell signaling needed to help regenerate tissue to prevent scarring and accelerate healing.

The tissue papers are made from natural organs or tissues. The cells are removed, leaving the natural structural proteins known as the extracellular matrix that then are dried into a powder and processed into the tissue papers. Each type of paper contains residual biochemicals and protein architecture from its original organ that can stimulate cells to behave in a certain way.

In the lab of reproductive scientist Teresa Woodruff, the tissue paper made from a bovine ovary was used to grow ovarian follicles when they were cultured in vitro. The follicles (eggs and hormone-producing cells) grown on the tissue paper produced hormones necessary for proper function and maturation.

This could provide another option to restore normal hormone function to young cancer patients who often lose their hormone function as a result of chemotherapy and radiation, Woodruff, a study coauthor, said.

A strip of the ovarian paper with the follicles could be implanted under the arm to restore hormone production for cancer patients or even women in menopause.

Woodruff is the director of the Oncofertility Consortium and the Thomas J. Watkins Memorial Professor of Obstetrics and Gynecology at Feinberg.

In addition, the tissue paper made from various organs separately supported the growth of adult human stem cells. Scientists placed human bone marrow stem cells on the tissue paper, and all the stem cells attached and multiplied over four weeks.

“Thats a good sign that the paper supports human stem cell growth, said first author Adam Jakus, who developed the tissue papers. Its an indicator that once we start using tissue paper in animal models it will be biocompatible.

The tissue papers feel and behave much like standard office paper when they are dry, Jakus said. Jakus simply stacks them in a refrigerator or a freezer. He even playfully folded them into an origami bird.

Even when wet, the tissue papers maintain their mechanical properties and can be rolled, folded, cut and sutured to tissue, he said.

Jakus was a Hartwell postdoctoral fellow in Shahs lab for the study and is now chief technology officer and cofounder of the startup company Dimension Inx, LLC, which was also cofounded by Shah. The company will develop, produce and sell 3-D printable materials primarily for medical applications. The Intellectual Property is owned by Northwestern University and will be licensed to Dimension Inx.

An Accidental Spill Sparked Invention

An accidental spill of 3-D printing ink in Shahs lab by Jakus sparked the invention of the tissue paper. Jakus was attempting to make a 3-D printable ovary ink similar to the other 3-D printable materials he previously developed to repair and regenerate bone, muscle and nerve tissue. When he went to wipe up the spill, the ovary ink had already formed a dry sheet.

When I tried to pick it up, it felt strong, Jakus said. I knew right then I could make large amounts of bioactive materials from other organs. The light bulb went on in my head. I could do this with other organs.

It is really amazing that meat and animal by-products like a kidney, liver, heart and uterus can be transformed into paper-like biomaterials that can potentially regenerate and restore function to tissues and organs, Jakus said. Ill never look at a steak or pork tenderloin the same way again.

Monica Laronda, who was a postdoctoral fellow in Woodruffs lab during the study, also is a coauthor. She is now an assistant professor of pediatrics at Feinberg and a researcher at the Stanley Manne Childrens Research Institute, Ann & Robert H Lurie Children’s Hospital of Chicago. Laronda and Woodruff also are members of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

The research was supported by grant P50 HD076188-02 from the Center for Reproductive Health After Disease of the National Centers for Translational Research in Reproduction and Infertility, Google and the Hartwell Foundation.

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

Reference:

Jakus, A. E., Laronda, M. M., Rashedi, A. S., Robinson, C. M., Lee, C., Jordan, S. W., . . . Shah, R. N. (2017). Tissue Papers from Organ-Specific Decellularized Extracellular Matrices. Advanced Functional Materials, 1700992. doi:10.1002/adfm.201700992

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‘Origami Organs’ Can Potentially Regenerate Tissues – Technology Networks

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Stem-cell treatment may harm heart disease patients – ISRAEL21c

For patients with severe and end-stage heart failure there are few treatment options left apart from transplants and stem-cell therapy. But a new Israeli study finds that stem-cell therapy may harm heart-disease patients.

The research, led by Prof. Jonathan Leor of Tel Aviv Universitys Sackler Faculty of Medicineand Sheba Medical Center and conducted by TAUs Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissue and contends that this can prove toxic for patients.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, said Leor. Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

Tissue or adult stem cells blank cells that can act as a repair kit for the body by replacing damaged tissue encourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, many heart-failure patients have turned to stem-cell therapy as a last resort.

But our findings suggest that stem cells, like any drug, can have adverse effects, said Leor. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

The researchers, who published their study in the journal Circulation, also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. Afterward, they focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cells those drawn from the patients themselves in cardiac therapy, Leor said.

We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells, said Leor. Our findings determine the potential negative effects of inflammation on stem-cell function as theyre currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions.

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. We hope our engineered stem cells will be resistant to the negative effects of the immune system, said Leor.

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Stem-cell treatment may harm heart disease patients – ISRAEL21c

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Techshot system headed to space – Evening News and Tribune

GREENVILLE Onboard the next SpaceX cargo spacecraft launching to the International Space Station (ISS) from Pad 39A at the Kennedy Space Center will be a commercial research system owned and operated by Techshot Inc. The equipment will conduct regenerative medicine experiments onboard the station before returning to Earth in the same capsule for a splashdown off the coast of Southern California approximately 30 days later.

Techshots ADvanced Space Experiment Processor (ADSEP) is a device approximately the size of a microwave oven that contains three separate modules, each of which simultaneously can process experiments in three separate on-orbit replaceable automated mini-laboratory cassettes. Two of the three cassettes on the mission will conduct research for a team led by Robert Schwartz, Ph.D., from the University of Houston.

Funded by the Center for the Advancement of Science in Space (CASIS), the study will evaluate a new approach to growing human tissue for transplant. The microgravity environment onboard the ISS could improve cell growth and development and 3D tissue formation, enabling discoveries that will advance translational disease treatments. Previous studies on Earth by Schwartz and his collaborators at the Texas Heart Institute and the Baylor College of Medicine have found that low gravity environments help specially programmed stem cells move toward becoming new heart muscle cells, which may be used to repair damaged hearts on Earth.

The third cassette contains an experiment conducted by and for Techshot itself. The company is developing a 3D bioprinter for the ISS known as the Techshot BioFabrication Facility (BFF), which it expects to launch to the station near the end of 2018. Critical to the success of the printer will be the ability to provide nutrients and mechanical stress for organs and tissues it manufactures in space strengthening them and keeping them viable for transplantation back on Earth.

Approximately 36 hours prior to launch, Techshot scientists in a laboratory at the Kennedy Space Center will 3D print a one centimeter thick construct consisting of stem cells and heart muscle cells. Theyll then place it inside the prototype BFF cell culturing subsystem, which for this mission is temporarily housed inside an ADSEP cassette. The printer used in the lab will be the same modified nScrypt unit that was the first to 3D print cardiac constructs with adult human stem cells in microgravity aboard an aircraft in parabolic flight. Video captured inside the cassette during the month-long experiment, and the tissue itself which is expected to have developed its own micro blood vessels will be evaluated for effectiveness after return from space.

Techshots space bioprinting program leverages its terrestrially based technologies for cell isolation and vascular graft development, and its decades long experience culturing cells in space, said Techshot Chief Scientist Eugene Boland, Ph.D., in a news release. Being able to test our novel approach for culturing 3D printed cells more than a year before we fly the whole BFF is invaluable. The data from this mission will get us one step closer toward our goal of helping eliminate organ shortages.

Founded in 1988, Techshot Inc., develops technologies used in the aerospace, defense and medical industries. Through its Space Act Agreement with NASA, and its role as an official CASIS Implementation Partner, the company provides equipment and services that help federal, institutional and industrial customers live and work in space. http://www.Techshot.space

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Techshot system headed to space – Evening News and Tribune

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Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair – Texas Medical Center (press release)

Earlier this year, Texas Heart Institute received Alpha Phi Foundations 2017 Heart to Heart Grant. The $100,000 grant will fund research led by Doris Taylor, Ph.D., director of the Regenerative Medicine Research and the Center for Cell and Organ Biotechnology at the Texas Heart Institute, to study cardiac repair in women at the cellular level.

Were just really passionate about these projects that have long-term clinical relevancy, as a women-driven organization and being committed to womens heart health, said Colleen Sirhal, vice chair of the Alpha Phi Foundation.

The study will explore sex differences in blood, bone marrow and stem cells of patients enrolled in cell therapy clinical trials.

While bone marrow cell therapy has been used to treat cardiovascular disease in clinical trials, very few studies have been conducted to assess the sex differences in efficacy and outcomes. By performing a proteomic analysis of the samples and evaluating the proteins that cells produce and secrete, the results could shed light on unanswered questions related to critical sex-specific differences in cardiovascular disease, potentially leading to improved cell therapies.

Its about time that were paying attention to sex differences, Taylor said. Were not just small men. The biology is different.

Heart disease remains the No. 1 cause of death in both men and women in the United States, yet theres a limited understanding in the scientific community as to why it affects men and women differently. For example, women 45 years old and younger have a higher likelihood than men of dying within a year of their initial heart attack.

In addition, women have a higher risk of developing small vessel disease, in which the walls of tiny vessels within the heart muscle become blocked rather than larger arteries, causing heart-related chest pain. Because the major coronary arteries may look normal, women with small vessel disease can have a heart attack go undiagnosed and untreated.

We know heart disease happens differently in men and women, Taylor said. More young women than men die of heart disease. Why is that? Is there something that happens early? If we only look at these women who are older, are we missing something major? By looking at healthy, normal younger women, were going to be able to do comparisons across time, comparisons by disease, and comparisons by sex. I think thats really exciting.

Historically, women and minorities have largely been underrepresented in research and clinical trials, especially pertaining to cardiovascular disease.

Dr. Taylors colleague at the Texas Heart Institute, Stephanie Coulter, M.D., a cardiologist and the director of the Center for Womens Heart and Vascular Health at Texas Heart Institute and a recipient of the 2013 Heart to Heart Grant, is actively recruiting younger women to participate in her research registry.

Since women are typically affected by heart disease a decade or more later than men, age may also have played a role in this underrepresentation, Coulter said. Our Womens Center research is focusing on women age 18 and older to address this very issue.

Coulter added that trials focusing on prevention in women, such as the Womens Health Initiative and Womens Health Study, have, in fact, had clinical impact. However, the percentage of women enrolling in clinical trials continues to be disproportionate to the prevalence of cardiovascular disease in women, but we are seeing improvements thanks to multiple initiatives in the U.S. that continue to address the issue of women in clinical trials.

Its easy for people to assume that if you study men, itll apply to women, but it seems anathema to people to assume that if you study women it might benefit men, Taylor said. At the end of the day, when it comes time to look at the data and ask, How does this treatment work in women? How does this treatment work in men?, oftentimes there arent enough women enrolled in the trials to split that out. Statistically, youd be doing yourself a disservice.

Taylor has spent nearly two decades studying key contributors to cardiac repair at the cellular level, specifically looking at proteins cells produce and secrete based on gender as a new frontier in cell therapy.

Early on in Taylors career, she studied how bone marrow cells behaved based on gender. She extracted cells from male mice and administered them to female mice and vice versa, allowing her to track the Y chromosome. The results showed that only the males treated with female cells improved. This phenomenon raised the question of whether or not the bone marrow cells were the same.

After measuring the bone marrow cells that were present in males and females, Taylor discovered that the cells were inherently different: In the male mice, there were more inflammatory cells, fewer progenitor and stem cells and a different number of immune cells than in the female mice. In addition, when the bone marrow cells were placed in a petri dish, the female cells produced more growth factors responsible for recruiting repair cells after an injury.

Taylor conducted follow-up experiments in which she gave female and male cells to both female and male mice. The results confirmed her hunch: The only cells that were reparative were the female cells.

It made me realize a critical detail for the first time:Every time we take bone marrow from a different person with the intention of delivering it back to them as a therapy, if we look at the cells present in the marrow, theyd be different, Taylor said. Which means, every time were doing an autologous cell therapytrial, in which you take bone marrow and deliver it back to an individual, you are giving each person a completely different or unique drug in that trial.

Through the Heart to Heart grant, the data from Taylors research will allow her to build upon her early research on sex differences and, hopefully, identify a way to optimize cell therapy.

Already cells are as good as some drugs. If we optimize them and choose the right cells for the right patient at the right time, maybe well hit the home run, Taylor said.

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Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair – Texas Medical Center (press release)

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VistaGen Receives Notice of Allowance from US Patent and Trademark Office for US Patent regarding Breakthrough … – Marketwired (press release)

SOUTH SAN FRANCISCO, CA–(Marketwired – August 08, 2017) – VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that the Company has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for U.S. Patent Application No. 14/359,517 regarding proprietary methods for producing hematopoietic precursor stem cells, which are stem cells that give rise to all of the blood cells and most of the bone marrow cells in the body, with potential to impact both direct and supportive therapy for autoimmune disorders and cancer.

The breakthrough technology covered by the allowed U.S. patent was discovered and developed by distinguished stem cell researcher, Dr. Gordon Keller, Director of the UHN’s McEwen Centre for Regenerative Medicine in Toronto, one of the world’s leading centers for stem cell and regenerative medicine research and part of the University Health Network (UHN), Canada’s largest research hospital. Dr. Keller is a co-founder of VistaGen and a member of the Company’s Scientific Advisory Board. VistaGen holds an exclusive worldwide license from UHN to the stem cell technology covered by the allowed U.S. patent.

“We are pleased to report that the USPTO has allowed another important U.S. patent relating to our stem cell technology platform, stated Shawn Singh, Chief Executive Officer of VistaGen. “Because the technology under this allowed patent involves the stem cells from which all blood cells are derived, it has the potential to reach the lives of millions battling a broad range of life-threatening medical conditions, including cancer, with CAR-T cell applications and foundational technology we believe ultimately will provide approaches for producing bone marrow stem cells for bone marrow transfusions. As we continue to expand the patent portfolio of VistaStem Therapeutics, our stem cell technology-focused subsidiary, we enhance our potential opportunities for additional regenerative medicine transactions similar to our December 2016 sublicense of cardiac stem cell technology to BlueRock Therapeutics, while focusing VistaStem’s internal efforts on using stem cell technology for cost-efficient small molecule drug rescue to expand our drug development pipeline.”

About VistaGenVistaGen Therapeutics, Inc. (NASDAQ: VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen’s lead CNS product candidate, AV-101, is in Phase 2 development, initially as a new generation oral antidepressant drug candidate for major depressive disorder (MDD). AV-101’s mechanism of action is fundamentally different from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a small Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with an inadequate response to standard, FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Company’s Phase 2 adjunctive treatment study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including neuropathic pain, epilepsy, Huntington’s disease, and levodopa-induced dyskinesia associated with Parkinson’s disease and other disorders where modulation of the NMDA receptors, activation of AMPA pathways and/or key active metabolites of AV-101 may achieve therapeutic benefit.

About VistaStemVistaStem Therapeutics is VistaGen’s wholly-owned subsidiary focused on applying human pluripotent stem cell (hPSC) technology, internally and with third-party collaborators, to discover, rescue, develop and commercialize (i) proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases and (ii) cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. VistaStem’s internal drug rescue programs are designed to utilize CardioSafe 3D, its customized cardiac bioassay system, to develop small molecule NCEs for VistaGen’s pipeline. To advance potential regenerative medicine (RM) applications of its cardiac stem cell technology, in December 2016, VistaStem exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established in 2016 by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac cells for the treatment of heart disease. In a manner similar to its exclusive sublicense agreement with BlueRock Therapeutics, VistaStem may pursue additional collaborations and potential RM applications of its stem cell technology platform, including using blood, cartilage, and/or liver cells derived from hPSCs, for (i) cell-based therapy, (ii) cell repair therapy, and/or (iii) tissue engineering.

For more information, please visit http://www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook.

Forward-Looking StatementsThe statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH’s Phase 2 (monotherapy) and/or the Company’s planned Phase 2 (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, including neuropathic pain and L-DOPA-induced dyskinesia associated with Parkinson’s disease, the potential for the Company’s stem cell technology to produce NCEs, cellular therapies, regenerative medicine or bone marrow stem cells to treat any medical condition, including autoimmune disorders and cancer, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the AV-101 clinical development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen’s filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC’s website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Receives Notice of Allowance from US Patent and Trademark Office for US Patent regarding Breakthrough … – Marketwired (press release)

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Modern Fertility is offering a comprehensive fertility test for women who hope to be moms someday – TechCrunch

There are a number of ways to find out more about your fertility these days including from several at-home fertility test startups that have started to pop up in the last few years.Modern Fertility hopes to soon operate in much the same way, but with a more affordable option for testing 10 key hormones affecting womens fertility.

Though Modern Fertilitys at-home test wont be available till later this year, you can pre-order it on their website for $149 though the price will go up after the pre-order at a yet-to-be determined date. Should you want to get started now, the startup also offers the comprehensive screening through a lab near you, though its not clear what the price is for that.

The kit includes checking your hormone levels for:

Anti-mullerian hormone (AMH)

Follicle stimulating hormone (FSH)

Estradiol (E2)

Luteinizing hormone (LH)

Thyroid stimulating hormone (TSH)

Free thyroxine (FT4)

Progesterone (P4)

Prolactin (PRL)

Free Testosterone (Free T)

Total Testosterone (T)

Modern Fertility competitor Future Family, a startup offering financing optionsfor egg freezing and IVF procedures, also sells two separate fertility tests you can take at home. The first test kit goes for $300 and includes the three most key hormone tests: AMH, FSH and E2. Future Familys second test, Fertility Age Test Plus, includes testing for the first three hormones and three tests for thyroid dysfunctions TSH, TPO (thyroperoxidase) and T3/T4. (triiodothironine andthyroxine levels) for a similar price.

Everlywell, a startup offering myriad home health tests, includes a similarly comprehensive fertility kitas Modern Fertility for $400, but with 11 hormone tests and not all of them are the same ones.

Half the price for more hormone testing seems like a deal. However, theres a hot debate among these startups over just how many of these hormone tests, and which ones, are necessary. Everlywell, for instance, doesnt include AMH because they consider that only necessary if you are about to undergo IVF. Future Family told TechCrunch only the three key tests are necessary unless you need thyroid testing, because the other hormone tests are widely accepted by doctors as not being true indicators of fertility.

How does each startup determine what is necessary? Everlywell and Future Family are staffed with a chief medical officer to guide them. Modern Fertility is currently in search of the same, but says it pulls its information from medical advisors and has held initial conversations with fertility doctors.

Obviously, ask your doctor which kit is right for you (or if theres another they suggest). The overall goal for all three is the same empower women with knowledge about their fertility.

Modern Fertilitys main target is young women who want a family someday, but not necessarily today.

Were building a test that makes this info accessible to women early in their lives,co-founder Afton Vechery said. We believe that information is the first step.

Vechery, who was a product lead at 23andMe before starting Modern Family, says she became interested in the space after doing some due diligence in the infertility space for a healthcare private equity firm earlier in her career.

Thats when I learned the emotional aspect of infertility. Its crazy to me that infertility is not seen as a medical condition in the majority of the U.S. and that such a small percentage of women get the education and services they need to start a family, she said. Thats the part that stuck with me.

She then went through some testing at a clinic to find out more about her own fertility. That was an impactful moment for her. That is, until she got the $1,500 bill in the mail.

As I started talking to more women it was clear there was a lot of anxiety over fertility but no way to afford to test it, Vechery said. Every woman should have access to this information that is a better predictor than just our ages.

Modern Fertility is currently in Y Combinators latest batch. You can catch them later this month at YC Demo Day.

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Modern Fertility is offering a comprehensive fertility test for women who hope to be moms someday – TechCrunch

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Transgender clinic for kids and teens opens in St. Louis | WGN-TV – WGN-TV

St. Louis is now home to a first-of-its-kind clinic for transgender teens and kids. It’s being administered by Washington University Physicians based out of St. Louis Children’s hospital.

St. Louis is now home to a first-of-its-kind clinic for transgender teens and kids. It’s being administered by Washington University Physicians based out of St. Louis Children’s hospital.

St. Louis, MO St. Louis is now home to a first-of-its-kind clinic for transgender teens and kids. Its being administered by Washington University physicians based out of St. Louis Childrens hospital.

The clinic aims to provide transgender children and teens with comprehensive health care including mental health resources, hormonal therapy, voice therapy, and reconstructive surgery. Washington University physicians have been taking care of transgender children and teens since 2009 and noticed the growing demand which sparked the push to establish a fully operating clinic.

In 2016, Washington University physicians had 74 transgender patients. From just January to May 2017, theyve already seen 71 patients.

This clinic is a huge milestone for the Seay family this month, especially for 15-year-old Leslie.

Sometimes I identify as a girl, sometimes a boy, sometimes neither, said Leslie Seay.

She started exploring gender identity at 13-years-old, ultimately assigning to being gender fluid, which means she will always feel a mix of identifying between a boy and a girl.

I would really like to go on hormone blockers so that my voice doesnt get any more feminine and [no] more feminine features show up, said Seay.

Leslies identity is simple to her, so she needs a pediatrician who understands transgender health simply too.

Having support and acceptance is extremely important for this patient population, said Dr. Christopher Lewis, founder and physician of the Transgender clinic. Transgender patients already deal with harassment and discrimination within the medical community and that is a barrier to them accessing care.

Leslies dad, Peter Seay, is thrilled to know his child is in safe, supportive care with an expertise in transgender health.

To find out that the gender center was opening this month was something weve been celebrating for a little while. Weve been very excited about this, said Peter Seay. There could not be a greater value, the gratitude will not stop.

The Transgender Center of Excellence opened the first week of August. They are already booked through mid-September with new patient appointments. Its the only clinic of its kind within a 250-mile radius.

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Transgender clinic for kids and teens opens in St. Louis | WGN-TV – WGN-TV

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Elevated TSH level doesn’t always mean medication is necessary – Post-Bulletin

DEAR MAYO CLINIC: I started taking levothyroxine more than five years ago for hypothyroidism. I had my TSH level tested about six months after I began taking it but have not had it checked since. I recently read a study saying this medication is often prescribed even when it’s not necessary. Should I see my doctor to be retested?

Yes. Make an appointment to have your condition re-evaluated at this time. For some people, lifelong treatment of hypothyroidism with the drug levothyroxine is necessary. But studies have found that for many others who have elevated levels of thyroid-stimulating hormone, or TSH, the medication isn’t needed. In fact, if it’s taken incorrectly or in doses that are too large, levothyroxine can cause harmful side effects.

Your thyroid is a small, butterfly-shaped gland at the base of the front of your neck. The thyroid gland makes two hormones — triiodothyronine, or T3, and thyroxine, or T4 — that have a large impact on your health, affecting all aspects of your metabolism. They maintain the rate at which your body uses fats and carbohydrates, help control your body temperature, influence your heart rate, and help regulate the production of proteins. The rate at which your thyroid makes T3 and T4 is regulated by another hormone that your pituitary gland produces, called TSH.

Hypothyroidism, sometimes called underactive thyroid, is a condition in which your thyroid gland doesn’t produce enough T3 or T4. When blood tests show that you have high levels of TSH and low levels of T3 or T4 in your body, then a diagnosis of hypothyroidism is clear. Treatment with levothyroxine — a synthetic version of thyroid hormones — is necessary in almost all cases. But hypothyroidism is rare, affecting only about 0.2 percent of the population.

Much more common, affecting about 12 percent of the population, is a condition known as subclinical hypothyroidism. With this condition, your TSH level is above normal, but T3 and T4 levels are normal.

If a blood test shows you have subclinical hypothyroidism, and you don’t have any symptoms — such as fluid retention, fatigue, increased sensitivity to cold, constipation, muscle weakness or painful joints — treatment typically is not recommended. There are a few reasons for that.

First, about 30 percent of people whose condition falls into the category of subclinical hypothyroidism have their TSH levels return to normal within one year without treatment. Only 3 percent per year go on to develop hypothyroidism. Second, if you take too much levothyroxine or if you don’t take it correctly, it can negatively affect a variety of your body’s systems, including your brain, heart and muscle function. It also can interfere with how your body handles fluid and fats.

If, as in your case, you are receiving treatment for hypothyroidism, it’s important to have regular checkups. Testing TSH is one way to see if treatment is working. It’s also important for your health care provider to check your T4 levels.

Talk with your health care provider about the goals of treatment, too. If you started taking levothyroxine to control symptoms, make sure that you are seeing some benefit. Also, keep in mind that the symptoms of hypothyroidism often can be vague. If your symptoms don’t go away when you’re taking thyroid medication, it’s possible those symptoms could be linked to another medical condition.

Getting your TSH and T4 levels checked and reviewing any symptoms you may have with your health care provider should help clarify whether you need to continue taking levothyroxine. — Juan Brito Campana, M.B.B.S., Endocrinology, Mayo Clinic, Rochester.

Mayo Clinic Q & A is an educational resource and doesn’t replace regular medical care. Email a question to MayoClinicQ&A@mayo.edu. For more information, visit http://www.mayoclinic.org.

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Elevated TSH level doesn’t always mean medication is necessary – Post-Bulletin

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Silverstein-backed startup will test gene therapy for Parkinson’s – FierceBiotech

Regenxbio has joined forces with investment firm OrbiMed and a new nonprofit foundation to create Prevail Therapeutics, a startup focused on new biologics and gene therapiesfor Parkinson’s disease (PD).

Prevail will draw on the expertise of the Silverstein Foundation for Parkinson’s with GBA, which concentrates on a particular form of the disease caused by mutations in the glucocerebrosidase gene.

The foundation was set up this year by OrbiMed’s co-head of private equity Jonathan Silverstein, who was diagnosed with GBA-linked PD in February and is mobilizing efforts to discover a cure for the disease. Silverstein backed the foundation with $10 million of his own money, and is intent on accelerating research into PD with GBA as well as other forms of the disease.

Prevail says it will focus initially on research coming out of the lab of its co-founder and CEO Asa Abeliovich, M.D., Ph.D., who is on the faculty of Columbia University as well as being a scientific adviser to the Silverstein Foundation and co-founder of neurodegenerative disease biotech Alector.

By joining forces with Regenxbio, Prevail launches with an exclusive license to the gene therapy specialist’s adeno-associated virus (AAV) based vector technology NAV AAV9 for PD and other neurodegenerative disorders.

Silverstein said that the NAV platform and Dr. Abeliovich’s “deep expertise in the molecular mechanisms of neurodegeneration provides us with a promising opportunity to develop potential life-changing therapies for patients suffering from Parkinson’s disease and other neurodegenerative diseases.”

He told CNBC today that Prevail’s board will also have some big names, including Leonard Bell, co-founder and former CEO of Alexion, OrbiMed venture partner and Alexion co-founder Steve Squinto and serial entrepreneur Peter Thompson of Silverback Therapeutics and Corvus Pharmaceuticals.

The new company will initially focus on GBA1, the most common of the PD mutations, which is estimated to be present in up to 10% of U.S. PD patients and perhaps 100,000 people worldwide. The disease mechanism linked to the mutationan accumulation of alpha-synuclein in the brainmay have implications for the broader PD population and other neurodegenerative diseases.

“Many of the drugs we are trying for Parkinson’s with GBA may work in the broader Parkinson’s population,” said Silverstein. The aim will be to get drugs approved for use in GBA patients first, and then expand their use into other patient groups.

The work of the foundation is attracting investment from companies who are not even active in PD, with cancer specialist Celgene today pledging a grant of $5 million.

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Pfizer investing $100M in Sanford plant expansion, adding jobs … – Triangle Business Journal


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Pfizer investing $100M in Sanford plant expansion, adding jobs …
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Pfizer has confirmed plans to invest $100 million in the expansion of its Sanford research and manufacturing plant.

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Pfizer investing $100M in Sanford plant expansion, adding jobs … – Triangle Business Journal

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Pfizer chooses Sanford, North Carolina site for $100m gene therapy plant – BioPharma-Reporter.com

Pfizer has chosen a site in Sanford, North Carolina for a gene therapy production plant, just 40 miles from its recent acquisition Bamboo Therapeutics Inc.

The US drug firm had been search for a site since March.

According to North Carolina Governor Roy Cooper, Pfizer will spend $100m (85m) on the new facility and has also committed $4m to support postdoctoral fellowships in North Carolina universities for training in gene therapy research.

The project will create jobs that deliver a total payroll impact of more than $3.9m each year to the community according to the North Carolina Department of Commerce and the Economic Development Partnership.

The project will be part funded by a $250,000 grant previously awarded to Wyeth which was acquired by Pfizer in 2009 – by the One North Carolina Fund, which helps local Governments attract economic investment.

Bamboo buy

The decision follows a little over a year after the US drug manufacturer acquired Bamboo Therapeutics, a North Carolina-based gene therapy developer.

The deal included a recombinant Adeno-Associated Virus (rAAV) vector design and production technology, a Phase I candidate for Giant Axonal Neuropathy and a preclinical programme targeting Duchenne Muscular Dystrophy (DMD).

Pfizer also gained a 11,000sq ft gene therapy manufacturing facility in Chapel Hill that Bamboo bought from the University of North Carolina in 2016.

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Pfizer chooses Sanford, North Carolina site for $100m gene therapy plant – BioPharma-Reporter.com

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Springfield Mom Works to Raise Awareness after Son Diagnosed with Rare Genetic Disorder – KSMU Radio

Parker Atchley is your typical four-year-old in many ways. Hes shy at first but usually opens up after he gets to know you, he loves anything with wheels, baseball and splashing in water, and he sticks pretty close to his mom, but a grandma will do if moms not available.

Hes an adorable, blonde boy with dark-rimmed glasses and a stare that makes you feel like hes thoroughly assessing you but that also melts your heart.

He also is suffering from a rare diseaseor at least a disease that only a few have been diagnosed with so far.

Sitting on the floor of her living room in southeast Springfield, Parkers mom, Kathryn Atchley, said they got a diagnosis in January: KIF1A-related disorder. It was a long journey to get to that point.

“When he was around one, we realized that something was off. We weren’t sure. The director of the day care he was going to actually called a meeting with us to talk about some concerns because he wasn’t walking at that point. He had just started traversing around furniture,” she said.

His balance was also off. Kathryn and her husband, Tyler, thought maybe it was an ear or eye problem. They took Parker to a pediatrician, an ear, nose and throat doctor and a neurologist. All tests came back normal. So they enrolled him in physical therapy. As he got older, they held onto hope that Parker would get better, but he didnt and they grew more and more frustrated.

“It started getting really worrisome when we realized that he was losing skills. Two years ago he used to be able to stand up on his own, and he can’t do that anymore. He was taking nine steps at a time, and he can’t do that anymore,” said Kathryn.

The Atchleys took Parker to a second neurologist who recommended genetic testing. They finally got the diagnosis that Kathryn said changed everything.

“Cause then we realized it wasn’t just a rare disorder, it was neurodegenerative,” she said.

They no longer clung to the hope that Parker was just delayed and that physical therapy could help him catch up.

Dr. Wendy Chung, a physician with Columbia University in New York, saidKIF1A-related disorder is a genetic condition, caused by a mutation in the KIF1A gene, which was identified fairly recentlyin the last 10 years or so. It affects the brain and nervous system and, while some with the disorder are affected mildly, others, including Parker, can have severe disabilities. Those include muscle tightening, spasticity and difficulty developing. The most disheartening thing about the condition, she said, is that its degenerative.

“That is that children take steps back. They lose abilities. They lose vision,” said Dr. Chung

The first piece of advice given to the Atchleys by the neurologist was to go online and find other families affected by KIF1A. Kathryn said the doctor had never heard of the disorder before.

“He was very upfront and honest and said, you know, ‘I’m learning this as you guys learn it,'” she said.

She found a Facebook support group for families with children who have KIF1A-related disorder, and, at the time, there were only 20 families in the group. Its since grown some as more kids get diagnosed through whole genome testing.

Dr. Chung said shed venture to guess that the vast majority of people with KIF1A-related disorder dont even realize they have this condition.

“There are a lot of individuals out there that have symptoms who just don’t know what they are. There’s powerful new sequencing and genetic technology to figure that out, and, you know, I think that’s what people like me are trying to do is make sure patients get access to that type of testing,” said Dr. Chung.

Whole Exome Sequencing, a fairly expensive test that can be cost prohibitive, isnt covered by all insurance companies.

But Dr. Chung said other tests on children to get a diagnosis, such as MRIs and brain scans, are also expensive and dont always result in an answer. And she said having a diagnosis is valuable at many levels.

“Certainly I think for the families just having an answer about what this is and what to expect and what they can do to keep their child healthy and learning and, you know, improve their quality of life or maintain their quality of life, I think is huge if you ask any of the families. I think the other portion of this is that you don’t waste your time doing things that are not necessary,” she said.

According to Dr. Chung, more diagnoses ofKIF1A-related disorder will hopefully lead to more research to find a cure.

Parents of kids with KIF1A-related disorder, including Kathryn Atchley, are working to raise awareness about the condition and money for research.

“You know, in connecting with other families I think there’s a lot of us parents that, ‘I don’t want to sit by and do nothing and just kind of let the medical community figure it out,'” said Kathryn.

Dr. Chung has taken KIF1A parents under her wing and tells them shes their Sherpatheir guide through a frightening diagnosis up what she calls a large mountain as they try to seek treatment for their children. Currently, treatment is supportive and consists of things like physical therapy, getting seizures under control and helping patients maintain their mobility.

Dr. Chung calls herself an optimistic person and is hopeful a cure will be found one day, but she doesnt know if it will be five, ten or twenty years down the road. She also calls herself a realist and said scientists are just beginning to understand KIF1A-related disorder. But she knows that scientific knowledge, especially in neuroscience is growing much more rapidly than ever before.

“So I’m confident that treatments are going to be available in the future. The question though is, you know, how quickly can we accelerate that? You know…is the future going to be 20 years off? Can we accelerate it to be five years off? And that, I think, is still fundamentally unknown,” said Dr. Chung.

After the diagnosis, Kathryn said they began adjusting their hopes and dreams for their son. She couldnt look at Parker without crying.

“And one day I was sitting at the coffee table playing with him, and he trailed tears down my face, and I realized that I was really robbing us of our present with Parker,” she said.

Kathryn, who calls herself an optimist in training, doesnt want to stand by and let the disease take away the progress Parker has made. She wants to do all she can to help find a cure.

“I have to get people to care about Parker and the rest of these kids if they’re going to have any hope of finding treatment or a cure,” she said.

Dr. Chung said funding is needed to support research on genetic disorders like KIF1A. You can find out more and donate at kif1a.org. Read about the Atchley family’s journey with KIF1A-related disorder on Kathryn’s blog.

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Springfield Mom Works to Raise Awareness after Son Diagnosed with Rare Genetic Disorder – KSMU Radio

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Wild new microchip tech could grow brain cells on your skin – CNET

Researchers demonstrate a process known as tissue nanotransfection (TNT). When it comes to healing, this TNT is the bomb.

It’s usually bad news to have something growing on your skin, but new technology uses that all important layer as a sort of garden to “grow” whatever types of cells your body might need to treat an injury or disease, be it in a limb or even the brain.

Researchers atthe Ohio State University Wexner Medical Centerhave developed a nanochip that uses a small electrical current to deliver new DNA or RNA into living skin cells, “reprogramming” them and giving them a new function.

“It takes just a fraction of a second. You simply touch the chip to the wounded area, then remove it,”Chandan Sen, director of the Center for Regenerative Medicine and Cell-Based Therapies at Ohio State, said in a statement. “At that point, the cell reprogramming begins.”

In a study published in the journal Nature Nanotechnology, Sen’s team used a technology called Tissue Nanotransfection (TNT) to create new blood vessels in pigs and mice with badly injured limbs that lacked blood flow.

They zapped the animals’ skin with the device, and within about a week, active blood vessels appeared, essentially saving the creatures’ legs. The tech was also used to create nerve cells from skin that were then harvested and injected into mice with brain injuries to help them recover.

“By using our novel nanochip technology, injured or compromised organs can be replaced,” Sen said. “We have shown that skin is a fertile land where we can grow the elements of any organ that is declining.”

While it sounds futuristic, reprogramming skin cells is not a new idea. The ability to change skin into pluripotent stem cells, sometimes called “master” cells, earned a few scientists a Nobel Prize half a decade ago. But the new nanochip approach improves upon that discovery by skipping the conversion from skin to stem cell and instead converting a skin cell into whatever type of cell is desired in a single step.

“Our technology keeps the cells in the body under immune surveillance, so immune suppression is not necessary,” Sen says.

By now I think we’ve all learned that beauty is only skin deep, but it might take a while to learn that the same could go for cures, at least if the system works just as well on people.

Next up, the scientists hope to find out by continuing to test their technology in human trials. The aim is that it could eventually be used to treat all sorts of organ and tissue failure, including diseases like Parkinson’s and Alzheimers.

Crowd Control: A crowdsourced science fiction novel written by CNET readers.

Solving for XX:The tech industry seeks to overcome outdated ideas about “women in tech.”

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Wild new microchip tech could grow brain cells on your skin – CNET

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A Chip That Reprograms Cells Helps Healing, At Least In Mice – NPR

The chip has not been tested in humans, but it has been used to heal wounds in mice. Wexner Medical Center/The Ohio State University hide caption

The chip has not been tested in humans, but it has been used to heal wounds in mice.

Scientists have created an electronic wafer that reprogrammed damaged skin cells on a mouse’s leg to grow new blood vessels and help a wound heal.

One day, creator Chandan Sen hopes, it could be used to be used to treat wounds on humans. But that day is a long way off as are many other regeneration technologies in the works. Like Sen, some scientists have begun trying to directly reprogram one cell type into another for healing, while others are attempting to build organs or tissues from stem cells and organ-shaped scaffolding.

But other scientists have greeted Sen’s mouse experiment, published in Nature Nanotechnology on Monday, with extreme skepticism. “My impression is that there’s a lot of hyperbole here,” says Sean Morrison, a stem cell researcher at the University of Texas Southwestern Medical Center. “The idea you can [reprogram] a limited number of cells in the skin and improve blood flow to an entire limb I think it’s a pretty fantastic claim. I find it hard to believe.”

When the device is placed on live skin and activated, it sends a small electrical pulse onto the skin cells’ membrane, which opens a tiny window on the cell surface. “It’s about 2 percent of the cell membrane,” says Sen, who is a researcher in regenerative medicine at Ohio State University. Then, using a microscopic chute, the chip shoots new genetic code through that window and into the cell where it can begin reprogramming the cell for a new fate.

Sen says the whole process takes less than 0.1 seconds and can reprogram the cells resting underneath the device, which is about the size of a big toenail. The best part is that it’s able to successfully deliver its genetic payload almost 100 percent of the time, he says. “No other gene delivery technique can deliver over 98 percent efficiency. That is our triumph.”

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice. Wexner Medical Center/The Ohio State University hide caption

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice.

To test the device’s healing capabilities, Sen and his colleagues took a few mice with damaged leg arteries and placed the chip on the skin near the damaged artery. That reprogrammed a centimeter or two of skin to turn into blood vessel cells. Sen says the cells that received the reprogramming genes actually started replicating the reprogramming code that the researchers originally inserted in the chip, repackaging it and sending it out to other nearby cells. And that initiated the growth of a new network of blood vessels in the leg that replaced the function of the original, damaged artery, the researchers say. “Not only did we make new cells, but those cells reorganized to make functional blood vessels that plumb with the existing vasculature and carry blood,” Sen says. That was enough for the leg to fully recover. Injured mice that didn’t get the chip never healed.

When the researchers used the chip on healthy legs, no new blood vessels formed. Sen says because injured mouse legs were was able to incorporate the chip’s reprogramming code into the ongoing attempt to heal.

That idea hasn’t quite been accepted by other researchers, however. “It’s just a hand waving argument,” Morrison says. “It could be true, but there’s no evidence that reprogramming works differently in an injured tissue versus a non-injured tissue.”

What’s more, the role of exosomes, the vesicles that supposedly transmit the reprogramming command to other cells, has been contentious in medical science. “There are all manners of claims of these vesicles. It’s not clear what these things are, and if it’s a real biological process or if it’s debris,” Morrison says. “In my lab, we would want to do a lot more characterization of these exosomes before we make any claims like this.”

Sen says that the theory that introduced reprogramming code from the chip or any other gene delivery method does need more work, but he isn’t deterred by the criticism. “This clearly is a new conceptual development, and skepticism is understandable,” he says. But he is steadfast in his confidence about the role of reprogrammed exosomes. When the researchers extracted the vesicles and injected them into skin cells in the lab, Sen says those cells converted into blood vessel cells in the petri dish. “I believe this is definitive evidence supporting that [these exosomes] may induce cell conversion.”

Even if the device works as well as Sen and his colleagues hope it does, they only tested it on mice. Repairing deeper injuries, like vital organ damage, would also require inserting the chip into the body to reach the wound site. It has a long way to go before it can ever be considered for use on humans. Right now, scientists can only directly reprogram adult cells into a limited selection of other cell types like muscle, neurons and blood vessel cells. It’ll be many years before scientists understand how to reprogram one cell type to become part of any of our other, many tissues.

Still, Morrison says the chip is an interesting bit of technology. “It’s a cool idea, being able to release [genetic code] through nano channels,” he says. “There may be applications where that’s advantageous in some way in the future.”

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A Chip That Reprograms Cells Helps Healing, At Least In Mice – NPR

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Chip reprograms skin cells with a short electric pulse – New Atlas

Technologies that reprogram one type of cell to perform the role of another hold a huge amount of potential when it comes to medicine, possibly changing the way we treat everything from Parkinson’s to pancreatic cancer to brain tumors. One broader outcome of all of this could be a game-changing ability to repair and restore damaged tissue and organs. Scientists are now reporting a promising advance in the area, in the form of patch that they say can use an electric pulse to turn skin cells into the building blocks of any organ.

The new technology has been dubbed tissue nanotransfection and was developed by scientists at The Ohio State University’s Wexner Medical Center. According to the researchers, it uses the skin as a kind of regenerative cellular factory, where it produces any cell type that can then be used to repair injured or aging tissues, organs and blood vessels. It consists of a nanotechnology-based chip that is applied to the skin, which is then struck with a short electric pulse to deliver genetic instructions into the cells of the tissue.

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“These are genes that induce tissue plasticity allowing the flexibility to direct the fate,” Chandan Sen, first author of the paper, explains to New Atlas. “Thus, for example, skin cells can be directed to form blood vessels, or neural cells, or some other cell of interest.”

We have seen a number of promising approaches to reprogramming cells for various regenerative health purposes. In 2012, a Japanese researcher won a Nobel Prize for his discovery that skin cells from mice could be harvested and converted into stem cells in the lab, work that has inspired a number of exciting breakthroughs since.

But according to Sen, one of the main advantages his tissue nanotransfection technology holds over other approaches is the fact that the cell conversion takes place in the body. This avoids the thorny issue of immune response, in which the host cells react to the newcomers and possibly attack them, something that can cause a raft of complications.

“Ours is reprogramming of not just cells but tissue within the live body under immune surveillance,” he tells us. “Our strategy must co-operate with physiological factors to achieve the end goal.”

That end goal is still a while away, but his team is making promising progress all the same. It put the technology to the test on animals, and in one experiment involving mice with badly injured legs lacking blood flow, it was able to convert skin cells into vascular cells. Within about a week, the legs featured active blood vessels. By the second week they were saved.

In a separate experiment, the team was also able to use the technology to reprogram skin cells into nerve cells, which were then injected into brain-injured mice to assist with stroke recovery.

“This is difficult to imagine, but it is achievable, successfully working about 98 percent of the time,” said Sen. “With this technology, we can convert skin cells into elements of any organ with just one touch. This process only takes less than a second and is non-invasive, and then you’re off. The chip does not stay with you, and the reprogramming of the cell starts. Our technology keeps the cells in the body under immune surveillance, so immune suppression is not necessary.”

The team hopes to move onto clinical trials some time next year, but Sen tells us they must first test the technology on larger animals and design the device to work on humans.

You can hear from Sen in the video below, while the research was published in the journal Nature Nanotechnology.

Source: Ohio State University

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Chip reprograms skin cells with a short electric pulse – New Atlas

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Miraculous Burn-Healing Through Stem Cell Treatment – Fox Weekly

A med-tech startup has developed a fast and easy way to treat certain burn wounds with stem cells. This technology is developed by German researcher Dr. Jrg Gerlach. He is the worlds first ever person who use a patients stem cells to directly heal the skin. The technique is meant to reduce the healing time and minimize complications, with aesthetically and functionally satisfying outcomes. There are no scars, no residual pain and its like there wasnt any burn to start with. Its not less than a miracle.

The medical technology startup has now transformed the proof-of-concept device from a complicated prototype into a user-friendly product called a SkinGun, which it hopes doctors will be able to use outside of an experimental setting. RenovaCare CEO Thomas Bold believes, the SkinGun can compete with, or even replace, todays standard of care. The sprayer allows us to have a generous distribution of cells on the wound, explained Roger Esteban-Vives, director of cell sciences at RenovaCare.

RenovaCares SkinGun sprays a liquid suspension of a patients stem cells onto a burn or wound in order to re-grow the skin without scars. Stem-cell methods helped cut this risk by quickening healing and providing a source of new skin from a very small area. Cell Mist method gets a greater yield from its harvest than mesh grafting, a more common way to treat burns. At a maximum, grafting can treat six times the size of its harvest area. Cell Mist can cover 100 times its harvest area.

When dispensing cells over a wound, its important that they make the transition without any damage. Damaged cells reduce the effectiveness of the treatment.

High cell viability also contributes to faster healing. When a wound heals naturally, cells migrate to it to build up the skin. That process can take weeks.

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions.

There is still a lot to learn about stem cells, however, and their current applications as treatments are sometimes exaggerated by the media and other parties who do not fully understand the science and current limitations

Beyond regulatory matters, there are also limitations to the technology that make it unsuitable for competing with treatments of third-degree burns, which involve damage to muscle and other tissue below the skin.

When burn victims need a skin graft they typically have to grow skin on other parts of their bodies. This is a process that can take weeks. A new technique uses stem cells derived from the umbilical cord to generate new skin much more quickly.The umbilical cord consists of a gelatinous tissue that contains uncommitted mesenchymal stemcells (MSC)

Research is underway to develop various sources for stem cells, and to apply stem-cell treatments for neurodegenertive diseasesand conditions such as diabetes, heart disease, and other conditions.

Tens of thousands of grafts are performed each year for burn victims, cosmetic surgery patients, and for people with large wounds having difficulty healing. Traditionally, this involves taking a large patch of skin (typically from the thigh) and removing the dermis and epidermis to transplant elsewhere on the body. Burns victims are making incredible recoveries thanks to a revolutionary gun that sprays stem cells on to their wounds, enabling them to rapidly grow new skin. Patients who have benefited say their new skin is virtually indistinguishable from that on the rest of the body.

Thomas Bold, chief executive of RenovaCare, the company behind SkinGun, said: The procedure is gentler and the skin that regrows looks, feels and functions like the original skin.

If you are planning to have stem cell treatments dont forget to remember these points

Stem cell researchers are making great advances in understanding normal development. They are trying to figure out what goes wrong in disease and developing and testing potential treatments to help patients. They still have much to learn. However, about how stem cells work in the body and their capacity for healing. Safe and effective treatments for most diseases, conditions and injuries are in the future.

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Daiichi Sankyo invests in Osaka University spin-off – The Pharma Letter (registration)

Japanese pharma major Daiichi Sankyo (TYO: 4568) revealed this morning that it has signed an investment contract with Cuorips Inc, an Osaka University spin-off venture to receive an option right concerning the worldwide commercialization of iPS-derived cardiomyocyte (iPS-CM) sheet developed by Cuorips.

The iPS-CM sheet is an allogeneic cell therapy product consisting of cardiomyocyte derived from human iPS cells. Its transplantation is expected to provide improvement of cardiac function and amelioration of heart failure and become a new treatment option for patients with severe heart failure, who have no remedies other than heart transplantation or artificial heart implantation.

Based on the cutting-edge cell therapy research targeting heart diseases, the team at the Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, led by Professor Yoshiki Sawa, has been working on the iPS-CM research and development by participating in the Research Center Network for Realization of Regenerative Medicine, which is run by the Japan Agency for Medical Research and Development (AMED). They are currently preparing for clinical research as well as investigator initiated clinical study.

Cuorips was founded to develop and commercialize iPS-CM sheets based on the research data and technologies developed by the university.

Daiichi Sankyo has been conducting research on iPS cell-derived cardiomyocyte and their production, and is currently working on the efficient production process capable for commercial supply. Daiichi Sankyo and Cuorips are aiming to commercialize iPS-CM sheets as a pioneering treatment for severe heart failure.

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Lipocine (LPCN) Resubmits NDA for Oral Testosterone Product Candidate, LPCN 1021, for Treatment of Hypogonadism – StreetInsider.com

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Lipocine Inc. (NASDAQ: LPCN), a specialty pharmaceutical company, today announced the resubmission of a New Drug Application (NDA) for LPCN 1021, its oral testosterone product candidate for testosterone replacement therapy (TRT) in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.

Lipocine had previously submitted an NDA for LPCN 1021 and received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) in June 2016. The CRL identified a deficiency related to the dosing algorithm for the proposed label. With the goal of addressing this deficiency, the company successfully completed a dosing validation (DV) study, which confirmed the validity of a fixed dose approach without the need for dose titration to orally administer LPCN 1021. The efficacy results of the DV study, as well as an integrated safety set (ISS) from all previously conducted clinical trials, including 52-week safety results from the Phase 3 Study of Androgen Replacement (SOAR) clinical study, form the basis for the NDA resubmission.

Resubmission of this NDA as planned is an important milestone in bringing LPCN 1021 to patients, said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. We believe the results from the recently completed DV study address the label-related deficiency identified by the FDA in the CRL. We consider LPCN 1021 to be a differentiated TRT option for treating hypogonadism in men. We anticipate a six-month review by the FDA with a projected PDUFA date in the first quarter of 2018 assuming the FDA acknowledges our submission is a complete response.

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Lipocine (LPCN) Resubmits NDA for Oral Testosterone Product Candidate, LPCN 1021, for Treatment of Hypogonadism – StreetInsider.com

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Your brain can form new memories while you are asleep … – Washington Post

A sleeping brain can form fresh memories, according to a team of neuroscientists.The researchers played complex sounds to people while they were sleeping, and afterward the sleepers could recognizethose sounds when they wereawake.

The idea that humans canlearn while asleep, a concept sometimes called hypnopedia, has a long and odd history. It hit a particularly strange note in 1927, when New York inventor A. B. Saliger debuted thePsycho-phone. He billed the device as anautomatic suggestion machine. The Psycho-phone was a phonograph connected to a clock. It playedwax cylinder records, which Saliger made and sold.The records hadnames like Life Extension, Normal Weight orMating. That last one went: I desire a mate. I radiate love My conversation is interesting. My company is delightful. I have a strong sex appeal.

Thousands of sleepers bought the devices, Saligertold theNew Yorkerin 1933. (Those included Hollywood actors,he said, though he declined to name names.) Despite his enthusiasm for the machine Saligerhimself dozed off to Inspiration and Health the device was a bust.

But the idea that we can learn while unconscious holds more meritthan gizmos namedPsycho-phone suggest. In the new study, published Tuesday in the journalNature Communications, neuroscientistsdemonstrated that it is possible to teach acoustic lessons to sleeping people.

We proved that you can learn during sleep, which has been a topic debated for years, said Thomas Andrillon, an author of the study and a neuroscientist at PSL Research University in Paris.Just don’t expect Andrillon’s experiments to make anyonefluent in French.

Researchersin the 1950s dismantled hypnopedia’s more outlandish claims. Sleepers cannot wake up with brains filled withnew meaning or facts, Rand Corp. researchers reported in 1956. Instead, test subjectswho listened to trivia at night woke up with non-recall. (Still, the Psycho-phone spirit endures, at least in the app store, where hypnopedia software claims to promoteforeign languages, material wealth andmartial artsmastery.)

Yet success is possible, if you’re not trying to learn dictionary definitions or kung fu. In recent years, scientists have trained sleepers to make subconscious associations. In a 2014 study, Israeli neuroscientists had 66 people smell cigarette smoke coupled with foul odorswhile they were asleep. The test subjects avoided smoking for two weeks after theexperiment.

In the new research, Andrillon and his colleagues moved beyondassociation into pattern learning. While a group of20 subjects was sleeping, the neuroscientists played clips of white noise. Most of the audio was purely random, Andrillon said. There is no predictability. But there were patterns occasionally embedded within the complex noise: sequences of a single clip of white noise, 200 milliseconds long, repeated five times.

The subjects remembered the patterns. The lack ofmeaning worked in their favor; sleepers can neither focus on what they’re hearing nor make explicit connections, the scientist said. This is why nocturnal language tapes don’t quite work thebrain needs to register sound and semantics.But memorizing acoustic patterns like white noise happens automatically. The sleeping brain is including a lot of information that is happening outside, Andrillon said, and processing it to quite an impressive degree of complexity.

Once the sleepersawoke, the scientists played back the white-noise recordings. The researchers asked the test subjects to identify patterns within the noise. It’s not an easy task,Andrillon said, and one that you or I would struggle with. Unless you happened to rememberthe repetitions from a previous night’s sleep. The test subjects successfullydetected the patterns far better than random chance would predict.

What’s more, the scientists discovered that memories of white-noise pattern formed only during certain sleep stages. When the authors played the sounds during REM and light sleep, the test subjects could remember the pattern the next morning. Duringthe deeper non-REM sleep, playing the recording hampered recall. Patternspresented during non-REM sleep led to worse performance,as if there were a negative form of learning, Andrillon said.

This marked the first time that researchers had evidence for the sleep stages involved in the formation of completely new memories, said Jan Born, a neuroscientist at the Universityof Tbingen in Germany, who was not involved with the study.

In Andrillon’s view, the experiment helps to reconcile two competing theories about the role of sleep in new memories: In one idea,our sleeping brains replay memories from our waking lives. Asthey’re played back, the memories consolidate and grow stronger, written more firmly into our synapses. In the other hypothesis, sleep instead cuts away at older, weaker memories. But the ones that remain stand out, like lonely trees in a field.

The study indicates that the sleeping brain can do both,Andrillon said. They might simply occur at separatemoments in the sleep cycle, strengthening fresh memories followed by culling.

A separate team of neuroscientists had suspected that the two hypotheses might be complementary. But until now they did not have any explicit experimental support. It is a delight to see these results, since we proposed already, quite a few years ago, that the different sleep stages may have a different impact on memory, said Lisa Genzel,aneuroscientist atRadboud University in the Netherlands. And here they are the first to provide direct evidence for this idea.

Not all neuroscientists were so convinced. Born, an early proponent of the idea that sleep strengthens andconsolidates memories, said this study showed what happens when we form memories while asleep. The average memorya recollection from a waking experience might not work in the same way, he said. I would be skeptical about inferring from this type of approach to what happens during normal sleep.

Andrillon acknowledged the limitations ofthis research, including thatthe scientists did not directly measure synapses. We interpret our results in the light of cellular mechanisms, he said, meaning strengthening or weakening of synapses, that we could not directly measure, since they require invasive recording methods that cannot be applied in humans.

When asked whether understanding the roles of sleep cycles and memory could lead to future sleep-hacks, a la thePsycho-phone,Andrillonsaid, We are in the big unknown. But, he noted, sleep is not just about memory. Trying to hijack the recommended seven-plus hours of sleep could disrupt normal brain function. Which is to say, even if you could learn French while asleep, it mightultimately do more harm than good. I would be very cautious about the interest in this kind of learning, he said, whether this is detrimental to the other functions of sleeping.

Read more:

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Meet the scientist who dreams of fixing your sleep

Dear Science: How do I stop snoring?

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Your brain can form new memories while you are asleep … – Washington Post

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Local Doppler radar down through end of August – WDBJ7

The Doppler radar located in Blacksburg, Virginia was shut down August 1st for a nation wide project called the Service Life Extension Program, or SLEP.

The WSR-88D or NEXRAD radars were built with a service life of 20 years. The program started over 20 years ago and the purpose of the SLEP program is to bring the radar up to date with the newest technology. This upgrade would extend the life of the radar into the 2030s.

After installation of the new hardware and software, engineers ran test on the radar before placing it into operation and found a larger problem — a cracked bearing on the main gear that moves the radar. The unit was immediately turned off.

To repair the bearing and the bull gear the entire dome and 28 foot radar dish will need to be removed. This will require a 6 person team and heavy equipment to make the repairs. The team is currently doing the same repair in Ohio and will make their way to Blacksburg next week. The work is schedule to be complete by August 30.

In the meantime, multiple radar sites can cover our area and will be unnoticeable to app or website users. The NWS can attempt to run the radar in a time of need is a tornadic storm or a tropical system were to impact the region before the engineering team starts the repair work.

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Local Doppler radar down through end of August – WDBJ7

Recommendation and review posted by Bethany Smith

The Growing World of Libertarian Transhumanism – The American Conservative

Transhumanists are curiosity addicts. If its new, different, untouched, or even despised, were probably interested in it. If it involves a revolution or a possible paradigm shift in human experience, you have our full attention. We are obsessed with the mysteries of existence, and we spend our time using the scientific method to explore anything we can find about the evolving universe and our tiny place in it.

Obsessive curiosity is a strange bedfellow. It stems from a profound sense of wanting something better in lifeof not being satisfied. It makes one search, ponder, and strive for just about everything and anything that might improve existence. In the 21st century, that leads one right into transhumanism. Thats where Ive landed right now: A journalist and activist in the transhumanist movement. Im also currently a Libertarian candidate for California Governor. I advocate for science and tech-themed policies that give everyone the opportunity to live indefinitely in perfect health and freedom.

Politics aside, transhumanism is the international movement of using science and technology to radically change the human being and experience. Its primary goal is to deliver and embrace a utopian techno-optimistic worlda world that consists of biohackers, cyborgists, roboticists, life extension advocates, cryonicists, Singularitarians, and other science-devoted people.

Transhumanism was formally started in 1980s by philosophers in California. For decades it remained low key, mostly discussed in science fiction novels and unknown academic conferences. Lately, however, transhumanism seems to be surging in popularity. What once was a smallish band of fringe people discussing how science and technology can solve all humanitys problems has now become a burgeoning social mission of millions around the planet.

At the recent FreedomFest, the worlds largest festival on liberty, transhumanism was a theme explored in numerous panels, including some I had the privilege of being on. Libertarian transhumanism is one of the fastest growing segments of the libertarian movement. A top priority for transhumanists is to have freedom from the government so radical science experiments and research can go on undisturbed and unregulated.

So why are so many people jumping on the transhumanist bandwagon? I think it has to do with the mishmash of tech inundating and dominating our daily lives. Everything from our smartphone addictions to flying at 30,000 feet in jet airplanes to Roombas freaking out our pets in our homes. Nothing is like it was for our forbearers. In fact, little is like it was even a generation ago. And the near future will be many times more dramatic: driverless cars, robotic hearts, virtual reality sex, and telepathy via mind-reading headsets. Each of these technologies is already here, and in some cases being marketed to billions of people. The world is shifting under our feetand libertarian transhumanism is a sure way to navigate the chaos to make sure we arrive at the best future possible.

My interest in transhumanism began over 20 years ago when I was a philosophy and religion student at Columbia University in New York City. We were assigned to read an article on life extension techniques and the strange field of cryonics, where human beings are frozen after theyve died in hopes of reviving them with better medicine in the future. While Id read about these ideas in science fiction before, I didnt realize an entire cottage industry and movement existed in America that is dedicated to warding off death with radical science. It was an epiphany for me, and I knew after finishing that article I was passionately committed to transhumanism and wanted to help it.

However, it wasnt until I was in the Demilitarized Zone of Vietnam, on assignment for National Geographic Channel as a journalist, that I came to dedicate my life to transhumanism. Walking in the jungle, my guide tackled me and I fell to the ground with my camera. A moment later he pointed at the half-hidden landmine I almost stepped on. Id been through dozens of dangerous experiences in the over 100 countries I visited during my twenties and early thirtieshunting down wildlife poachers with WildAid, volcano boarding in the South Pacific, and even facing a pirate attack off Yemen on my small sailboat where I hid my girlfriend in the bilge and begged masked men with AK47s not to shoot me. But this experience in Vietnam was the one that forced a U-turn in my life. Looking at the unexploded landmine, I felt like a philosophical explosive had gone off in my head. It was time to directly dedicate my skills and hours to overcoming biological human death.

I returned home to America immediately and plunged into the field of transhumanism, reading everything I could on the topic, talking with people about it, and preparing a plan to contribute to the movement. I also began by writing my libertarian-minded novel The Transhumanist Wager, which went on to become a bestseller in philosophy on Amazon and helped launched my career as a futurist. Of course, a bestseller in philosophy on Amazon doesnt mean very many sales (theres been about 50,000 downloads to date), but it did mean that transhumanism was starting to appear alongside the ideas of Plato, Marx, Nietzsche, Ayn Rand, Sam Harris, and other philosophers that inspired people to look outside their scope of experience into the unknown.

And transhumanism is the unknown. Bionic arms, brain implants ectogenesis, artificial intelligence, exoskeleton suits, designer babies, gene editing tech. These technologies are no longer part of some Star Trek sequel, but are already here or being worked on. They will change the world and how we see ourselves as human beings. The conundrum facing society is whether were ready for this. Transhumanists say yes. But America may not welcome that.

In fact, the civil rights battle of the century may be looming because of coming transhumanist tech. If conservatives think abortion rights are unethical, how will they feel about scientists who want to genetically combine the best aspects of species, including humans and animals together? And should people be able to marry their sexbots? Will transhumanist Christians try to convert artificial intelligence and lead us to something termed a Jesus Singularity? Should we allow scientists to reverse aging, something researchers have already had success with in mice? Finally, as we become more cyborg-like with artificial hips, cranial implants, and 3D-printed organs, should we rename the human species?

Whether people like it or not, transhumanism has arrived. Not only has it become a leading buzzword for a new generation pondering the significance of merging with machines, but transhumanist-themed columns are appearing in major media. Celebrity conspiracy theorists like Mark Dice and Alex Jones bash it regularly, and even mainstream media heavyweights like John Stossel, Joe Rogan, and Glenn Beck discuss it publicly. Then theres Google hiring famed inventor Ray Kurzweil as lead engineer to work on artificial intelligence, or J. Craig Ventures new San Diego-based genome sequencing start-up (co-founded with Peter Diamandis of the X-Prize Foundation and stem cell pioneer Robert Hariri) which already has 70 million dollars in financing.

Its not just companies either. Recently, the British Parliament approved a procedure to create babies with material from three different parents. Even President Obama, before he left office, jumped in the game by giving DARPA $70 million dollars to develop brain chip technology, part of Americas multi-billion dollar BRAIN Initiative. The future is coming fast, people around the world are realizing, and theres no denying that the transhumanist age fascinates tens of millions of people as they wonder where the species might go and what health benefits it might mean for society.

At the end of the day, transhumanism is still really focused on one thing: satisfying that essential addiction to curiosity. With science, technology, and a liberty-minded outlook as our tools, the species can seek out and even challenge the very nature of its being and place in the universe. That might mean the end of human death by mid-century if governments allow the science and medicine to develop. It will likely mean the transformation of the species from biological entities into something with much more tech built directly into it. Perhaps most important of all, it will mean we will have the chance to grow and evolve with our families, friends, and loved ones for as long as we like, regardless how weird or wild transhumanist existence becomes.

Zoltan Istvan is the author of The Transhumanist Wager, and a Libertarian candidate for Governor in California.

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The Growing World of Libertarian Transhumanism – The American Conservative

Recommendation and review posted by Bethany Smith

Genetic home testing: why it’s not such a great guide to your ancestry or disease risk – The Conversation UK

Genetic variation is an unavoidable feature of life. As a consequence of this and unless you are an identical twin you are genetically unique. You have a distinctive combination of genetic variants, almost all of which are shared with your parents, although shuffled into new combinations. With few exceptions, all of your characteristics, such as hair colour, height, ear wax quality, and disease susceptibility, are influenced by your genetics.

As a consequence, increasing numbers of people want to learn more about their genetic inheritance, which can be driven by a desire to find out whether they are carriers of, or possibly sufferers from, serious genetic disease. Other people are interested in what a genetic test might be able to tell them about their ancestry. Some people are simply curious.

Direct-to-consumer genetic testing companies which provide genetic tests based on a saliva sample that you send in from home have proliferated in response to this demand in recent years. Big names include 23andMe, AncestryDNA and Orig3n.

But what can these tests actually tell you about disease risk? And how reliable are ancestry predictions derived from an analysis of your genetic makeup?

The key problem in understanding both disease risk and genetic ancestry when interpreting someones results is that our knowledge of both is mostly based on studying large populations. The person taking the test will want specific, personal predictions but extrapolating results from population level measures of genetic risk to that of the individual isnt straightforward.

For instance, our understanding of the genetic basis of a given human trait comes from making statistical associations between the trait and a specific genetic variant. For rare genetic diseases, such as Huntingtons Disease, the connection between the disease and the genetic variant that causes it is very strong, so disease risk can be predicted with some confidence. For more common diseases, or traits such as height and BMI, however, there are hundreds of genetic variants that make relatively small contributions to these. Predictions of individual risk in these cases then becomes extremely hard.

The difficulty in making personal disease risk predictions becomes more apparent when you discover that the most comprehensive direct-to-consumer genetic tests look for about 700,000 genetic variants. This sounds like a lot, but it is actually only a fraction of the genetic diversity that exists in each person. And without knowing the full complement of genetic variants, risk predictions for most diseases will be inaccurate. What this means is that at the level of an individual, our understanding of the genetic basis for most human traits is incomplete, complex and messy.

Ultimately, what most people will learn from such genetic tests will be of limited health care value; you are much better off putting the money you would spend on a genetic test towards a gym membership, or a pair of trainers something that will have a proven effect on health and well-being.

Our individual genetic diversity is obviously influenced by our ancestry, and some genetic testing companies will offer to connect you to famous people in the past, or to particular locations. However, here again we encounter the problem of inferring individual narratives from population-based information as what we know about human ancestry comes from studying the genetic histories of populations, not individuals.

And as your genome the complete set of your genes is a patchwork of your ancestors DNA, each segment will have a different pattern of ancestry. This is why scientists have called statements about your ancestry based on genetic testing genetic astrology. Put simply, what genetic testing says about your ancestry may seem personal and precise, but could be true of thousands, possibly millions, of other people.

For instance, if we assumed European ancestry then I wouldnt need to charge your credit card or ask for a cheek swab to tell you that Abd-ar-Rahman III, the founder of the Caliphate of Crdoba (and descendant of the Prophet Mohammed), is your ancestor. Recent studies suggest that every person living in Europe about 1,000 years ago, if they left descendants, is an ancestor of every European today.

This is consistent with an earlier global mathematical modelling approach, which made the more startling prediction that every individual alive several thousand years ago (2,000-5,000BC, depending on the parameters of the model), assuming they left descendants, was the ancestor of every living person today. Everyone. So, without doing a genetic test, I can tell you, whoever you are, that you are descended from the people who founded Mesopotamia, as well as the rice farmers living on the banks of the Yangtze river. And theyre my ancestors, too.

Ultimately, genetic home testing can be an entertaining way of engaging with your genome. However, if you are wanting to get a better perspective on your personal disease risk, these kits are no substitute for professional healthcare services. And they are unlikely to tell you anything profound about your ancestral connections to famous people or locations in history.

This article was amended on August 9 to replace the emperor Justinian I with Abd-ar-Rahman III.

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Genetic home testing: why it’s not such a great guide to your ancestry or disease risk – The Conversation UK

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Researchers call on province to fund genetic testing for cerebral … – Toronto Star

Julian Cappelli, 16, who has cerebral palsy, enjoys some time with his mother Donna in their Toronto home. A new study from Holland Bloorview and SickKids has found there’s a stronger genetic link in cases of hemiplegic cerebral palsy, the most common form of CP, than previously thought.(Bernard Weil / Toronto Star) | Order this photo

By Ainslie CruickshankStaff Reporter

Tues., Aug. 8, 2017

Researchers say genetic testing should be standard practice when diagnosing cerebral palsy after a new study found that genetic variations could be a factor in hemiplegic cerebral palsy, the most common form of the motor disability.

Standardizing a genetic workup for children with cerebral palsy, though, would depend on government funding.

There should be genetic testing that happens as soon as possible; thats the take home message in this study, said Stephen Scherer, director of the Centre for Applied Genomics at SickKids and one of the authors of the study, which was done by researchers at Holland Bloorview Kids Rehabilitation Hospital and the Hospital for Sick Children.

For families, genetic testing could help explain why their child developed cerebral palsy. For researchers, it offers new directions for research aimed at preventing and treating the condition, which affects three out of every 1,000 children born in Canada each year.

The impact of this I think is going to be very, very significant, Scherer said.

Sitting in the kitchen of his east-end home, Julian Cappelli, 16, is wearing a red Toronto FC T-shirt. Soccers his favourite sport and TFC is his favourite team.

Julian has quadriplegic cerebral palsy, which affects all four of his limbs. His family received that diagnosis when he was one year old. Julian said if he had the chance to have genetic testing done, hed take it. Even 15 years later, he wants to know why he has a motor condition that, for him, means he wont get the chance to try for a professional soccer career.

His mom, Donna, doesnt spend much time anymore wondering why he has cerebral palsy. Were kind of just moving forward and dealing with what we have, she said.

But that doesnt mean shes not excited about the new research. She is.

She remembers what it was like 15 and 16 years ago, wanting answers.

It was so overwhelming and you do want to know: Why did this happen? Was it something that you did?

Its good to see that theyre still looking into the reasons why this is happening and trying to help prevent it and make these kids lives better, she said.

Cerebral palsy is the most common physical disability in children, said Dr. Darcy Fehlings, a senior clinician scientist at Holland Bloorview specializing in cerebral palsy research and another of the studys authors.

Although it is a permanent disability that affects childrens motor movements, it manifests itself differently in every child. In some cases, children may have difficulty using their hands or walking. In other cases, they may have trouble communicating or might need to use a wheelchair.

Though cerebral palsy is often thought to be caused when a baby doesnt get enough oxygen before, during or after birth, causing damage to their brain or other organs, by stroke or infection in a childs brain, researchers found a significant genetic link in hemiplegic cerebral palsy, which affects only one side of the body.

The study, published in the Genetics in Medicine journal and promoted by Nature.com, outlines the results of DNA analysis on 97 children with hemiplegic cerebral palsy and their parents compared with more than 10,000 population control samples.

The researchers found that structural variations to the DNA that affect the genes for brain development and function were factors in 20 per cent of hemiplegic cerebral palsy cases and probably the major cause in five per cent of cases.

We didnt even look for this before, Scherer said. In retrospect, we should have.

Diagnosing cerebral palsy can be difficult, especially in children, who arent fully developed. In about 10 per cent of cases, children diagnosed with cerebral palsy may actually have a different disorder, he said. A genetic workup can help confirm the diagnosis and make sure the best treatment plan is developed.

In a statement, a spokesperson for the Ministry of Health and Long-Term Care said the ministry recognizes the importance of genetic testing in providing patient care.

Generally, with new or emerging tests, the test would undergo evidence-based evaluation that would guide decisions whether or not the test is used as part of routine standard of care, and making the best use of public healthcare resources.

Genetic testing is already used to help diagnose and develop treatment for cystic fibrosis and muscular dystrophy. More recently, it is being used to help diagnose and develop treatment for autism spectrum disorder.

Theres way more data here than we had in our early autism studies, Scherer said.

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Researchers call on province to fund genetic testing for cerebral … – Toronto Star

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Pfizer to invest $100M in Sanford gene therapy operation, add jobs … – WRAL Tech Wire

Updated Aug. 8, 2017 at 7:02 a.m.

Published: 2017-08-07 16:07:00 Updated: 2017-08-08 07:02:05

Sanford, N.C. Pharmaceutical giant Pfizer Inc. plans to invest $100 million in its Sanford operations as part of a push into gene therapy, officials said Monday.

The effort builds on a technology developed at the University of North Carolina at Chapel Hill and will create 40 jobs in Sanford.

“Pfizer is proud to further expand our presence in North Carolina, particularly as we build our leadership in gene therapy,” Lynn Bottone, site leader at Pfizer Sanford, said in a statement. “We look forward to the next phase of this expansion as we build a clinical and commercial manufacturing facility.”

Preliminary work on the expansion and initial hiring have already begun. The 230-acre campus employs about 450 people, reports the N.C. Biotechnology Center.

Gene therapy is a potentially transformational technology for patients that involves highly specialized, one-time treatments to address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patients cells to compensate for a defective or missing gene.

Last year, Pfizer acquired Bamboo Therapeutics Inc., a privately held biotechnology company in Chapel Hill focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. Pfizer also committed $4 million to support postdoctoral fellowships in North Carolina universities for training in gene therapy research.

“We are excited that Carolinas research will improve lives and create jobs for North Carolinians,” UNC-Chapel Hill Chancellor Carol Folt said in a statement. “This is a perfect example of how placing innovation at the center of our university creates new opportunities. We are proud to be a part of the technologies, expertise and infrastructure that went into Bamboo Therapeutics and helped make this manufacturing expansion in Sanford possible. Gene therapy is a strength at Carolina, and we look forward to continue to help advance this industry.”

Pfizer is also expanding a drug-manufacturing facility in Rocky Mount that it acquired from Hospira in 2015. The $190 million project will add 65,000 square feet of sterile injectable facilities but will not create any new jobs. The plant employs about 300 people.

Gov. Roy Cooper visited Pfizers Sanford facility last week to take a tour and meet with the companys senior leaders.

“North Carolina is one of the few places in the country with the biotech resources to take an idea all the way from the lab to the manufacturing line,” Cooper said in a statement. “Pfizers investment in Lee County is a prime example of how North Carolinas world-class universities and cutting-edge industries work together to move our state forward.”

Pfizer qualified for a performance-based grant of $250,000 from the One North Carolina Fund, which provides state assistance matched by local governments to help attract economic investment and create jobs. Companies receive no money upfront and must meet job and investment targets to obtain payment.

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Pfizer to invest $100M in Sanford gene therapy operation, add jobs … – WRAL Tech Wire

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Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

By Ben Hirschler

LONDON (Reuters) – The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.

Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.

In the past five years, European regulators have approved two gene therapies – the first of their kind in the world, outside China – but only three patients have so far been treated commercially.

UniQure’s Glybera, for a very rare blood disorder, is now being taken off the market given lack of demand.

The future of GlaxoSmithKline’s Strimvelis for ADA-SCID – or “bubble boy” disease, where sufferers are highly vulnerable to infections – is uncertain after the company decided to review and possibly sell its rare diseases unit.

Glybera, costing around $1 million per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.

“It’s disappointing that so few patients have received gene therapy in Europe,” said KPMG chief medical adviser Hilary Thomas. “It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment.”

These first two therapies are for exceptionally rare conditions – GSK estimates there are only 15 new cases of ADA-SCID in Europe each year – but both drugs are expected to pave the way for bigger products.

The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.

Spark Therapeutics hopes to win U.S. approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get a U.S. go-ahead as early as next month for its gene-modified cell therapy against leukaemia – a variation on standard gene therapy.

At the same time, academic research is advancing by leaps and bounds, with last week’s successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.

Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe’s first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera’s patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.

He expects Spark will do better. It plans to have treatment centers in each country to address a type of blindness affecting about 6,000 people around the world.

Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400 million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.

A one-time cure, even at $1 million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.

But gene therapy companies – which also include Bluebird Bio, BioMarin, Sangamo and GenSight – may need new business models.

One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.

“In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant,” Marrazzo said.

Haemophilia, a hereditary condition affecting more than 100,000 people in markets where specialty drugmakers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.

Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

Shire CEO Flemming Ornskov – who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder – sees both the opportunities and the difficulties of gene therapy.

“Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model.”

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Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

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