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11 Incredible Things CRISPR Has Helped Us Achieve in 2017 – Futurism

In BriefIn 2017, the hot new gene editing technique CRISPR has made unparalleled advancements in gene engineering. Here are 11 highlights.

The CRISPR//Cas9 gene editing tool has quickly earned a reputation as a revolutionary technology, and its merits support the clout. This year has, in fact, seen so many CRISPR-related breakthroughs that its worthwhile to take a step backand take in all of the many accomplishments.

1. This week, circulating reports about the successful application of gene-editing human embryos in the US were confirmed by a research paper published in Nature. The researchers corrected one-cell embryo DNA to remove the MYBPC3 gene known to cause hypertrophic cardiomyopathy (HCM), a heart disease that affects 1 in 500 people.

2. This year, scientists successfully used gene editing to completely extract HIV from a living organism, with repeated success across three different animal models. In addition to the complete removal of the virus DNA, the team also prevented the progress of acute latent infection.

3. Semi-synthetic organisms were developed by breeding E.coli bacteria with an anomalous six-letter genetic code, instead of the normal four-base sequence. Additional gene editing was implemented to ensure that the new DNA molecules were not identified as an invasive presence.

4. The CRISPR method successfully targeted the command center of cancer called the hybrid fusion which leads to abnormal tumor growths. A cut-and-paste method allowed the creation of a cancer-annihilating gene that shrinks tumors in mice carrying human prostate and liver cancer cells.

5. Scientists also slowed the growth of cancerous cells, by targeting Tudor-SN, a key protein in cell division. Its expected that this technique could also slow the growth of fast-growing cells.

6. Gene editing techniques have also made superbugs kill themselves. By adding antibiotic resistant gene sequences into bacteriophage viruses, self-destructive mechanisms are triggered which protect bacteria.

7. Gene editing may even make mosquito-born diseases an extinct phenomenon. By hacking fertility genes, scientists have gained the ability to limit the spread of mosquitoes a success they credit to CRISPRs ability to make multiple genetic code changes simultaneously.

8. Using CRISPR, researchers have edited out Huntingtons disease from mice, pushing the symptomatic progression of the condition into reverse. Experts expect this promising technique to be applied to humans in the near future.

9. Outside of the medical field, CRISPR might also provide a more abundant and sustainable biofuel. By connecting several gene-editing tools, scientists engineered algae that produce twice the biofuel material as wild (or natural) counterparts.

10. Very recently, the first-ever molecular recorder was developed a gene editing process that encodes a film directly into DNA code and with this ability, scientists embedded information into an E.coli genome.

11. Last but not least, and on the macro-scale, the US Defense Advanced Research Projects Agency (DARPA) invested $65 million in a project called safe genes, designed to improve the accuracy and safety of CRISPR editing techniques. In addition to serving the public interest of avoiding accidental or intentional (cue ominous music) misuse, the seven research teams will remove engineered genes from environmentsto return them to baselinenatural levels.

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11 Incredible Things CRISPR Has Helped Us Achieve in 2017 – Futurism

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MilliporeSigma to Be Granted European Patent for CRISPR Technology – Genetic Engineering & Biotechnology News

“Significant and Exciting”

This is a significant and exciting decision by the EPO, and we view this announcement as recognition of MilliporeSigma’s important contributions to the genome-editing field, MilliporeSigma CEO Udit Batra, Ph.D., said. This patent provides protection for our CRISPR technology, which will give scientists the ability to advance treatment options for the toughest medical challenges we face today.

MilliporeSigma also predicted that it would be awarded patents for the technology in other countries as well.

The European patent to MilliporeSigma comes five months after the EPO announced an intention to grant a patent broadly covering CRISPR technology to Emmanuelle Charpentier, Ph.D., a director at the Max-Planck Institute in Berlin, together with the University of California (UC), and University of Vienna.

The patent consisted of broad claims directed to the CRISPR/Cas9 single-guide gene-editing system for uses in both noncellular and cellular settings, including in cells from vertebrate animals such as human or mammalian cellsas well as composition claims for use in any setting, including claims for use in a method of therapeutic treatment of a patient. The technology has been licensed to companies that include CRISPR Therapeuticswhose co-founders include Dr. Charpentierand ERS Genomics, both of which announced the EPO decision.

Dr. Charpentier, UC, and University of Vienna are in a legal battle royal with the Broad Institute of MIT and Harvard over who invented the gene-editing platform. Late last month, the European patent holders filed a brief with the U.S. Court of Appeals for the Federal Circuit seeking to reverse the February 15 decision by the Patent Trial and Appeal Board (PTAB). The PTAB found no interference in fact between 12 patents related to CRISPR technology that list as inventor Feng Zhang, Ph.D., of the Broad, and a patent application by Dr. Charpentier and Jennifer Doudna, Ph.D., of UC Berkeley.

The #CRISPR #patent situation in Europe just got a LOT more complicated, tweeted Jacob S. Sherkow, J.D., associate professor at the Innovation Center for Law and Technology, New York Law School, who has closely followed the CRISPR legal wrangle, on August 5.

Until now, he tweeted, the EPO granting of a patent to Dr. Charpentier, UC, and the University of Vienna didn’t mean Zhang couldn’t get his. Now, it’s unclear.

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MilliporeSigma to Be Granted European Patent for CRISPR Technology – Genetic Engineering & Biotechnology News

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CRISPR Screen Identifies Top 100 Essential Genes for Cancer … – Genetic Engineering & Biotechnology News

Existing cancer immunotherapies harness T cells that recognize and home in on tumor-specific targets and kill the cancer cells. Immunotherapy using checkpoint inhibitors, for example, disconnects immune system restraints so that the T cells can attack the cancer cells. Other forms of immunotherapy, including cancer vaccines and adoptive T-cell therapy, increase the numbers of cytotoxic T cells that are mobilized to the tumor.

Immunotherapy can be highly effective against advanced cancers in some patients, but in other cases treatment doesnt work. To try to understand the genetic basis of these differing responses, scientists at the U.S. National Institutes of Health developed a genome-scale CRISPR/Cas9 screen that allowed them to knock out every single gene in a melanoma cell line and then systematically test each gene for its effect on T-cell responses against the melanoma. Using this “two-cell type” (2CT)-CRISPR assay, the researchers, led by Shashank Patel, Ph.D., and Nicholas Restifo, M.D., who is a senior investigator with NCI’s Center for Cancer Research, identified more than 100 “essential” genes that were required in the melanoma line for T cells to effectively engage with and kill the cells. When these genes were knocked out, the tumor cells were more able to resist exposure to T cells that had been engineered specifically to recognize tumor-associated antigens.

The researchers published their studies in the August 7 issue of Nature, in a paper entitled “Identification of Essential Genes for Cancer Immunotherapy. The NIH team worked in close collaboration with Feng Zhang, Ph.D., from MIT, one of the original innovators of the CRISPR technology. Neville Sanjana, Ph.D., from the New York Genome Center and New York University was co-first author of the study.

CRISPR/Cas9 screens have previously been used to identify genes that play key roles in cancer cell proliferation, drug resistance, and metastasis, the authors point out. To identify which genes in tumors are requisite for the “effector function of T cells,” the team developed the 2CT-CRISPR assay, consisting of human T cells as effectors and melanoma cells as targets, to evaluate the effects of individual gene knockouts on cancer cell susceptibility to T-cell killing. Many of the hundred or so genes identified were directly involved in cytokine release, or in antigen processing and presentation, but dozens of the genes identified were not known to be required for cytotoxic T-cell-based immunotherapy.

This indicated that the loss of genes that T cells need to kill cancer cells might be at least partially responsible for why immunotherapy fails in some patients, Dr. Restifo suggested to GEN. However, we were really surprised to find dozens of tumor genes that had major impacts on tumor cell survival, which hadnt previously been linked with the ability of T cells to kill target cancer cells. Exploring potentially new signaling pathways mediated by these genes could help us to understand how T cells interact with cancer cells to bring about cell death, and how cancers can evade the immune system.

With their list of the 100 most necessary tumor genes in hand, the researchers looked at the gene expression profiles of nearly 11,500 human tumors from The Cancer Genome Atlas (TCGA) database, across 36 tumor types, to see whether loss of these tumor genes associated with decreased cytolytic activity. The analysis identified a set of 19 genes that correlated with cytolytic activity across most of the cancer types. Ten of these were inducible by interferon- (IFN), which indicated that they might be upregulated in cancers because of increased T-cell mobilization. Loss of expression of these 19 genes within tumors could diminish or extinguish the presentation of tumor antigens (including HLA-A, HLA-F, B2M, TAP1 and TAP2); T-cell co-stimulation (ICAM1, CLECL1, LILRA1 and LILRA3); or cytokine production and signaling (JAK2 and STAT1) in the tumor microenvironment that drive infiltration and activation of T cells, and thus serve as a principal mechanism in immune evasion, the researchers write in their published paper.

The team next focused on one gene, APLNR, which codes for the apelin receptor, a G protein-coupled receptor (GPCR) that hadnt previously been associated with T-cell killing of cancer, but which is known to be mutated in a number of different tumor types. They identified seven different mutations in this gene in the genetic makeup of metastatic melanoma and lung cancer patients who had failed therapy using immune checkpoint inhibitors.

When the team introduced these same mutations into a melanoma cell line, the cancer cells were more resistant to T-cell attack. And when they injected engineered melanoma cells that lacked the APLNR gene into experimental mice, the resulting tumors were resistant to checkpoint inhibitor therapy and didnt respond as well to adoptive cell transfer as tumors with a normal APLNR gene. Dr. Patel concluded that these data demonstrate that APLNR loss reduces the effectiveness of T-cell-based cancer therapies, including immune checkpoint blockade and ACT.

More work will be needed to validate the relevance of all the genes identified by the studies, Dr. Restifo stressed to GEN. We hope that this comprehensive list of genes will act as a blueprint for further study so that we can better understand tumor resistance to cancer therapies that hinge on T-cell attack. Looking at mutations in these genes in individual patients who failed immunotherapy may enable physicians to devise the most appropriate treatments for each individual patient, according to their essential gene profiles. More importantly, we are working toward developing new approaches to cancer therapy that help more patients with cancer.

The study findings also indicate that the success of cancer immunotherapy depends on the interplay between a far greater number of genes than previously thought, Dr. Restifo commented. A deeper understanding of how T cells interact with potential target cells could also help us to develop more effective treatments for infectious and autoimmune diseases.

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CRISPR Screen Identifies Top 100 Essential Genes for Cancer … – Genetic Engineering & Biotechnology News

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CRISPR Skin Grafts Could Replace Insulin Shots For Diabetes – Futurism

The Potential of CRISPR

The potential of the gene editing toolCRISPRjust seems to keep growing and growing, and the latest experimental use of the technology is creating skin grafts that trigger the release of insulin and help manage diabetes.

Researchers have successfully tested the idea with mice that gained less weight and showed a reversed resistance to insulin because of the grafts (high insulin resistance is a common precursor to type 2 diabetes).

In fact, the team from the University of Chicago says the same approach could eventually be used to treat a variety of metabolic and genetic conditions, not just diabetes its a question of using skin cells to trigger different chemical reactions in the body.

We didnt cure diabetes, but it does provide a potential long-term and safe approach of using skin epidermal stem cells to help people with diabetes and obesity better maintain their glucose levels,says one of the researchers, Xiaoyang Wu.

If youre new to theCRISPR(Clustered Regularly Interspaced Short Palindromic Repeats) phenomenon, its a new and innovative way of editing specific genes in the body, using a biological copy and paste technique: it can doeverything fromcut out HIV virus DNA to slow thegrowth of cancer cells.

For this study, researchers used CRISPR to alter the gene responsible for encoding a hormone calledglucagon-like peptide-1(GLP-1), which triggers the release of insulin and then helps remove excess glucose from the blood.

Type 2 diabetescomes about due to a lack of insulin, also known as insulin resistance.

Using CRISPR, the GLP-1 gene could be tweaked to make its effects last longer than normal. The result was developed into skin grafts that were then applied to mice.

Around 80 percent of the grafts successfully released the edited hormone into the blood, regulating blood glucose levels over four months, as well as reversing insulin resistance and weight gain related to a high-fat diet.

Significantly, its the first time the skin graft approach has worked for mice not specially designed in the lab.

This paper is exciting for us because it is the first time we show engineered skin grafts can survive long term in wild-type mice, and we expect that in the near future this approach can be used as a safe option for the treatment of human patients,says Wu.

Human treatments will take time to develop but the good news is that scientists are today able to grow skin tissue very easily in the lab using stem cells, so that wont be an issue.

If we can make it safe, and patients are happy with the procedure, then the researchers say it could be extended to treat something likehaemophilia, where the body is unable to make blood clots properly.

Any kind of disease where the body is deficient in specific molecules could potentially be targeted by this new technique. And if it works with diabetes, it could be time to say goodbye to needles and insulin injections.

Other scientists who werent directly involved in the research, including Timothy Kieffer from the University of British Columbia in Canada, seem optimistic.

I do predict that gene and cell therapies will ultimately replace repeated injections for the treatment of chronic diseases, Kieffer told Rachel Baxter atNew Scientist.

The findings have been published inCell Stem Cell.

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Genetic Testing Tells More Than Just Ancestry – Springfield Business Journal

Dynamic DNALaboratories

Street Address:

2144 E. Republic Rd. Suite B204

Springfield, MO 65804

Phone: 417-319-1047

Fax: 417-319-7142

WebAddress: http://www.dynamicdnalabs.com

Top Executives: Austin OReilly (Owner/Senior Scientist)

Dr. Christopher Gilbert (Medical Director)Year Founded: 2015

Number ofEmployees: 6

Product or Service:

Clinical and Direct-to-Consumer Genetic Testing

Consumer demand for genetic-makeup testing is growing, not only to give clues about the past but also to learn how to improve the future. The CDC has identified that genetic testing has the potential to help prevent common disease and improve the health of individuals and populations.Dynamic DNA Laboratories, owned by Austin OReilly, is a genetic testing facility in Springfield, MO, working with individuals and businesses locally, as well as across the U.S. and eight other countries.

The most well known use of DNA testing is for tracing ancestry and paternity.But the fast emerging use is to develop personalized diet and exercise plans, so individuals can achieve their optimum fitness level. This service is available to companies as part of their wellness plan in addition to individuals motivated to improve their health.

Dynamic DNA provides the following services:

DNA Fitness Testing

DNA Nutrition Testing

Personalized Medicine Reporting

DNA Ancestry Testing

Paternity Testing

DNA/Microscopic artwork

Biomedical research projects (upon request)

Company Partnerships

Dynamic DNA recently partnered with Prime Trucking and Trinity Healthcare to be a part of Primes corporate wellness program for their employees, offering Nutrition and Fitness Panels to their 7,000-plus staff members and truck drivers.

Local Testing

Most genetic testing services outsource their testing but Dynamic DNA conducts all of its testing in-house at the Springfield laboratory.

This ensures the integrity of results and affords a quicker turnaround time. Where most labs take months to deliver results, Dynamic DNA provides results in less than two weeks.

Low Cost Option

We believe that everyone should have the opportunity to participate in DNA testing, so our number one goal is to make advanced genetic testing accessible to everyone by keeping our price points at the lowest in the industry, says Austin OReilly, owner and senior scientist.

Easy-to-Understand Results

Some DNA results can also be a challenge to read, let alone understand. Dynamic DNA removes this hindrance by presenting results in a clean and concise manner, and providing clients direct access to the scientists, genetic consultants or doctors on staff, should they have any questions or concerns.

Whether someone is looking to learn more about their health and predispositions, how to improve their performance, what medications work best for them, or where their family came from, we are happy to help, says OReilly.

Dynamic DNA recently had a peer-reviewed article published in the Journal of Nutrition and Health on research conducted for a local product called Re:iimmune. The company will be featured on The Doctor Show on PBS in September.

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Genetic Testing Tells More Than Just Ancestry – Springfield Business Journal

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Study: Most Newborns with Epilepsy Benefit from Genetic Testing – Sleep Review

Because of genetic testing, Orion Maynards parents knew the cause of his epilepsy weeks after he was born. The results influenced his treatment, qualified him for immediate intervention services, and led to the discovery that future siblings had a 50% chance of being born with the same condition.

Most newborns with epilepsy like Orion would benefit from genetic testing because the majority of cases are linked to identifiable genetic causes, finds a study led by University of Michigan C.S. Mott Childrens Hospital.

In the study, among 29 babies with epilepsy not linked to congenital brain malformations, 83% had a genetic cause. Research published in Neurology analyzed data from the Neonatal Seizure Registry that includes enrollees from 7childrens hospitals, including Mott.

A parallel, multisite study in JAMA Pediatrics, for which Mott was a major contributor, found similar results for children under age 3.

The younger the epilepsy begins, the more likely we are to find a genetic cause that may help with treatment, says Renee Shellhaas, MD, a pediatric neurologist at Mott and the lead author of the Neurology work, in a release.

Parents and physicians always want to know why a specific child develops epilepsy. Genetic testing benefits our youngest epilepsy patients and their families because it can not only assist with their care and prognosis but also in connecting families with condition-specific support groups and specialists, access to research studies, and counseling about family planning.

Genetic testing involves a simple blood test but is not always approved by health insurance providers.

These findings reinforce that genetic tests are incredibly valuable for childhood epilepsy, yet we still struggle getting them covered because of the cost, Shellhaas says.

We need to work on a broader policy level to increase access to appropriate genetic testing for children with epilepsy. Finding the reason for a childs epilepsy provides comfort and closure for families, helps them to connect with other families, can allow for tailored treatment, and ends the diagnostic odyssey faced by so many of our patients. Genetic testing is a standard of care for children with global developmental delays; our research results suggest we should consider a similar standard for children with epilepsy.

For Orions parents, Lindsay and Robin, the test brought important answers after their baby boy began having back-to-back seizures at just a few days old.

Orion was referred to Mott where he was diagnosed with epilepsy. A genetic test easily found the source: a missing piece of one of his chromosomes (16p13.11). A few months later, both parents had the same test and learned Lindsay was a carrier.

The test also helped with immediate eligibility for Early on Michigan, which offers early intervention and services for families with children under 3 experiencing developmental delays, disabilities, or special needs. Now 3 years old, Orion has benefited from several of the programs resources, including speech, physical, and occupational therapy.

Without the testing, we may not have qualified for the program until he was closer to 1and a half or 2when speech and motor development delays were noticeable, Lindsay says. Orion has been getting services since he was 3months old.

Shellhaas findings come on the heels of a separate study she also led, which analyzed how clinicians treat epilepsy in children under three across 17 pediatric epilepsy centers in the United States. The work, published in Pediatric Neurology, found that even though there are 20 different antiseizure medications, the drug levetiracetam is most commonly prescribed.

She says this consistency in practice is surprising as there are currently no evidence-based guidelines on the preferred approach to treating early-life epilepsy.

Even though clinicians seem to have informally agreed on the best way to start treating early life epilepsy, that doesnt mean it is evidence-based treatment, she says. We dont have that much data on how a lot of medicines influence the developing brain. We are working hard to change that.

Factors in the preference toward levetiracetam could include that the medication has a generic form, doesnt have interactions with other drugs, can be taken as a liquid or be given through an IV. Still, more study on its efficacy is needed.

We have to keep working to find the best way to diagnose and tailor treatment as early as possible in a childs life, Shellhaas says. If you can improve the way a childs neurodevelopment begins early on, youve influenced his or her ability to grow, develop and participate in society. That not only benefits the child but the whole family.

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Study: Most Newborns with Epilepsy Benefit from Genetic Testing – Sleep Review

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You’re getting a DNA test start-up Clear Genetics is building chatbots to help you understand the results – CNBC

George Frey | AFP | Getty Images

A lab technician at Myriad Genetics in Salt Lake City, Utah.

Thousands of people are getting genetic tests, for everything from their cancer risk to their likelihood of passing on a disease to a child.

But many doctors aren’t adequately trained to interpret these results, or tell patients how to act on them. And genetic counselors — who do have that knowledge — are in short supply. There are only about 4,000 genetic counselors in the country today. That’s one for every 80,000 Americans. That means some patients have to wait months to get a consultation.

Start-up Clear Genetics, which launches this week after raising $2.5 million in financing, is trying to make genetic expertise more widely available.

The start-up has developed a conversational chatbot to guide a user through their results, collect information and review options for genetic testing, and answer questions about things like whether the test will be covered by insurance. If there’s a need for additional support, the patient can then schedule a consultation with a human expert via video or in-person.

“We’re finding that it’s working really well with patients,” said Moran Snir, Clear Genetics’ CEO, who was previously a software engineer with the Israeli military.

Clear Genetics is working with several large health systems in the United States to test out a beta version of its product.

“I think this is a very good use for AI,” said David Ledbetter, executive vice president and chief scientific officer at hospital network Geisinger Health System, in an interview with CNBC.

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You’re getting a DNA test start-up Clear Genetics is building chatbots to help you understand the results – CNBC

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Trump Administration Withdraws Proposed Obama Ban on Compensation for Bone Marrow – Reason (blog)

Marrow Drives

The Office of Management and Budget has withdrawn a proposed rule banning compensation for hematopoietic stem cells. In other words, you can get paid when someone harvests stem cells from your bone marrow.

Bone marrow transplantation is used to treat a variety of ailments, including aplastic anemia, sickle cell anemia, bone marrow damage during chemotherapy, and blood cancers such as leukemia, lymphoma, and multiple myeloma. In 1984, Congress passed the National Organ and Transplant Act, which outlawed compensation to the donors of solid organs like kidneys and livers. Oddly, the act also defined renewable bone marrow as a solid organ.

Originally, hematopoietic stem cells were obtained from bone marrow obtained by inserting a needle into donors’ hip bones. Researchers later developed a technique in which donors are treated with substance that overstimulates the production of hematopoietic stem cells, which then circulate in their bloodstreams. In a process similar to blood donation, the hematopoietic stem cells are then filtered from the donors’ blood. The red blood cells and plasma are returned to the donors.

More Marrow Donors, a California-based nonprofit, wanted to set up a system to encourage hematopoietic stem cell donations with $3,000 awards, in the form of scholarships, housing allowances, or gifts to charity. The Institute for Justice, a libertarian law firm, brought suit on their behalf, and in 2012 a federal appeals court sensibly ruled that the law’s ban on compensation for solid organ donations did not apply to stem cells obtained from donors’ bloodstreams. The Obama administration reacted by proposing a regulation defining stem cells obtained from blood as the equivalent of a solid organ.

Now the new administration has withdrawn the proposal.

“Banning compensation for donors would have eliminated the best incentive we havemoneyfor persuading strangers to work for each other,” Jess Rowes, a senior attorney with the Institute for Justice, say in a press release. “Predictably, the ban on compensation for blood stem cell donors created chronic shortages and waiting lists. During the past four years, thousands of Americans needlessly died because compensation for bone marrow donors was unavailable.”

The system of uncompensated donation is falling far short of meeting patient needs. As the Institute for Justice notes:

At any given time, more than 11,000 Americans are actively searching for a bone marrow donor. According to the New England Journal of Medicine, Caucasian potential donors are available and willing to donate about 51 percent of the time; Hispanic and Asian about 29 percent; and African-American about 23 percent. Caucasian patients can find a matching, available and willing donor about 75 percent of the time; Hispanic about 37 percent; Asian-American about 35 percent; and African-American patients only about 19 percent of the time. This demonstrates the huge gap between the need for compatible donors and the supply.

This is even more true in the case of solid organs from live and brain-dead donors. Right now there are more than 116,000 Americans waiting for a life-saving transplant organ. My colleagues and I at Reason have been arguing for decades in favor of compensating live donors for kidneys and pieces of their livers and the next-of-kin of brain-dead donors for other solid organs. If researchers and entrepreneurs succeed in boosting bone marrow donations by implementing various compensation schemes, perhaps that will prompt Congress to repeal its ill-conceived ban on compensation for organs donated for transplant.

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Trump Administration Withdraws Proposed Obama Ban on Compensation for Bone Marrow – Reason (blog)

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Trust those cells to help cure cancer – The Hindu

Nalini Ambady, the first Indian-American woman to teach psychology at three major universities in the U.S., died in 2013 due to leukaemia when she was just 54.

For the medical fraternity in Kerala, her native place, it turned the spotlight on the lack of awareness of stem cell transplant, which could have saved her life.

Four years down the lane, doctors say the situation has changed only marginally, as many patients who require the treatment have not been able to do it because of high expenses, lack of matching donors, and lack of facilities at hospitals.

Doctors note that stem cell transplant is being proposed as an effective treatment for cancers such as leukaemia and lymphoma, and primary immune deficiency disorders. Stem cells do not develop normally in such patients and it affects the blood cells that they make.

By a transplant, the patient gets new stem cells that can make new and healthy blood cells. Earlier, stem cells were collected from the bone-marrow. Now, it is being collected from blood cells.

Neeraj Sidharthan, bone marrow transplant physician at Amrita Institute of Medical Sciences, Kochi, told The Hindu that in Prof. Ambadys case, though matching donors were found, they had all dropped out.

Lack of awareness is still a major issue though there are some positive signs. In some cases, because of lack of infrastructure, cancer cases are not being diagnosed early, and treatment is delayed too, he said.

Ajith Kumar V.T., professor, department of paediatrics, Government Medical College, Manjeri, said donors could not be found often from the same families because of the nuclear family system. There are not many places where you can match the human leukocyte antigen (HLA) typing with donors. Another problem is the lack of stem cell registries in the State from where matching unrelated donors could be found. Even if doctors suggest a stem cell transplant, many families dont opt for it because of the high cost involved. If the donor is from the same family, the cost is relatively low.

But for unrelated donors, it is very high, Dr. Sidharthan said. The solution, Dr. Ajith Kumar said, was government intervention to set up HLA registries and bone marrow transplant centres. nestCare Foundation, a not-for-profit organisation based in the U.S., had recently approached us expressing interest to set up these facilities in the State. Talks are on, he said. Dr. Sidharthan said that in Tamil Nadu, there was a government scheme enabling poor patients to avail themselves of a financial assistance of Rs. 7 lakh for bone-marrow transplant. We need to have similar schemes here too, he added.

A.S. Jayanth

Lack of awareness is a major issue though there are positive signs. In some cases, because of lack of infrastructure, cancer cases are not being diagnosed early, and treatment is delayed too

Neeraj Sidharthan,

Bone marrow transplant expert

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Trust those cells to help cure cancer – The Hindu

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‘Tissue Paper’ Organs Show Promise – R & D Magazine

A team from Northwestern University has developed a novel type of bioactive tissue that is thin and flexible enough to be folded into origami-like shapes and could be used in a variety of treatment programs.

The tissue paper was created from the structural proteins excreted by cells that give organs their form and structure, combined with a polymer to make the material pliable.

The researchers made the tissue papers from ovarian, uterine, kidney, liver, muscle and heart proteins that were obtained by processing pig and cow organs.

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 in a statement. It’s versatile and surgically friendly.

The cells are removed from the tissues, leaving the natural structural proteinsextracellular matrixthat are then dried into a powder and processed into the tissue papers.

Each paper has a type containing residual biochemical and protein architecture from its original organ that can stimulate cells to act in a certain way.

The researchers made tissue paper from a bovine ovary to grow ovarian follicleseggs and hormone producing cellscultured in vitro, which when 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, reproductive scientist Teresa Woodruff, a co-author on the study, said in a statement.

The ovarian paper with follicles could potentially be implanted under the arm to restore hormone production for cancer patients or women in menopause. Tissue paper made from other organs separately supported the growth of human adult stem cells when scientists placed human bone marrow stem cells on the tissue paper with all the stem cells attached and multiplied over the course of four weeks.

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

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‘Tissue Paper’ Organs Show Promise – R & D Magazine

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Orphan Black Science Recap: One Fettered Slave – The Mary Sue

Welcome to ourOrphan Black science recaps, where Casey, a graduate student in genetics and developmental biology, and Nina, a professional science communicator, examine the science in each episode of OB and talk you through it in (mostly) easy-to-digest terms.

If you havent watched the latest episode of Orphan Black, be forewarned: there will be spoilers. There will also be crazy science.

Nina: The penultimate episode (can you believe that theres only one episode left?) of Orphan Black was all about Helena, who is about to deliver her babies.

Casey: Unfortunately, Helena has gotten herself captured by Coady and Westmorland, and their intentions are to further their personal research using Helenas babies. Of particular interest is the babies cord blood the blood from the placenta and umbilical cord that remains after birth and contains stem cells. It is becoming more and more common for people to save cord blood after they give birth due to the myriad uses these stem cells provide. Today there are almost 80 different diseases, varying from cancers to blood disorders, in which cord blood stem cells can be used as a cure.

Nina: Westmorland is ready and eager to perform a cesarean section to claim Helenas babies but Coady stops him. According to her, a cesarean section could have unpredictable epigenetic effects. The differences and benefits of c-sections versus vaginal births have long been studied for their impacts on health, and its been known that babies delivered via c-section tend to develop more immune diseases, asthma, and allergies. For one thing, babies born by c-section arent exposed to the microbes in their mothers vaginal tract, which can have impacts on how their immune functions and gut flora (digestive functions) develop.

For another: birth is a stressful experience, but that stress plays out differently depending on how the baby is born. Typically, a vaginal birth has a gradual build of stress as its pushed out, but a c-section creates a sudden shift from one environment to a new one. More than one study has remarked that these differences in stress experiences mark different patterns of methylation on their DNA. Methylation is a process that can change the activity of a DNA sequence and dictate whether a gene is expressed or not (as a rule, more methylation = repressed gene expression).Methylation is important for normal development and processes, but when it happens where it shouldnt (or doesnt happen where it should) it can cause problems.

We saw methylation mentioned once earlier this season, when Cosima was looking at Aishas medical files. She found that Aisha had low promoter methylation, which meant that a region of DNA that should have been turned off wasnt and was causing Aisha to develop tumours. One study that looked specifically at methylation of hematopoietic stem cells (the blood cell-producing stem cells found in bone marrow) in babies born vaginally versus babies born by c-section found major differences in methylation in genes linked to metabolism and immune function.

Westmorland doesnt so much care about these potential epigenetic impacts as long as they dont affect his would-be fountain of youth gene. For Coady, however, keeping these changes to a minimum is critical.

Casey: Of course, Coady doesnt want the cord blood for therapeutic uses. Getting her hands on these stem cells would provide her with a limitless source of clone genome for experimentation. While it may not be the exact genome as Project Leda, these stem cells are similar to cells obtained from Kira they contain enough of the clone genome to make them worthy of Neolutions interest.

Nina: Theres only one episode left. Lets hope Helena and her babies stay safe.

(image via BBCAmerica)

Like our science recaps? We wrote The Science of Orphan Blackthe official science companion for the show! Coming August 2017; available for pre-order now.

Casey Griffin is a graduate student in genetics and developmental biology. She obsesses over the blood-brain barrier, plays around with frog embryos, and nerds (and cries and screams) about Orphan Black. You can check out her OB Science Time Tumblr posts here.

Nina Nesseth is a professional science communicator, writer, and serial tea-drinker. Shes happiest when science-ing at people (yes, thats science as a verb). You can find her on Twitter @cestmabiologie.

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Anthony Mills: Trump’s incidental firestorm – Vallejo Times Herald

Since President Trump announced that transgenders are not allowed to join the United States military, which reverses President Obamas permissive policy, a predictable news media firestorm has taken place. It appears that the Chief Executive gravitates toward that which creates political firestorms. The transgender issue is incidental.

Be that as it may, a basic review of genetics and transgenderism is in order.

Each human has a genetic code: Male XY, female XX. On very rare occasions there a variations to this code, like XXY which would be a female hermaphrodite.

It has now become the fashion for people, especially young people, to question the genetic code that they were born with. This thinking has progressed to confusion and insecurity. Then there older and once successful people like Bruce Jenner who subscribed himself to synthetic hormone therapy and re-assignment surgery to gain the semblance of woman. No matter what surgery any male (or female) takes, not matter how much female hormones Jenner or any other males takes (or male hormones a female takes) the genetic code remains the same.

Jenner along with other transgenders cannot reproduce, hence they are sterile.

The opposition counters what is written above comes from hate, another form of gay-bashing.

The oppositions counters by stating that the genetic code of a person remains the same no matter what surgery, no matter what hormones are taken, that (same-sex marriage) and transgenderism has been accepted by much of the of the world. This is true.

In Israel there are gay pride parades. All of North America and much of South America, along with western European countries, accept transgenderism and same-sex marriage. I met with an Anglican Navy chaplain and made notice to the subject that the Queen of England, who is the ultimate head of the Anglican Church, signed the document that allows same-sex marriage in England. He denied this, he then looked downward and could not contest the fact.

It has been said, it has been written and repeated that there will come a time of the Great Apostasy.

It has been said, it has been written and repeated that some of the elect will be among they who are deceived.

That time is now.

Anthony Mills/USNS Carl Brahsear. Bahrain

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Losing My Hair: If I’m Not Bothered, Why Are You? – Yahoo News UK

Going bald was always a foregone conclusion for me. From a young age, I was aware that I had inherited most of my physical traits from my maternal family. Whereas my father’s side was blessed with voluminous mops of thick, black hair and olive skin, all three of my mother’s brothers and my grandfather were (and obviously still are) bald as coots. I grew up being told repeatedly that a similar fate awaited me and, as foretold, my hairline began to recede around the age of 16. To be honest, given the truly hideous ‘curtain’ hairstyle I was rocking in 1995, many might say this was no bad thing.

However, the years passed and somehow, most of my hair remained. In 2006, my hairdresser, who was always very keen to reassure me that I was ‘unlikely to go bald’ given that I hadn’t done so already, suggested a hairstyle which involved sweeping my hair forward over my forehead and spiking the hair around my crown. This was fairly la mode at that time (but then, so were mullets) and I literally thought nothing of it. It’s only with the benefit of hindsight that I can see that actually, my hairdresser was concealing the inevitable truth and helping me to forestall my follicular fate.

I fared quite well. It wasn’t until the age of 32 that the hair loss was of such an extent that I began to shave my head. Given that the aforementioned uncles had, by all accounts, lost their hair by their late teens, I felt fortunate to have held on to mine for so long. What’s more, I was lucky enough to have a partner who reassured me that they found my shaven look attractive. I am, for want of less dramatic terminology, at peace with my premature baldness. Why, then, are so many other people so disappointed on my behalf?

From my mother – who, frankly, should know better given that it’s her contribution to my genetics that has caused it – to random people I barely know, there is never a shortage of people ready and willing to express their sympathy with my ‘plight’. ‘Are you gutted to have lost your hair at such a young age?’. ‘Have you ever considered a hair transplant?’. ‘It’s such a shame as you had such lovely hair’. The comments are numerous and made without a second thought as to how they might make me feel. For some unfathomable reason, unsolicited remarks about this aspect of someone’s appearance seem to be socially acceptable. Conversely, it is rightly considered to be inappropriate or downright offensive to casually mention a person’s weight gain, physical ageing or acne, for example, ‘Are you devastated to have become so wrinkly?’ is certain to offend and understandably so. There is a double standard at play and it could, for some people, be incredibly damaging.

The curiosity, misplaced sympathy and callousness does make me question whether I should be more perturbed about losing my hair than I actually am. Should I, in fact, be spending more time dolefully gazing into the mirror, lamenting the gradual disappearance of my golden locks and frantically researching ways to return to the ‘glory days’ of hairbrushes, combs, shampoo and regular trips to the barber? I think not. After all, for many, hair loss is symptomatic of serious illness, stress and trauma. To self-indulgently bemoan my male pattern baldness as a relatively healthy man headed for 40 with relatively little to complain about would, for me, feel unseemly.

That’s not to say everyone does or should feel the same. Men who seek to regain (see what I did there?) their beautiful barnets should be neither mocked nor castigated – but neither should those who are at ease with the hand dealt to them by genetics, hormones or a mix of the two. Male pattern baldness continues to be open season for uninvited jest, lampooning and commiseration. It’s insensitive, anti-social and wildly inappropriate. Balding, like any other physical change, affects individuals in a variety of ways – a little consideration for the feelings of others costs nothing.

Now, if you’ll excuse me, I have a date with a certain Mr Remington…

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

Marielle Mohs

St. Louis, MO — 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.

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, they’ve 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 doesn’t get any more feminine and [no] more feminine features show up,” said Seay.

Leslie’s 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.”

Leslie’s 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 we’ve been celebrating for a little while. We’ve 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. It’s the only clinic of its kind within a 250-mile radius.

KMOV

TM & 2017 Cable News Network, Inc., a Time Warner Company. All rights reserved.

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Health Matters: Answering Questions about Menopause – NBC2 News

Any health change can bring questions and concerns. One of the most common issues patients ask OB/GYN Dr. Lesley Furman about is menopause. A lot of women come into the office with either questions about menopause or symptoms of menopause and want to know whats going on? Is what Im going through normal?

Dr. Furman says while the symptoms are normal, they can be uncomfortable. Hot flashes are the biggest one. Hot flashes, night sweats, sleeplessness, decrease in libido, vaginal dryness, those are the main ones, said Dr. Furman.

The average age of menopause is 51, but doctors say symptoms can start when women are in their 40s and can last a few years. Its important for them to know that it is a natural process. Its not going to last forever but there are treatment options, said Dr. Furman.

Treatment options, like medications, lifestyle changes, even hormone therapy can help. Each treatment option should be tailored for each patient, not just one size fits all. They should be aware that there are certain factors in their lifestyle that they can alter to help. There can be medications that we can offer that will help, said Dr. Furman.

Doctors may recommend different treatments to help with different symptoms. Patients often have a lot of symptoms; in fact most of the symptoms start way before that last menstrual period occurs, said Dr. Furman.

Women may even experience anxiety or depression during menopause. Whatever the symptoms are, doctors say they are normal and its important to explain them to your physician so treatment can be started.

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WBIR: Genetic Testing Kits Help Reveal Family History – Tennessee Today

August 7, 2017

WBIR TV Channel 10 recently interviewed Bruce McKee, UT professor in the Department of Biochemistry and Cellular and Molecular Biology, for a story examining genetic testing.

WBIRs Marketing Director Kara McFarland knew from a very young age she was adopted. It was never a secret. But all these years later she still knows very little about her biological family. McFarland turned to science to get some answers by using the genetic testing kit 23andMe. Its one of many for sale online and can be taken at home. She collected a saliva sample, sent it off the in mail and a month lager the results were in her computer inbox.

McKee said this type of genetic testing is called microarray technology and is reliable and validated. He noted that thisinformation could be of value for people like Kara who dont know much about their family background. But he added, you need to be prepared for the information your genes hold.

Read the full story online.

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Funding debate aside, this is why we need a new heart hospital – The Sydney Morning Herald

Current debate about the future of the Victorian Heart Hospital, which when completed will be Australia’s first cardiac hospital,focuses on issues such as cost and contracts. And, in these tight economic times, it is right to ask these questions.

However, Australia’s first dedicated specialist heart hospital will be so much more. Thehospital will be in the same league as some of the great cardiac hospitals, such as the Barts Heart Centre in London and the Montreal Heart Institute in Canada.

More Victorians, men and women, die from heart disease than any other cause. People are living longer long enough to have, and survive, heart attacksthat may become heart disease and heart failure further down the line.

In the catchment area that will feed into the Victorian Heart Hospital the population projections for people at risk of heart disease are even worse. Aboutone-quarter (or eight out of 31) of the metropolitan local government areas with above average heart attack rates fall into the catchment area of the new hospital. This is an area whose population needs a facility like this.

But the hospitalwill be so much more than a hospital for patients with cardiovascular disease and events. Much has been said about the dedicated areas for Monash University and Monash Health researchers devoted to cardiac research.

Having the researchers sitting in the midst of the clinicians and patients, and in many cases being situated within the hospital means the problems the scientists address are the ones that are identified by those at the coalface, the clinicians and health professionals.

One of the hospital’score research areas, for example, will be stem cell research. We have recruited some of the best stem cell scientists in the world. They will work with Monash University’s Australian Regenerative Medicine Institute and heart hospital clinicians to develop cellular patches that can be created from a patient’s own cells to replace the areas of the heart left dead by a heart attack. This damaged tissue, currently cannot be fixed, and often leads to heart failure, so the need for this sort of research is paramount.

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Monash Health has an outstanding international reputation for attracting clinical trials into new heart procedure techniques, with more than 30 trials currently being conducted. As an example, the international medical device makerMedtronicchose Monash Heart cardiologists to conduct the first trial of a new way to replace mitral valves in the hearts of patients whose health would not withstand traditional open-heart surgery. These trial patients have had their life saved by this device.

This is translational research at its best taking new discoveries and therapies and making sure they are safe in patients. These innovations then become, as fast as possible, treatments we can offer all Victorians. It is no surprise that many of Australia’s largest medical device manufacturers and innovators are situated around Monash University and benefit from the strong biomedical focus the university offers.

Co-location of the Victorian Heart Hospital at the Monash University campus will strengthen the nexus between industry, biomedical research and clinical care, including clinical trials that will result in Victorians benefiting from the best advances in cardiac care.

The Victorian Heart Hospitalis a way for Victoria to future-proof its citizens against heart disease for the next five decades. It will be where we develop new technologies, devices and treatments that can be used to deal with the patients that come throughour doors.

There will be more non-surgical alternatives and prevention strategies developed and offered. We will provide a health and wellness department that assists patients in dealing with the depression that can follow cardiac surgery, as well as assisting patients in techniques that can help them lower their risk of further cardiac events.

The hospitalwill not only put Victoria on the world map, it will be a groundbreaking commitment to the health of Victorians.

Sarah Newton is deputy dean, external relations, Monash University’s faculty of medicine, nursing and health sciences.

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Indian-origin doctor helps gene editing of human embryos – Times of India

NEW DELHI: For the first time, genetically modified human embryos have been developed in the US and Kashmir-born doctor Sanjeev Kaul has played a lead role in this breakthrough.

Scientists have now demonstrated an effective way of using a gene-editing tool to correct a disease-causing gene mutation in human embryos and stop it from passing to future generations.

Though this is not a full-fledged start of a revolution of having ‘designer babies’, the first steps, however, have been laid. China attempted this earlier.

A team of scientists has altered human embryos using a new technique called CRISPR CAS9 that edits genes and in this case it helped remove a fatal mutation that leads to heart attacks.

This now opens up an ethical ‘Pandora’s Box’ if germline repairs and enhancements may become a thing in vogue.

As of now, the human embryos were not implanted in humans. But this now opens up exciting prospects of the world having designer babies soon.

The research published in British journal Nature shows the first genetically modified human embryos made in America.

A team of South Korean, Chinese and American scientists has identified how they could edit out a faulty gene that causes heart attacks in later life due to the thickening of heart walls.

One of the team members is Dr Kaul, who was born in Kashmir, studied in New Delhi and later immigrated to America.

“Although the rare heart mutation affects men and women of all ages, it is a common cause of sudden cardiac arrest in young people, and it could be eliminated in one generation in a particular family,” said co-author Kaul, a professor of medicine (cardiovascular medicine) in the OHSU School of Medicine and director of the OHSU Knight Cardiovascular Institute.

“Thanks to advances in stem cell technologies and gene editing, we are finally starting to address disease-causing mutations that impact potentially millions of people,” says Juan Carlos Izpisua Belmonte, a professor in California-based Salk Institutes Gene Expression Laboratory and a corresponding author of the paper.

“Gene editing is still in its infancy so even though this preliminary effort was found to be safe and effective, it is crucial that we continue to proceed with the utmost caution, paying the highest attention to ethical considerations.”

CRISPR CAS9 or Clustered Regularly Interspaced Short Palindromic Repeats is a kind of a precise molecular scissor the scientists use to edit faulty genes.

Only selected healthy embryos were allowed to grow further that too only for a few days. The embryos were not implanted in humans.

The big step forward is that a higher percentage embryos were found to have been repaired in this American experiment than earlier attempts.

CRISPR holds promise for correcting mutations in the human genome to prevent genetic disease. Using an enzyme called Cas9, it is possible to snip a specific target sequence on a mutant gene.

The new study found that human embryos effectively repair these breaks in the mutant gene using the normal copy of this gene from a second parent as a template.

The resulting embryos contain now repaired, mutation-free copies of this gene.

The technique already has been used in animals for generating mutant models; however, the new study is the first to demonstrate that technique can be used in human embryos to convert mutant genes back to normal.

The study also demonstrated a way for overcoming a crucial problem in genome editing in embryos known as mosaicism.

Mosaicism refers to an outcome when not all cells in a multicellular embryo get repaired and some cells still carry a mutation.

“Every generation on would carry this repair because we have removed the disease-causing gene variant from that family’s lineage,” said senior author Shoukhrat Mitalipov, PhD, who directs the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University (OHSU), in Portland, Oregon, USA.

“By using this technique, it is possible to reduce the burden of this heritable disease on the family and eventually the human population.”

The study provides new insight into a technique that could apply to thousands of inherited genetic disorders affecting millions of people worldwide.

The gene-editing technique described in this study, done in concert with in vitro fertilisation, could provide a new avenue for people with known heritable disease-causing genetic mutations to eliminate the risk of passing the disease to their children.

“If proven safe, this technique could potentially decrease the number of cycles needed for people trying to have children free of genetic disease,” said co-author Paula Amato, associate professor of obstetrics and gynaecology in the OHSU School of Medicine.

Designer babies could be in the offing.

“Our results demonstrate the great potential of embryonic gene editing, but we must continue to realistically assess the risks as well as the benefits,” adds Belmonte.

In this landmark study, the researchers worked with healthy donated human oocytes and sperm carrying the genetic mutation that causes cardiomyopathy or the thickening of heart walls.

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Gene editing breakthrough: Perspective from a geneticist and a pastor – WTSP 10 News

A test eliminated a disease-causing gene, but some fear the technique could lead to designer babies.

Mark Rivera, WTSP 11:25 PM. EDT August 02, 2017

Genetics (iStock)

ST. PETE People have been sharing aNew York Times story all over social media: For the first time, scientists in the US have successfully changed a disease-causing gene inside a human embryo.

It was just a basic test. But the technique could one day stop genetic diseases and even birth defects.

The possibilities are incredible but where do you draw the line?

Could the same technique be used to change genes so babies are smarter, taller, and have the exact eye colors you want?

We spoke with a geneticist and a pastor to get perspective on the debate.

Scientists say diseases like breast and ovarian cancer, Huntington’s disease and more could be cured before birth – never to be passed on again.

Before birth – during fertilization – doctors use a natural enzyme to cut out the bad gene and replace it with the good one.

The possibilities are enormous, said Johns Hopkins All Childrens Hospital geneticist Dr. Maxine Sutcliffe.

She says this could be a new paradigm of disease prevention in humans.

But there are two big issues. Safety first.

If you’re going to cut into something. you want to make sure you don’t damage something else, Sutcliffe said.

And ethics.

We have the potential, we have the expertise, we have the ability to keep this under control and let it work for the good of mankind as opposed to the destructive side, the manipulative side, or the wrong side, she said.

Sutcliffe said we are decades upon decades away from being able to pick the traits for our kids — if we ever will be — but should we cure a disease in a fetus if we can?

I mean who benefits from it? If it’s only the wealthy that can benefit from this, then the wealthy that become healthier, the wealthy become smarter, the wealthy become better looking, whatever that is. Is that what we want as a culture? asked Rev. Dr. Craig Nelson.

Nelson pastors the First United Methodist Church in St. Petersburg.

There can be great benefits and people can become incredible gifts to society with all kinds of diversity that we have, he said.

Rev. Nelson said he is alive today in part because he received a gene therapy treatment to fight his stage 4 lung cancer.

First doctor said I had 6 months to a year to live 5 1/2 years ago, he said.

Where technologies can help us in eliminating pain and suffering, where it can lead to a healthier world and culture, pursue it, but you can’t just say, ‘Oh yeah it can do this, we’re all in.’

When there’s a preference that would start permeating culture, then that leads to uniformity. That leads to stormtroopers on ‘Star Wars,’ you know? … I mean, Hitler tried it, you know? And where did that get us?

Makeit easy to keep up-to-date with more stories like this.Download the 10 Newsapp now.

Have a news tip? Email tips@wtsp.com, visit ourFacebook pageorTwitter feed.

2017 WTSP-TV

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WBZ-TV Riders Take On Pan-Mass Challenge – CBS Boston / WBZ

WELLESLEY (CBS) Several members of the WBZ-TV team took part in the Pan-Mass Challenge this weekend.

Among the 600 riders heading to Foxboro from Wellesley Sunday morning was WBZ-TV meteorologist Barry Burbank, riding his second PMC as a member of the WBZ Cyclones.

Its a fantastic ride, its great to be with everybody here, Burbank told WBZ NewsRadio 1030s Doug Cope. Were here for a common goal. Were all getting to the end line, and we want to find that cure for this horrendous disease cancer.

He and WBZ anchor David Wade talked to WBZ-TVs Nick Giovanni Sunday morning about why they ride the PMC.

WBZ-TVs David Wade at the start of day 2 of the Pan-Mass Challenge. (WBZ-TV)

This year was Wades fourth ride. He was riding for Team Gene Therapy. They ride in honor of Gene Aaron, a doctor who delivered Wades sons. Aaron is now battling pancreatic cancer, and the team is working to raise money for research.

The energy is incredible, and you get to meet some of the kids, some of the survivors, some of the people that youre riding for, he said.

Burbank said cancer has struck some of his friends, family, both young and old.

WBZ-TV anchor Lisa Hughes did the complete 192-mile ride.

It is the most perfect biking day, its beautiful, Hughes said. We saw the sun come up over the Cape Cod Canal, and all the fishermen and the blue herons, and he whole day has been perfect so far.

WBZ-TV reporter Mike LaCrosse also rode the PMC this year.

He finished the 192-mile route Sunday afternoon.

WBZ NewsRadio 1030s Doug Cope reports

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Scientists successfully doctor human embroyo – Examiner Enterprise

By Melissa Healy Los Angeles Times

(TNS) Using a powerful gene-editing technique, scientists have rid human embryos of a mutation that causes an inherited form of heart disease often deadly to healthy young athletes and adults in their prime.

The experiment marks the first time that scientists have altered the human genome to ensure a disease-causing mutation would disappear not only from the DNA of the subject on which its performed, but from the genes of his or her progeny as well.

The controversial procedure, known as germ-line editing, was conducted at Oregon Health & Science University using human embryos expressly created for the purpose. It was reported Wednesday in the journal Nature.

The new research comes less than six months after the National Academies of Science, Engineering and Medicine recommended that scientists limit their trials of human germ-line editing to diseases that could not be treated with reasonable alternatives at least for now.

In a bid to make the experiment relevant to real-life dilemmas faced by parents who carry genes for inherited diseases, the researchers focused their editing efforts on a mutation that causes inherited hypertrophic cardiomyopathy.

In this genetic condition, a parent who carries one normal and one faulty copy of a the MYBPC3 gene has a 50-50 chance of passing that mutation on to his or her offspring. If the child inherits the mutation, his or her heart muscle is likely to grow prematurely weak and stiff, causing heart failure and often early death.

In diseases where one parent carries such an autosomal dominant mutation, a couple will often seek the assistance of fertility doctors to minimize the risk of passing such a mutation on to a child. A womans egg production is medically stimulated, and eggs and sperm meet in a lab a process called in vitro fertilization. Then embryologists inspect the resulting embryos, cull the ones that have inherited an unwanted mutation, and transfer only unaffected embryos into a womans uterus to be carried to term.

In the new research, researchers set out to test whether germ-line gene editing could make the process of choosing healthy embryos more effective and efficient by creating more of them.

In the end, their experiment showed it could. The targeted correction of a disease-causing gene carried by a single parent can potentially rescue a substantial portion of mutant human embryos, thus increasing the number of embryos available for transfer, the authors wrote in Nature. Co-author Dr. Paula Amato, an Oregon Health & Science University (OHSU) professor of obstetrics and gynecology, said the technique could potentially decrease the number of cycles needed for people trying to have children free of genetic disease if its found safe for use in fertility clinics.

Along the way, though, many of the researchers findings were scientifically surprising. Long-feared effects of germ-line editing, including collateral damage to off-target genetic sequences, scarcely materialized. And mosaicism, a phenomenon in which edited DNA appears in some but not all cells, was found to be minimal.

The studys lead author, OHSU biologist Shoukhrat Mitalipov, called these exciting and surprising moments. But he cautioned that there is room to improve the techniques demonstrated to produce mutation-free embryos. As for conducting human clinical trials of the germ-line correction, he said those would have to wait until results showed a near-perfect level of efficiency and accuracy, and could be limited by state and federal regulations.

Eventually, Mitalipov said, such germ-line gene editing might also make it easier for parents who carry other gene mutations that follow a similar pattern of inheritance including some that cause breast and ovarian cancers, cystic fibrosis and muscular dystrophy to have healthy children who would not pass those genes to their own offspring.

There is still a long road ahead, predicted Mitalipov, who heads the Center for Embryonic Cell and Gene Therapy at the Portland university.

The research drew a mix of praise and concern from experts in genetic medicine.

Dr. Richard O. Hynes, who co-chaired the National Academies report issued in February, called the new study very good science that advances understanding of genetic repair on many fronts. Hynes, who was not involved with the latest research effort, said he was pleasantly surprised by researchers clever modifications and their outcomes.

Its likely to become feasible, technically not tomorrow, not next year, but in some foreseeable time. Less than a decade, Id say, said Haynes, a biologist and cancer researcher at MIT and the Howard Hughes Medical Institute.

University of California, Berkeley molecular and cell biologist Jennifer Doudna, one of pioneers of the CRISPR-Cas9 gene-editing technique, acknowledged the new research highlights a prospective use of gene editing for one inherited disease and offers some insights into the process.

But Doudna questioned how broadly the experiments promising results would apply to other inherited diseases. She said she does not believe the use of germ-line editing as a means to improve efficiency at infertility clinics meets the criteria laid out by the National Academies of Science, which urged that the techniques only be explored as treatment for diseases with no reasonable alternative.

Already, 50 percent of embryos would be normal, said Doudna. Why not just implant those?

Doudna said she worried that the new findings will encourage people to proceed down this road before the scientific and ethical implications of germ-line editing have been fully considered.

A large group of experts concluded that clinical use should not proceed until and unless theres broad societal consensus, and that just hasnt happened, Doudna said. This study underscores the urgency of having those debates. Because its coming.

What is clear is that the researchers a multinational team of geneticists, cardiologists, fertility experts and embryologists from OHSU and from labs in South Korea and China tried a number of innovations in an effort to improve the safety, efficiency and fidelity of gene editing. And most yielded promising results.

After retrieving eggs from 12 healthy female volunteers, researchers simultaneously performed two steps that had never been combined in a lab: At the same moment that they fertilized the eggs with the sperm of a man who carried a single copy of the mutated gene, they introduced the CRISPR-Cas9 repair machinery.

The resulting embryos took up the genetic-editing program so efficiently and uniformly that, after five days of incubation, 72.4 percent of the embryos (42 of 58) created and tested were free of the MYBPC3 mutation. By comparison, when sperm carrying the single mutation was used to fertilize eggs without any genetic manipulation, just 47.4 percent of embryos were free of the mutation linked to the deadly heart condition.

The researchers believe the timing and the techniques they used prompted the embryos to rely on the healthy maternal copy of the gene as a model for fixing the MYBPC3 mutation, and not a repair template they introduced alongside the editing machinery when the eggs were fertilized. Only one of the 42 embryos used the introduced template for repair. The scientists contrasted this process to the DNA-repair mechanism operating in stem cells, which do use repair templates.

As the embryos cells divided and they matured to the blastocyst stage the point at which they would usually be ready for transfer to a womans uterus _ they did so normally. After extensive testing, the embryos were used to make embryonic stem-cell lines, which are stored in liquid nitrogen and can be used in future research.

Researchers also noted that genetic mosaicism _ a concern raised by earlier experimental efforts at gene editing _ was virtually absent from the 42 embryos that were free of the disease-causing mutation. Only one of the 42 embryos exhibited mosaicism, a condition in which cells did not all carry the same mutation-free genetic code.

MITs Hynes said such findings offer important insights into how human embryos grow, develop and respond to anomalies, and will help families facing infertility and inherited illnesses.

Human embryogenesis is clearly different from that of a mouse, which we know a lot about, said Hynes. That needs to be studied in human embryos, and theres no other way to do it.

The results of the current study are not low enough yet for most applications _ certainly not for clinical applications, but its a big step forward, he added.

While calling the new research very nice science, Hynes downplayed fears that germ-line editing would soon lead to tinkering with such attributes as looks, personality traits and intelligence in human children. Were not looking at designed babies around the corner not for a long time, he said.

But we need to take advantage of the time and space we now have, he said, to make decisions about which uses of the technique are legitimate and which are not.

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Scientists successfully doctor human embroyo – Examiner Enterprise

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Selecting at negative 9 months – High Plains Journal

In 2014, the University of Arkansas estimated one purebred calf born from embryo transfer must have a market premium of $1,500 to $2,000 greater than naturally conceived calves in order to pay for the costs to produce it.

In the roughly 40 years or so that ET has been conducted in cattle, it wasnt until recently that embryos could be sorted according to sex. And most of that has been done in the dairy cattle businessnot the beef cattle business. That means even a successful ET calf on the ground may not in fact meet the ranchers selection requirements for that premium price that would pay their costs of production. Or, the calf may not exhibit the physiological traits that would match what his or her genetic potential had been on paper.

Months of planning and thousands of dollars invested in harvesting and implanting an embryo may either wind up in a stellar replacement female to advance your herds geneticsor it might result in a bull destined for the steer pen.

But Matt Barten, of Embruon, Salina, Kansas, is working to change that one beef calf embryo at a time. His company uses bovine embryo biopsy and genomic data captured by the various purebred beef cattle associations to evaluate embryos for producers. With a few cells, Bartens company can tell a rancher not only the sex of the embryo, but also what genetic traits the calf will exhibit in the pasture or in the feedlot.

I say that its making those genetic decisions at negative nine months, Barten said. By the time you have an embryo calf on the ground, you could have up to $2,000 in that calf. So, if you can make the decision to implant that embryo based on what that calf is in the nitrogen tank, it saves you time and money.

With embryo transfer, by and large, the biggest dollar figure and resources that you have tied up are in the recipient herd, Barten explained. Using his embryo biopsy technology a rancher can make transplant decisions as to not only what gender those embryos are, but if they have a recessive genetic profile or traits that a rancher would like to bring into a herd, before a pregnancy occurs.

Maybe more important is that it can help cattlemen build the desired genetics in their herds with more precision than they have ever had in the past.

Early adopter

Charlie Cartwright owns Cannon Ridge Angus in Shelbyville, Tennessee. He and his wife have built their Angus herd using artificial insemination, in vitro fertilization and embryo transfer since he retired from the military in 2013.

We bought the first 10 pregnant cows in September of 2013, just by going to various sales across the nation and trying to build our genetics, Cartwright said. Cannon Ridge markets replacement females to purebred cattlemen, so its important that he be able to select female embryos to transfer. But some ranchers might be more interested in the other capabilities of the testing, such as telling if an embryo carries harmful genetic traits that theyd like to select against.

My focus right now is on getting more females, Cartwright said. But as we get down the road and as GE-EPDs and DNA testing is more prevalent, we might start to look more at the DNA of the embryos. If Matt can tell me that these are her numbers, I can choose if I want to put that embryo in. If the embryo shows more traits coming from the mother or the sire, maybe I decide not to put the embryo in.

When we got started it was because I wanted to do full genomic profiling, Barten said. We wanted to offer the GE-EPD at Embruon. So now, using information from the Angus breed association, we can know genomically what that calf will be at the embryo level. We can tell within a group of 10 or 100 which embryos will have more carcass potential or more maternal potential genomically.

And, with each breed association collecting more genomic data on its cattle, the Embruon process can be used for practically any purebred cattle embryos.

Cell amplification

The process is breed-specific, Barten said. Probably the most applicable in the dairy industry because they have so many genotyped animals already. The Angus breed, though, is one that has built up its number of genotyped animals, he added.

Embruons process is fairly simple. A rancher like Cartwright can use conventional flushing methods to get embryos from his cows. Then, he overnight ships the embryos to Barten at Embruon in a culture media to grow while theyre on the way. Barten said its like culturing bacteria on a petri dish. The embryos are biopsied the next day.

The biopsy just takes a few cells from the outer layer of the embryo cell, what would eventually develop into the placenta for the calf in utero, Barten explained. This is where the process gets really delicate. Unlike a DNA sample from a live animals hair or tissue that is composed of hundreds of thousands of cells, there are fewer cells in an embryo to test. Barten works with Neogens GeneSeek Operations in Lincoln, Nebraska, to amplify the number of cells to get enough DNA for evaluation using the genomic data from the breed association.

Embryo before biopsy. (Photo courtesy ofEmbruon.)

Embryo after biopsy. (Photo courtesy ofEmbruon.)

Embryo recovering from biopsy. (Photo courtesy ofEmbruon.)

Embryo before biopsy. (Photo courtesy ofEmbruon.)

Embryo after biopsy. (Photo courtesy ofEmbruon.)

Embryo recovering from biopsy. (Photo courtesy ofEmbruon.)

The most difficult part of this was trying to get the DNA to amplify, Barten said. Its like having to turn a bushel basket of corn into two semi-loads so that you can run it through the pipeline.

Except, in this case every copy of those embryonic cellsevery corn kernelhas to be identical following amplification, or youll introduce errors. Barten said if theres an error in the cell amplification, it can introduce bias into the testing.

These cells have to go through amplification somewhere on the scale of 2,100 times, Barten explained. If you introduce an error, then the genetic prediction starts to get really skewed. Think about a sniper taking a shot at a half of a mile. If youre just one-eighth of an inch off, when you shoot, by the time the bullet reaches the target its a foot off. It took Barten working with GeneSeek a year to get the process fine-tuned so that theres a high degree of accuracy.

We need two things at the end of the day, Barten said. We have to have a high degree of accuracy in predicting what the embryo will become, and we have to be able to transfer the embryo for pregnancy.

Following their biopsy, the embryos stay on their culture for a little while to recover. Barten will look at the embryos under the microscope and evaluate if they are recovered and able to be implanted.

Moving the cost curve

From here, a breeder has two choices. The embryos can be frozen and the breeder can wait on the data to decide to implant them, or they can go ahead and implant them and decide after he gets the data if the pregnancies are what he desired. It all depends on their market goals, their labor resources and other factors.

This process works well for ranchers who dont have an infinite pool of recipient cows at their disposal, and who really need to make every decision count before they tie up their resources, Barten said. Its about moving the cost curve back to the point before theres a calf, and resources are devoted to something that isnt desired.

By making their decisions at negative nine months, the rancher can do in a year or two what it would take some other operation three to four years to do, Barten explained. Thats because every year hes making his decisions on the embryonic level, knowing what hell get. Barten worked with a Kansas State University graduate student, Dustin Aherin, to crunch the numbers. Using computer modeling they found the expense to run double the number of recipient cows in a year can add up to $40,000 in costs in a year, and a rancher could still wind up with calves he would not be able to sell at or above market value.

Bovine embryo biopsy isnt new, Barten said. The technology is used in many other applications. Barten developed his concept for Embruon after he graduated from Fort Hays State University. Hes worked as a bovine ultrasound technician and as an embryo transfer technician. In 2014, he had some clients who were dealing with a recessive trait in their herd, and he thought if he could identify the embryos that were free of the disorder versus those who were carriers, that he could help them. Eventually that led to the creation of Embruon, Barten said.

The science of the technology is what appeals to Cartwright and one of the reasons why hes an early adopter. Hes looking to see this fall in the 10 recipient cows hes implanted with Embruon-evaluated embryos what their pregnancy rates were and what were the final costs of his operation invested in the procedure.

Every cow and every rancher is different, and there are a lot of ways to do embryonic production, Cartwright said. I look at each one as a tool in the box. He added that ranchers need to evaluate for themselves if technology like this will work for them.

Barten is optimistic about the future of his company. They have plans to expand laboratory space into Wichita, Kansas, and adding staff to ease the workload. But for him, the real point of pride is helping cattlemen like his dad improve their herds more efficiently than they were ever able to before.

And all before an embryo is ever implanted.

Jennifer M. Latzke can be reached at 620-227-1807 or jlatzke@hpj.com.

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Researchers Explore the Science of Gender Identity – NBCNews.com

NEW YORK While President Donald Trump has thrust transgender people back into the conflict between conservative and liberal values in the United States, geneticists are quietly working on a major research effort to unlock the secrets of gender identity.

A consortium of five research institutions in Europe and the United States, including Vanderbilt University Medical Center, George Washington University and Boston Children’s Hospital, is looking to the genome, a person’s complete set of DNA, for clues about whether transgender people are born that way.

Two decades of brain research have provided hints of a biological origin to being transgender, but no irrefutable conclusions.

A worker checks the serial number on a slice of human brain before using a saw to cut a piece from the sample at a brain bank in the Bronx borough of New York City, New York, U.S. June 28, 2017. CARLO ALLEGRI / Reuters

Now scientists in the consortium have embarked on what they call the largest-ever study of its kind, searching for a genetic component to explain why people assigned one gender at birth so persistently identify as the other, often from very early childhood.

Researchers have extracted DNA from the blood samples of 10,000 people, 3,000 of them transgender and the rest non-transgender, or cisgender. The project is awaiting grant funding to begin the next phase: testing about 3 million markers, or variations, across the genome for all of the samples.

Knowing what variations transgender people have in common, and comparing those patterns to those of cisgender people in the study, may help investigators understand what role the genome plays in everyone’s gender identity.

“If the trait is strongly genetic, then people who identify as trans will share more of their genome, not because they are related in nuclear families but because they are more anciently related,” said Lea Davis, leader of the study and an assistant professor of medicine at the Vanderbilt Genetics Institute.

The search for the biological underpinnings is taking on new relevance as the battle for transgender rights plays out in the U.S. political arena.

One of the first acts of the new Trump administration was to revoke Obama-era guidelines directing public schools to allow transgender students to use bathrooms of their choice. Last week, the president announced on Twitter he intends to ban transgender people from serving in the military.

Related: Despite Trump’s Tweets, Trans Army Sergeant Keeps Proudly Serving

Texas lawmakers are debating a bathroom bill that would require people to use the bathroom of the sex listed on their birth certificate. North Carolina in March repealed a similar law after a national boycott cost the state hundreds of millions of dollars in lost business.

Currently, the only way to determine whether people are transgender is for them to self-identify as such. While civil rights activists contend that should be sufficient, scientists have taken their search to the lab.

That quest has made some transgender people nervous. If a “cause” is found it could posit a “cure,” potentially opening the door to so-called reparative therapies similar to those that attempt to turn gay people straight, advocates say. Others raise concerns about the rights of those who may identify as trans but lack biological “proof.”

Davis stressed that her study does not seek to produce a genetic test for being transgender, nor would it be able to. Instead, she said, she hopes the data will lead to better care for transgender people, who experience wide health disparities compared to the general population.

One-third of transgender people reported a negative healthcare experience in the previous year such as verbal harassment, refusal of treatment or the need to teach their doctors about transgender care, according to a landmark survey of nearly 28,000 people released last year by the National Center for Transgender Equality.

Related: Major Transgender Rights Case Returns to Lower Court

Some 40 percent have attempted suicide, almost nine times the rate for the general population.

“We can use this information to help train doctors and nurses to provide better care to trans patients and to also develop amicus briefs to support equal rights legislation,” said Davis, who is also director of research for Vanderbilt’s gender health clinic.

The Vanderbilt University Medical Center in Tennessee has one of the world’s largest DNA databanks. It also has emerged as a leader in transgender healthcare with initiatives such as the Trans Buddy Program, which pairs every transgender patient with a volunteer to help guide them through their healthcare visits.

The study has applied for a grant from the National Institutes of Health and is exploring other financial sources to provide the $1 million needed to complete the genotyping, expected to take a year to 18 months. Analysis of the data would take about another six months and require more funding, Davis said.

The other consortium members are Vrije University in Amsterdam and the FIMABIS institute in Malaga, Spain.

PROBING THE BRAIN

Until now, the bulk of research into the origins of being transgender has looked at the brain.

Neurologists have spotted clues in the brain structure and activity of transgender people that distinguish them from cisgender subjects.

A seminal 1995 study was led by Dutch neurobiologist Dick Swaab, who was also among the first scientists to discover structural differences between male and female brains. Looking at postmortem brain tissue of transgender subjects, he found that male-to-female transsexuals had clusters of cells, or nuclei, that more closely resembled those of a typical female brain, and vice versa.

Swaab’s body of work on postmortem samples was based on just 12 transgender brains that he spent 25 years collecting. But it gave rise to a whole new field of inquiry that today is being explored with advanced brain scan technology on living transgender volunteers.

Dr. Ivanka Savic points to a study on the screen of her computer at her home in Los Angeles, California, U.S. June 30, 2017. Lucy Nicholson / Reuters

Among the leaders in brain scan research is Ivanka Savic, a professor of neurology with Sweden’s Karolinska Institute and visiting professor at the University of California, Los Angeles.

Her studies suggest that transgender men have a weakened connection between the two areas of the brain that process the perception of self and one’s own body. Savic said those connections seem to improve after the person receives cross-hormone treatment.

Her work has been published more than 100 times on various topics in peer-reviewed journals, but she still cannot conclude whether people are born transgender.

“I think that, but I have to prove that,” Savic said.

Related: Pressure Mounts to Curtail Surgery on Intersex Children

A number of other researchers, including both geneticists and neurologists, presume a biological component that is also influenced by upbringing.

But Paul McHugh, a university professor of psychiatry at the Johns Hopkins School of Medicine, has emerged as the leading voice challenging the “born-this-way” hypothesis.

He encourages psychiatric therapy for transgender people, especially children, so that they accept the gender assigned to them at birth.

McHugh has gained a following among social conservatives, while incensing LGBTQ advocates with comments such as calling transgender people “counterfeit.”

Last year he co-authored a review of the scientific literature published in The New Atlantis journal, asserting there was scant evidence to suggest sexual orientation and gender identity were biologically determined.

The article drew a rebuke from nearly 600 academics and clinicians who called it misleading.

McHugh told Reuters he was “unmoved” by his critics and says he doubts additional research will reveal a biological cause.

“If it were obvious,” he said, “they would have found it long ago.”

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Gene therapy via skin could treat diseases such as obesity – UChicago News

A University of Chicago-based research team has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.

In a study inthe journal Cell Stem Cell, the researchers provide proof-of-concept. They describe gene-therapy administered through skin transplants to treat two related and extremely common human ailments: Type 2 diabetes and obesity.

We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, said study author Xiaoyang Wu, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin.

Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice.

The mouse system is less mature, Wu said. It took us a few years to optimize our 3-D skin organoid culture system.

This study is the first to show that an engineered skin graft can survive long term in wild-type mice with intact immune systems. We have a better than 80 percent success rate with skin transplantation, Wu said. This is exciting for us.

The researchers focused on diabetes because it is a common non-skin disease that can be treated by the strategic delivery of specific proteins.

They inserted the gene for glucagon-like peptide 1 (GLP1), a hormone that stimulates the pancreas to secrete insulin. This extra insulin removes excessive glucose from the bloodstream, preventing the complications of diabetes. GLP1 can also delay gastric emptying and reduce appetite.

Using CRISPR, a tool for precise genetic engineering, they modified the GLP1 gene. They inserted one mutation, designed to extend the hormones half-life in the blood stream, and fused the modified gene to an antibody fragment so that it would circulate in the blood stream longer. They also attached an inducible promoter, which enabled them to turn on the gene to make more GLP1, as needed, by exposing it to the antibiotic doxycycline. Then they inserted the gene into skin cells and grew those cells in culture.

When these cultured cells were exposed to an air/liquid interface in the laboratory, they stratified, generating what the authors referred to as a multi-layered, skin-like organoid. Next, they grafted this lab-grown gene-altered skin onto mice with intact immune systems. There was no significant rejection of the transplanted skin grafts.

When the mice ate food containing minute amounts of doxycycline, they released dose-dependent levels of GLP1 into the blood. This promptly increased blood-insulin levels and reduced blood-glucose levels.

When the researchers fed normal or gene-altered mice a high-fat diet, both groups rapidly gained weight. They became obese. When normal and gene-altered mice got the high-fat diet along with varying levels of doxycycline, to induce GLP1 release, the normal mice grew fat and mice expressing GLP1 showed less weight gain.

Expression of GLP1 also lowered glucose levels and reduced insulin resistance.

Together, our data strongly suggest that cutaneous gene therapy with inducible expression of GLP1 can be used for the treatment and prevention of diet-induced obesity and pathologies, the authors wrote.

When they transplanted gene-altered human cells to mice with a limited immune system, they saw the same effect. These results, the authors wrote, suggest that cutaneous gene therapy for GLP1 secretion could be practical and clinically relevant.

This approach, combining precise genome editing in vitro with effective application of engineered cells in vivo, could provide significant benefits for the treatment of many human diseases, the authors note.

We think this can provide a long-term safe option for the treatment of many diseases, Wu said. It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as hemophilia. Or it could function as a metabolic sink, removing various toxins.

Skin progenitor cells have several unique advantages that are a perfect fit for gene therapy. Human skin is the largest and most accessible organ in the body. It is easy to monitor. Transplanted skin can be quickly removed if necessary. Skins cells rapidly proliferate in culture and can be easily transplanted. The procedure is safe, minimally invasive and inexpensive.

There is also a need. More than 100 million U.S. adults have either diabetes (30.3 million) or prediabetes (84.1 million), according the Centers for Disease Control and Prevention. More than two out of three adults are overweight. More than one out of three are considered obese.

Additional authors of the study were Japing Yue, Queen Gou, and Cynthia Li from the University of Chicago and Barton Wicksteed from the University of Illinois at Chicago. The National Institutes of Health, the American Cancer Society and the V Foundation funded the study.

Article originally appeared on Science Life.

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Gene therapy via skin could treat diseases such as obesity – UChicago News

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Nano-chip promises to heal organs at a touch | Cosmos – Cosmos

Injured tissues can be repaired and damaged organs healed using a new nanotech device that adapts a patients own skin to generate stem cells, according to a paper published in the journal Nature Nanotechnology.

Researchers from Ohio State University call the new technology tissue nanotransfection (TNT).

They say TNT which is basically a lab on a chip can adapt skin cells to change into any type of tissue required, which can then be introduced to injured or degenerated areas. They claim a success rate of 98%.

With this technology we can convert skin cells into elements of any organ with just one touch, says co-author Chandan Sen. 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.”

Lead author Daniel Gallego-Perez says the new technology comprises two elements: the nanotech chip designed to introduce reprogrammed DNA into existing adult cells; and a specific biological cargo that induces the cells to change from one type to another.

The device works using a small electrical charge.

It does not require any laboratory-based procedures, according to Gallego-Perez, and can be used at the point of care a doctors office, say, or an outpatient clinic.

The paper describes experiments on mice and pigs. These included using the device to act upon badly injured legs that lacked blood flow. One week after the application of TNT, vascular vessels reappeared. Within a fortnight flow was back within normal parameters.

In a second experiment, skin cells were converted into nerve cells and introduced into the brains of mice crippled by stroke.

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

The concept is very simple, adds co-author James Lee: As a matter of fact, we were even surprised how it worked so well. In my lab, we have ongoing research trying to understand the mechanism and do even better. So this is the beginning, more to come.

Lee, Sen and Gallego-Perez were part of a group of researchers that lodged a patent application in 2016 for an earlier iteration of TNT: a device that enables compositions and methods for reprogramming somatic cells into induced endothelial cells.

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Nano-chip promises to heal organs at a touch | Cosmos – Cosmos

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