Category Archives: Gene Medicine

What happened

Shares of gene editing leader Editas Medicine (NASDAQ: EDIT) rose nearly 16% Monday on heavy trading volume, most of which occurred shortly after the market opened. The number of shares traded in the first few minutes of trading exceeded the daily average trading volume.

There was no major announcement. Instead, Editas Medicine decided to payoff a promissory note issued to the Broad Institute, the holder of its gene editing patents, by issuing shares of common stock on Friday, Aug. 4 that became the property of the research institution. The maximum conversion price was $21.49 per share, which was significantly higher than the recent stock price, and it appears the shares were sold Monday morning at that higher price. Therefore, the company's stock received a little boost.

Despite the honest explanation with almost no significance to Editas Medicine (it didn't receive proceeds from issuing the shares to the Broad Institute), the gains managed to stick around. As of 3:21 p.m. EDT, the stock had settled to a 10.6% gain.

Image source: Getty Images.

In early March, Editas Medicine granted the Broad Institute and Wageningen University promissory notes worth 800,000 shares of common stock related to licensing fees for specific gene editing patents. The notes were convertible to shares at the company's discretion or after 150 days. Since early August is about 150 days after early March, it appears the latter took place.

It's worth pointing out that the Broad Institute was issued 271,347 shares of common stock a few days ago. That's substantially less than the 800,000 shares spelled out in the March filing. However, it's not easy to tell if the balance was given to others named in the filing or if the research institution negotiated a lower payment from Editas Medicine.

Either way, the fact that the Broad Institute unloaded its shares from the converted promissory note is hardly worrisome. After all, the research institution is simply realizing a licensing fee from Editas Medicine. Monday's pop is nothing more, nothing less.

Sometimes there are simple reasons for major stock movements. That is certainly the case with Editas Medicine stock on Monday. But there's good news for investors hoping that more actionable events were the driver of Monday's pop: The gene editing pioneer announces second-quarter 2017 earnings this Wednesday, Aug. 9. That should provide more timely information for shareholders that cannot get enough updates.

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Here's Why Editas Medicine Rose as Much as 15.9% Monday - Madison.com

Chicago Tonight | WTTW

New Gene Editing Study Raises Possibilities, Questions Chicago Tonight | WTTW ... much harder for scientists to target genes associated with humor, creativity or physical traits). Cardiologist and geneticist Dr. Elizabeth McNally is the director of the Center for Genetic Medicine at Northwestern University. She joins Phil Ponce ... A Gene Editing BreakthroughWBUR Path breakingGulf Times Modification of genes in human embryos could mark turning point in human evolutionThe Globe and Mail Pittsburgh Post-Gazette -Gears Of Biz all 37 news articles »

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New Gene Editing Study Raises Possibilities, Questions - Chicago Tonight | WTTW

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 Pandoras 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 familys 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.

Embryos created in this study were used to answer pre- clinical questions about safety and effectiveness. The study noted that genome editing approaches must be further optimised before moving to clinical trials.

This research significantly advances scientific understanding of the procedures that would be necessary to ensure the safety and efficacy of germline gene correction, said Daniel Dorsa, senior vice president for research at OHSU.

The ethical considerations of moving this technology to clinical trials are complex and deserve significant public engagement before we can answer the broader question of whether its in humanitys interest to alter human genes for future generations.

Existing ethical guidelines did not permit the team to implant the genetically modified human embryos into women.

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Kashmiri doctor helps gene editing of human embryos in US - Hindustan Times

The secret to healing what ails you lies within your own DNA. (photo credit:DREAMSTIME)

Scientists have, for the first time, corrected a disease-causing mutation in early-stage human embryos using gene editing.

The technique, which uses the CRISPR- Cas9 system, corrected the mutation for a heart condition at the earliest stage of embryonic development so that the defect would not be passed on to future generations.

It could pave the way for improved in vitro fertilization outcomes as well as eventual cures for some thousands of diseases caused by mutations in single genes.

The breakthrough and accomplishment by American and Korean scientists, was recently explained in the journal Nature. Its a collaboration between the Salk Institute, Oregon Health and Science University and South Koreas Institute for Basic Science.

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, said Prof. Juan Carlos Izpisua Belmonte of Salks gene expression lab 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.

Though gene-editing tools have the power to potentially cure a number of diseases, scientists have proceeded cautiously partly to avoid introducing unintended mutations into the germ line (cells that become eggs or sperm).

Izpisua Belmonte is uniquely qualified to speak on the ethics of genome editing because, as a member of the Committee on Human Gene Editing at the US National Academies of Sciences, Engineering and Medicine, he helped author the 2016 roadmap Human Genome Editing: Science, Ethics and Governance.

Hypertrophic cardiomyopathy is the most common cause of sudden death in otherwise healthy young athletes, and affects approximately one in 500 people. It is caused by a dominant mutation in the MYBPC3 gene, but often goes undetected until it is too late. Since people with a mutant copy of the MYBPC3 gene have a 50% chance of passing it on to their own children, being able to correct the mutation in embryos would prevent the disease not only in affected children but also in their descendants.

The researchers generated induced pluripotent stem cells from a skin biopsy donated by a male with Hypertrophic cardiomyopathy and developed a gene-editing strategy based on CRISPR-Cas9 that would specifically target the mutated copy of the MYBPC3 gene for repair. The targeted mutated MYBPC3 gene was cut by the Cas9 enzyme, allowing the donors cells own DNA -repair mechanisms to fix the mutation during the next round of cell division by using either a synthetic DNA sequence or the non-mutated copy of MYBPC3 gene as a template.

Using IVF techniques, the researchers injected the best-performing gene-editing components into healthy donor eggs that are newly fertilized with donors sperm. All the cells in the early embryos are then analyzed at single-cell resolution to see how effectively the mutation was repaired.

They were surprised by the safety and efficiency of the method. Not only were a high percentage of embryonic cells get fixed, but also gene correction didnt induce any detectable off-target mutations and genome instability major concerns for gene editing.

The researchers also developed an effective strategy to ensure the repair occurred consistently in all the cells of the embryo, as incomplete repairs can lead to some cells continuing to carry the mutation.

Even though the success rate in patient cells cultured in a dish was low, we saw that the gene correction seems to be very robust in embryos of which one copy of the MYBPC3 gene is mutated, said Jun Wu, a Salk staff scientist and one of the authors.

This was in part because, after CRISPR- Cas9 mediated enzymatic cutting of the mutated gene copy, the embryo initiated its own repairs. Instead of using the provided synthetic DNA template, the team surprisingly found that the embryo preferentially used the available healthy copy of the gene to repair the mutated part.

Our technology successfully repairs the disease-causing gene mutation by taking advantage of a DNA repair response unique to early embryos, said Wu.

The authors emphasized that although promising, these are very preliminary results and more research will need to be done to ensure no unintended effects occur.

Our results demonstrate the great potential of embryonic gene editing, but we must continue to realistically assess the risks as well as the benefits, they added.

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Early gene-editing holds promise for preventing inherited diseases - The Jerusalem Post

FRIDAY, Aug. 4, 2017 (HealthDay News) -- Some adult survivors of childhood cancer go on to develop brain tumors, and now researchers say they've found a gene mutation that seems to increase that risk.

The researchers said their findings could lead to ways to prevent these brain tumors.

Adults who had radiation to their head and spine to treat childhood cancer have a greater risk of meningiomas. These tumors, which are often benign, are the most common type of brain tumor in adults, according to the American Brain Tumor Association.

In this study, Canadian researchers looked at 31 radiation-induced meningiomas in patients who underwent head and spine radiation during childhood. Most of them (74 percent) had survived either leukemia or pediatric brain cancer.

These brain tumors were compared with 30 meningiomas among people in the general population.

Gelareh Zadeh, the study's co-principal investigator, said that radiation-induced meningiomas appear to be the same as those that just occur sporadically. They look the same on MRI scans and under a microscope. And, they feel the same during surgery, Zadeh said.

"What's different is [that radiation-induced tumors] are more aggressive, tend to recur in multiples and invade the brain, causing significant morbidity and limitations (or impairments) for individuals who survive following childhood radiation," Zadeh said.

Zadeh is a brain tumor researcher and associate professor in the neurology division at the University of Toronto.

Ken Aldape is a co-principal investigator on the study. He said the research team found a specific rearrangement involving the NF2 gene in radiation-induced meningiomas. He said there are likely other genetic rearrangements caused by radiation-induced DNA damage.

"So one of the next steps is to identify what the radiation is doing to the DNA of the meninges," Aldape said in a University Health Network news release. Aldape is a professor of laboratory medicine and pathobiology at the University of Toronto.

Figuring out which group of childhood cancer patients have the highest risk of these radiation-induced tumors is critical. These patients could be followed closely for early detection and management, Aldape explained.

The study was published online Aug. 4 in the journal Nature Communications.

The American Brain Tumor Association has more on meningiomas.

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Childhood Cancer Radiation May Cause Unwanted Gene Mutation in Some - Sioux City Journal

August 4, 2017 Pictured are heart tissue sections showing a normal mouse heart (left) and one with heart failure (right). The tissue sections were stained to enhance visualization. The failing heart is larger, thinner, and contains a blood clot filling one of its atria (upper right chamber). Photo courtesy of the Grueter laboratory. Credit: Grueter laboratory, University of Iowa Health Care

Heart failure refers to a condition in which heart muscle becomes weakened over time, making it increasingly difficult for the heart to pump blood through the body like it should.

It's a progressive disease that begins when the heart adapts to stressorshigh blood pressure, coronary artery disease, or diabetes, for examplein order to work properly. These stressors can lead to dilated cardiomyopathy, in which the heart's left ventricle (pumping chamber) stretches, enlarges, and becomes thinner. Eventually, the heart cannot return to its normal shape, thus worsening its ability to pump blood and potentially leading to irregular heartbeats, blood clots, or even sudden death.

Researchers know that changes in gene expression occur during cardiomyopathy, but it remains unclear whether these changes are due to declining heart function or whether these changes are part of the progression to heart failure. A better understanding of the role transcription co-factorsproteins that are key to the regulation and expression of genescould provide important clues into how heart failure develops.

In a new study, University of Iowa Health Care researchers report on the role of a proteinpart of a large group of transcription co-factors called the Mediator complexin regulating gene expression in heart muscle cells.

"A key question is how does the heart go from a normal state to a failing one after undergoing stress in some manner?" says Duane Hall, research assistant professor of internal medicine in the UI Carver College of Medicine and lead author of the study published in the Aug. 3 issue of the journal JCI Insight. "A lot of labs are trying to understand how that progression occurs."

"It's known that many genes are expressed during heart failure that are representative of a developing heart, so in these instances the heart may be trying to re-install developmental programs in order to adapt to those pressures," adds Chad Grueter, assistant professor of internal medicine in the UI Carver College of Medicine and senior author of the study. "But we don't fully understand how that transcriptional gene regulation happens, so we looked at how gene expression occurs through this Mediator complex."

Grueter, Hall, and colleagues examined heart tissue samples from patients with heart failure and saw that levels of the protein Cdk8 in heart muscle cells were elevated. Knowing that Cdk8 is part of the Mediator complex and is involved in regulating the expression of thousands of genes, the researchers then over-expressed the protein in mouse heart cells. The increase in Cdk8 levels resulted in declining heart function and heart failure in these mice.

When the researchers examined the heart cells of the mice before a decrease in heart function was detectable, they found that more than 3,400 genes already were expressed with a profile similar to that of human heart muscle cells with dilated cardiomyopathy and heart failure.

"Other studies have looked at tweaking the contraction and metabolism in heart cells as a possible cure for heart failure," Hall says. "Our study is one of the first to show that something in the cell nucleus is capable by itself of inducing the structural changes that occur in heart failure."

The study results suggest that modifying gene expression may provide a path to preventive treatments for heart failure.

"In terms of disease progression, heart failure is the end stage. Our study suggests that the transition, or 'switch,' from a stressed, enlarged heart to a failing heart is key," Grueter says. "Looking ahead, hopefully we'll be able to test whether a drug can block that switch from occurring."

Explore further: Popular class of drugs reverse potentially harmful genetic changes from heart disease

More information: Duane D. Hall et al, Ectopic expression of Cdk8 induces eccentric hypertrophy and heart failure, JCI Insight (2017). DOI: 10.1172/jci.insight.92476

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Study examines altered gene expression in heart failure - Medical Xpress

Thats because none of those talents arise from a single gene mutation, or even from an easily identifiable number of genes. Most human traits are nowhere near that simple.

Right now, we know nothing about genetic enhancement, said Hank Greely, director of the Center for Law and the Biosciences at Stanford. Were never going to be able to say, honestly, This embryo looks like a 1550 on the two-part SAT.

Even with an apparently straightforward physical characteristic like height, genetic manipulation would be a tall order. Some scientists estimate height is influenced by as many as 93,000 genetic variations. A recent study identified 697 of them.

A new technique known as Crispr has revolutionized humans ability to edit DNA. See you if you can identify whether a given development has already happened, could eventually happen or is pure fiction.

You might be able to do it with something like eye color, said Robin Lovell-Badge, a professor of genetics and embryology at the Francis Crick Institute in London.

But if people are worried about designer babies, theyre normally thinking of doing special different things than the normal genetic stuff.

The gene-modification process used in the new study also turns out to be somewhat restrictive. After researchers snipped the harmful mutation from the male gene, it copied the healthy sequence from that spot on the female gene.

That was a surprise to the scientists, who had inserted a DNA template into the embryo, expecting the gene to copy that sequence into the snipped spot, as occurs with gene editing in other body cells. But the embryonic genome ignored that template, suggesting that to repair a mutation on one parents gene in an embryo, a healthy DNA sequence from the other parent is required.

If you cant introduce a template, then you cant do anything wild, Dr. Lovell-Badge said. This doesnt really help you make designer babies.

Talents and traits arent the only thing that are genetically complex. So are most physical diseases and psychiatric disorders. The genetic message is not carried in a 140-character tweet it resembles a shelf full of books with chapters, subsections and footnotes.

So embryonic editing is unlikely to prevent most medical problems.

But about 10,000 medical conditions are linked to specific mutations, including Huntingtons disease, cancers caused by BRCA genes, Tay-Sachs disease, cystic fibrosis, sickle cell anemia, and some cases of early-onset Alzheimers. Repairing the responsible mutations in theory could eradicate these diseases from the so-called germline, the genetic material passed from one generation to the next. No future family members would inherit them.

But testing editing approaches on each mutation will require scientists to find the right genetic signpost, often an RNA molecule, to guide the gene-snipping tool.

In the study reported this week, it took 10 tries to find the right RNA, said Juan Carlos Izpisua Belmonte, a co-author and geneticist at the Salk Institute.

Dr. Greely noted that while scientists work to get human embryonic editing ready for clinical trials (currently illegal in the United States and many countries), alternate medical treatments for these diseases might be developed. They may be simpler and cheaper.

How good one technique is depends on how good the alternatives are, and there may be alternatives, he said.

The authors of the new study do not dismiss ethical implications of their work. In fact, Dr. Belmonte served on a committee of the National Academies of Science, Engineering and Medicine that in February endorsed research into gene editing of human embryos, but only to prevent serious diseases and conditions, and as a last resort.

In theory this could lead to the kind of intervention which, of course, Im totally against, said Dr. Belmonte. The possibility of moving forward not to create or prevent disease but rather to perform gene enhancement in humans.

For example, soon we will know more and more about genes that can increase your muscle activity, he said. The hormone EPO, which some athletes have been disciplined for taking, is produced by a gene, so you could in theory engineer yourself to produce more EPO.

That is the kind of genetic engineering that raises alarm.

Allowing any form of human germline modification leaves the way open for all kinds especially when fertility clinics start offering genetic upgrades to those able to afford them, Marcy Darnovsky, executive director of the Center for Genetics and Society, said in a statement. We could all too easily find ourselves in a world where some peoples children are considered biologically superior to the rest of us.

Scientists and ethicists share the concerns about access. Any intervention that goes to the clinic should be for everyone, Dr. Belmonte said. It shouldnt create inequities in society.

Unequal access is, of course, a question that arises with almost any new medical intervention, and already disparities deprive too many people of needed treatments.

But there is a flip side to ethical arguments against embryo editing.

I personally feel we are duty bound to explore what the technology can do in a safe, reliable manner to help people, Dr. Lovell-Badge said. If you have a way to help families not have a diseased child, then it would be unethical not to do it.

Genetic engineering doesnt have to be an all or nothing proposition, some scientists and ethicists say. There is a middle ground to stake out with laws, regulation and oversight.

For example, Dr. Lovell-Badge said, Britain highly regulates pre-implantation genetic diagnosis, in which a couples embryos are screened for certain harmful mutations so that only healthy ones are implanted in the womans womb.

They allow sensible things to be done, and they dont allow non-sensible things, he said. And every single embryo is accounted for. If someone tries to do something they shouldnt have done, they will find out, and the penalties for breaking the law are quite severe.

According to a 2015 article in the journal Nature, a number of countries, including the United States, restrict or ban genetic modification of human embryos.

Other countries, like China, have guidelines but not laws banning or restricting clinical use, the article noted. Chinese researchers have conducted the only previously published gene editing experiments on human embryos, which were much less successful.

In the future, will there be nations that allow fertility clinics to promise babies with genetically engineered perfect pitch or .400 batting averages? Its not impossible. Even now, some clinics in the United States and elsewhere offer unproven stem cell therapies, sometimes with disastrous consequences.

But R. Alta Charo, a bioethicist at University of Wisconsin-Madison, who co-led the national committee on human embryo editing, said historically ethical overreach with reproductive technology has been limited.

Procedures like I.V.F. are arduous and expensive, and many people want children to closely resemble themselves and their partners. They are likely to tinker with genes only if other alternatives are impractical or impossible.

You hear people talking about how this will make us treat children as commodities and make people more intolerant of people with disabilities and lead to eugenics and all that, she said.

While I appreciate the fear, I think we need to realize that with every technology we have had these fears, and they havent been realized.

Nicholas Wade contributed reporting from New York.

A version of this news analysis appears in print on August 5, 2017, on Page A14 of the New York edition with the headline: Designer Babies Still Seem Unlikely.

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Gene Editing for 'Designer Babies'? Highly Unlikely, Scientists Say - New York Times

Unintended Effects

In a statement to Catholic News Agency earlier this week, Rev.Tadeusz Pacholczyk, Ph.D., director of education for the National Catholic Bioethics Center, expressed moral objection to germline genome editing on embryos: Their value as human beings is profoundly denigrated every time they are created, experimented upon, and then killed. Moreover, if such embryos were to grow up, as will doubtless occur in the future, there are likely to be unintended effects from modifying their genes.

The 11 organizations acknowledged numerous ethical issues arising from human germline genome editing, including:

At a minimum, the potential for harm to individuals and families, ramifications on which we can only speculate, provide a strong argument for prudence and further research, the policy statement asserted. By proceeding with caution, we can ensure better understanding of the potential risks and benefits of gene editing from a scientific perspective and, as such, provide families with a more fulsome exercise of their autonomous decision making through the consent process.

The statement added: We encourage ethical and social consideration in tandem with basic science research in the upcoming years.

Last October, You Lu, M.D., and colleagues at Sichuan Universitys West China Hospital in Chengdu launched the first known clinical trial using CRISPR to treat patientsspecifically, knocking out a gene encoding the programmed death protein 1 (PD-1) in patients with non-small-cell lung cancer.

Groups joining ASHG in issuing the policy statement included the Association of Genetic Nurses and Counsellors, the Canadian Association of Genetic Counsellors, the International Genetic Epidemiology Society, and the National Society of Genetic Counselors.

Additional groups authoring the policy statement were the American Society for Reproductive Medicine, the Asia Pacific Society of Human Genetics, the British Society for Genetic Medicine, the Human Genetics Society of Australasia, the Professional Society of Genetic Counselors in Asia, and the Southern African Society for Human Genetics.

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11 Organizations Urge Caution, Not Ban, on CRISPR Germline Genome Editing - Genetic Engineering & Biotechnology News (press release)

THURSDAY, Aug. 3, 2017 (HealthDay News) -- Scientists have created genetically altered skin cells that may control type 2 diabetes in lab mice. And they believe the general concept could someday be used to treat various diseases.

Using a combination of stem cells and "gene editing," the researchers created patches of skin cells that were able to release a hormone called GLP1 in a controlled manner.

The hormone, which is normally produced in the digestive tract, spurs the production of insulin -- the body's key regulator of blood sugar levels.

The scientists found that transplanting the engineered skin patches onto diabetic lab mice helped regulate their blood sugar levels over four months.

Xiaoyang Wu, a stem cell biologist at the University of Chicago, led the "proof of concept" study. He said it raises the possibility that "therapeutic skin grafts" could be used to treat a range of diseases -- from hemophilia to drug dependence.

Wu's team focused on type 2 diabetes in these initial experiments because it's a common condition.

However, a researcher not involved in the study doubted the usefulness of the approach for diabetes specifically.

People with type 2 diabetes already manage the disease with diet, exercise and medications -- including ones that target GLP1, said Juan Dominguez-Bendala.

Using high-tech gene therapy to get the same result seems unlikely, said Dominguez-Bendala, an associate professor at the University of Miami's Diabetes Research Institute.

"I don't see something like this coming to the clinic for diabetes," he said.

But Dominguez-Bendala also pointed to what's "cool" about the experiments.

Wu's team used a recently developed technology called CRISPR (pronounced "crisper") to create the skin patches. The technique, heralded as a major breakthrough in genetic engineering, allows scientists to make precision "edits" in DNA -- such as clipping a particular defect or inserting a gene at a specific location.

Before CRISPR, scientists could not control where an inserted gene would be integrated into the genome. It might end up in a "bad" location, Dominguez-Bendala explained, where it could, for example, "awaken" a tumor-promoting gene.

Wu and colleauges used CRISPR to make specific edits in GLP1, including one that allowed the gene to be turned "on" or "off" as needed, by using the antibiotic doxycycline.

The modified gene was inserted into mouse stem cells, which were then cultured into skin grafts in the lab. Finally, those grafts were transplanted onto lab mice.

The researchers found that when the mice were fed food with tiny amounts of doxycycline, the transplanted skin released GLP1 into the bloodstream. In turn, the animals' insulin levels rose and their blood sugar dipped.

The engineered skin also seemed to protect the mice from the ravages of a high-fat diet. When the mice were fed a fat-laden diet, along with doxycycline, they gained less weight versus normal mice given the same diet. They also showed less resistance to the effects of insulin, and lower blood sugar levels.

According to Wu, the study lays the groundwork for more research into using skin cells as a way to deliver "therapeutic proteins."

For instance, he said, skin cells could be engineered to provide an essential protein that is missing because of a genetic defect. As an example, he cited hemophilia -- a genetic disorder in which people lack a protein that allows the blood to clot properly.

Skin cells could be an ideal way to deliver such therapies, Wu said.

For one, the safety of skin grafts in humans is well-established, he pointed out. Since the 1970s, doctors have known how to harvest skin stem cells from burn victims, then use those cells to create lab-grown skin tissue.

Because the skin is generated from a patient's own stem cells, that minimizes the issue of an immune system attack on the tissue.

Dominguez-Bendala agreed that using skin cells has advantages. For one, he noted, the skin graft can be easily removed if something goes awry.

But a lot of work remains before therapeutic skin grafts could become a reality for any human disease. And research in animals doesn't always pan out in humans.

A next step, Wu said, is to see whether the skin grafts maintain their effects in lab mice over a longer period. The researchers will also monitor the animals for any immune system reactions against the GLP1 protein itself.

The findings were published online Aug. 3 in Cell Stem Cell.

The U.S. National Institutes of Health has a primer on gene therapy.

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Engineered Skin Cells Control Type 2 Diabetes in Mice: Study - Sioux City Journal

August 3, 2017 6:22 PM

NEW YORK (CBSNewYork) A major scientific breakthrough was announced this week, and it could wipe out certain diseases.

As CBS2s Dr. Max Gomez reported, researchers for the first time have fixed a faulty gene in a human embryo. The genetic engineering procedure could be revolutionary, but people are afraid of how it might be used.

Genetic engineering has been around for some years, but using it actually to repair DNA mutations that cause disease is very hard to do.

But now, in a landmark study, researchers from the U.S., China, and Korea have fixed a faulty gene that causes a lethal heart condition.

Medical science has known for many years that certain serious diseases are caused by genetic mutations that are passed along from generation to generation. Researchers have long dreamed of repairing those errors in DNA.

The goal of preventing and permanently eliminating terrible genetic diseases were talking here cystic fibrosis, Tay-Sachs disease, sickle cell, hemophilia thats been a long-sought goal of medicine, said Dr. Arthur Caplan of NYU Langone Medical Center.

The goal just took a big step toward reality with a study in the journal Nature. It involved using a sperm cell from a male with a mutation known to cause a lethal heart muscle condition, which was used to fertilize an egg without the mutation.

The key was using a new gene-editing technique called Clustered Regularly Interspaced Short Palindromic Repeats or CRISPR that found and removed the defective gene.

The unexpected result was that the fertilized egg used the copy of the normal gene from the mothe to repair and replace the edited gene.

And it worked in most of the embryos tested, according to Eric Schadt, dean for precision medicine at the Icahn School of Medicine at Mount Sinai Hospital.

The efficiency of the overall procedure was much higher than earlier, Schadt said. Over 70 percent of the embryos that were targeted were corrected, so it just brought it much closer to prime time and being able to think about its use in clinical applications.

But like most powerful technologies, the potential for good comes with the possibility for abuse.

You dont just fix diseases. You wind up trying to improve or enhance our offspring. You try to make super babies, Caplan said. Whos going to guarantee access? Whos going to try and guarantee reasonable pricing. Theres a lot of money to be made out here.

Another caution that both Caplan and Schadt pointed out is that while the embryos looked normal, they were only allowed to develop for a few days in a petri dish.

That is a long way from knowing that an engineered embryo would yield a normal baby. Information about safety is not known.

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Embryonic Gene Editing Could Wipe Out Several Conditions, Researchers Say - CBS New York