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Category Archives: Oregon Stem Cells

Portland, Oregon Stem Cell Transplants, West Haven Sylvan …

Posted: August 18, 2018 at 9:46 am

Portland, the largest city in Oregon and seat of Multnomah County, is now part of the development of an innovative medical weapon for combating diseases. Billions are being spent all over the world in research to explore the normal mechanisms whereby stem cells build, maintain and repair our body systems. Many viable therapies in Portland are already in clinical trial face. Diseases like Diabetes, Multiple Sclerosis, Osteoarthritis, Parkinsons diseases and many others are part of these trials.

Stem cells can be converted into different cell types such as nerve cells, liver cells or heart cells stem cells can be used to replace cells damaged by injury or illness. The potential is so great, and the discovery so revolutionary, the research made headlines around the world, sparking more than 2,000 news reports from Japan to South Africa.

Whether you live in West Haven Sylvan, West Linn, Rivergrove, Raleigh Hills, Oceanside or any other city in Oregon, now you can access approved Stem Cell Treatments, dont hesitate to contact us and get more information.

The Stem Cells Transplant Institute of Costa Rica specializes in the legal treatment of, Chronic Obstructive pulmonary disease, Osteoarthritis, Knee Injury, Diabetes, Neuropathy, Cardiovascular Disease, Myocardial infarction, Critical limb isquemia, Parkinson, Multiple Sclerosis, Lupus, Rheumatoid Arthritis, and Alzheimer.

Stem Cells therapies have been studied for years for their impressive curative potential, but there are only a few clinics legally approved in the USA so far. Stem Cells Transplant Institute in Costa Rica wants to offer legally approved therapies, so you wont have to wait years to hear about how Regenerative Medicine has changed some one else quality of live. Are you ready to be a living proof? Apply here

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Oregon Stem Cell Center Result of OHSU Research Strides …

Posted: July 26, 2018 at 3:49 pm

Portland, Ore.

New hub to focus on adult stem cells as organ transplant alternative

Oregon Health & Science University's fast-growing stem cell research program, which already has made significant strides in the hunt for human disease therapies, now has a place to call home.

The multidisciplinary Oregon Stem Cell Center, the first of its kind in the Northwest, is studying cell and gene therapy as an alternative to organ transplantation for pancreatic and liver disorders, diabetes, cancer and a host of other human diseases. Its focus will be on adult stem cells.

Launched in January, the center is housed among several laboratories on the Marquam Hill Campus, but it will occupy a single floor in the 270,000-square-foot Biomedical Research Building now under construction and to be completed in fall 2005.

Its director, Markus Grompe, M.D., professor of molecular and medical genetics, and pediatrics in the OHSU School of Medicine, said the center is a hub for all areas of OHSU's rapidly expanding stem cell biology program. It aims to maximize the potential of various stem cells as therapies for human diseases through a rapid, "bench-to-bedside" approach involving basic research in stem cell biology and preclinical trials of therapies in animal models, followed by human trials.

"This is something we needed but didn't have," said Grompe, who has long studied gene and cell therapy for metabolic genetic diseases, as well as the molecular genetics of a rare, cancer-susceptibility syndrome called Fanconi anemia. "The consensus here at OHSU is that this is an area that is going to be important."

While the center's offerings will widen over time to cover other diseases -- Parkinson's disease and diabetes are considered "hot" areas of stem cell research -- it will concentrate on two areas for now and "build on that."

"My idea is to focus on the liver and the pancreas, and focus on adult stem cells," he said. "Our research already is advanced in liver reconstitution by stem cells and the repair of liver disease. We're clearly identified as one of the leaders in that area."

OHSU researchers like Grompe are wasting no time demonstrating the importance of stem cells. His laboratory is renowned as an international leader in cell therapy for liver and pancreas diseases as an alternative to organ transplantation.

Last year, Grompe published a study showing that bone marrow-derived stem cells from mice can combine with liver cells through cell fusion, which occurs when two or more cells combine to form one cell containing more genetic material than normal. The method reversed liver damage.

Other studies indicate bone marrow stem cells can meld with cells of other tissues, such as brain, spinal cord, lung, intestine and heart muscle.

The center's formation reflects a swell of research at OHSU involving stem cells. Scientists are conducting basic research, including molecular-level studies, in cardiology, endocrinology, genetics, hematology and oncology, neurology, neurological and general surgery, and reproduction.

"Basically all diseases which are currently being treated by organ transplantation are, at least on paper, amenable to cell therapy," Grompe said. "Our hope is that procedures as effective as whole organ transplantation will come out of (the center)."

Of particular interest to School of Medicine scientists are recently discovered clonally self-renewing stem cells. These unique cells have energized stem cell research - and are broadening the discipline's appeal to a larger group of scientists - because of their ability to generate copies of themselves and further divide into special-purpose offspring.

Clonally self-renewing stem cells come in several forms, such as mesenchymal stem cells, neural stem cells and multipotent progenitor cells, and can be used to create multiple cell types, including nerve cells, liver cells and muscle cells. They can be isolated from mice and primates, including humans, manipulated outside the living organism, and transplanted for reconstituting tissue.

"Their ability to be expanded in culture and then differentiated make them attractive for use in cell therapy," Grompe said.

Dan Dorsa, Ph.D., OHSU vice president for research and professor of physiology and pharmacology in the School of Medicine, said stem cells hold promise for treating many disorders. As a result, OHSU has the potential to make "a very broad impact."

"The use of stem cells for treating diseases will be at the forefront," Dorsa said. "The question we want to answer is: What is it about stem cells that allow them to be viable and take on the roles in the body you hope they will?"

The Oregon facility is one of only a handful of stem cell research centers around the country. Other sites include the University of Minnesota, Stanford University, the University of California, San Francisco, and the University of California, San Diego.

The heart of the center is three core facilities that provide cell development and management services for all campus research. They include:

A flow-sorting core to identify and isolate stem cell populations and characterize their progeny using fluorescence. Its primary tool, a fluorescence-activated cell sorter, "fishes out living stem cells and keeps them alive for transplantation and study," Grompe said.

A cell isolation core to culture, store and distribute specific stem cells. This will allow many researchers at OHSU rapid and easy access to professionally isolated and maintained, high-quality stem cell sources.

A monoclonal antibody production core to develop the large quantities of novel antibodies needed for identifying and purifying specific stem cells. Such a service has not been commercially available. "We'll be able to give cells to the core and get antibodies back for researchers," Grompe said. "And the antibodies don't have to be against stem cells to be effective."

The monoclonal antibody production core will be especially useful to cancer researchers, said Grover Bagby Jr., M.D., professor of molecular and medical genetics, OHSU School of Medicine, and director of the OHSU Cancer Institute.

"Having the capacity to make antibodies is going to be a nice core to have," he said. "I think a good number of cancer researchers will come to use that core. It'll be used right out of the gate."

And that could help scientists better track the progression of cancer, most forms of which are mutant outgrowths of stem cells.

"Understanding the cause of cancer definitely leads squarely into the ballpark of stem cells. We know it's true of leukemia and I suspect it's probably true of all other tissues," Bagby said. "There are a lot of things we can learn about stem cells that can lead to an understanding of how to protect them."

Dorsa and Grompe hope the center bolsters the development of OHSU-born spinoff companies while enhancing the university's partnerships with local and national biotechnology firms. It also could make OHSU more of a target for federal grants.

"There are very likely new industries that will be created by virtue of the new activity of the center," Dorsa said. The antibody core, for example, "will be attractive to commercialization."

The Oregon Stem Cell Center is funded by a three-year, $4.5 million grant from the Oregon Opportunity, the statewide, $500 million biomedical research funding initiative supported by public and private dollars. Three faculty members specializing in stem cell research also will be hired during the next two years.

Dorsa believes the Oregon Stem Cell Center fits in well with the National Institutes of Health's "Roadmap" initiative, which strives to accelerate fundamental discovery and translation of that knowledge into effective prevention strategies and new treatments.

"NIH dollars will be attracted by the stem cell center and the investments it will create," Dorsa said. "We think this one will be well positioned to compete for those dollars."

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Stem Cells to Relieve Low Back Pain? WebMD

Posted: October 14, 2017 at 2:21 am

By Brenda Goodman

HealthDay Reporter

FRIDAY, April 12 (HealthDay News) -- Medical researchers are trying a new treatment for low back pain. Their hope is that harvesting and then re-injecting the body's own bone marrow -- which is rich in stem cells -- may repair worn-out discs in the spine.

In a small new study, the approach appeared to be safe -- and none of the patients reported that their pain got worse after the procedure.

But both the doctors who are testing the technique and outside experts say much more research is needed before they can say whether the treatment offers real relief.

"I tell everybody that this is experimental, with a capital E," said Dr. Joseph Meyer Jr., an anesthesiologist and pain medicine specialist at the Columbia Interventional Pain Center, in St. Louis. "We don't know if it works. I do believe that it's safe, but it might not do anything for you."

For the study, Meyer and his colleagues reviewed the case histories of 24 patients who were injected with their own bone marrow aspirate cellular concentrate (BMAC). Bone marrow concentrate contains adult stem cells, which have been called the body's own repair kit because they can change into -- and potentially heal -- different kinds of tissues.

Meyer's patients reported suffering from chronic low back pain for anywhere from three months to 12 years. Imaging tests showed that all the patients had some evidence of degeneration, or damage, to the discs that cushion the bones of the spine. Disc degeneration is common with age, and it is thought to be a major cause of low back pain.

Many times, exercise and weight loss can help people with persistent low back pain. But if conservative approaches fail and the pain becomes debilitating, Meyer said, the next option is invasive spinal fusion surgery.

"Fusion is a big, big step with questionable effectiveness," he said. "Often, you're back in the same boat a year later."

Meyer said he offered patients the bone marrow treatment as something to try before resorting to surgery.

For the procedure, he used a long needle to extract bone marrow from the back of the hip. The bone marrow was spun in a centrifuge to concentrate the cells and then injected into the space around a damaged disc. Meyer said the treatment costs a few thousand dollars and is not covered by insurance.

Of the 24 patients who initially received the bone marrow injections, half went on to have other procedures over the next 30 months, making it impossible to know what might have affected their back pain.

Of the 12 who had no other kinds of treatment, 10 reported that their pain lessened in the two to four months after their injections. After a year, eight patients were still reporting significant pain relief, while three said their back pain had not improved. One patient had not yet reached the 12-month mark. After two years, five said their back pain was better, and three had no improvement. For the other four, it was still too early to tell.

Meyer said none of the 24 patients who tried the technique had complications from their procedures, but injections always carry the risk of infection.

The study was scheduled for Thursday presentation at the annual meeting of the American Academy of Pain Medicine in Fort Lauderdale, Fla. Studies presented at scientific conferences usually haven't been scrutinized by independent experts, and their results are considered preliminary.

An expert who was not involved in the study said people with back pain shouldn't get too excited about these results, particularly since there was no control group used for comparison.

"Low back pain often gets better over time," said Dr. Richard Deyo, a professor of evidence-based medicine and a back pain expert at Oregon Health and Sciences University, in Portland. "Even patients who have chronic pain, their symptoms tend to wax and wane and fluctuate. They seek care when their symptoms are worst, and very often they drift back to their average level of pain, which looks like improvement."

"People grasp at straws, and they shouldn't. We have a long history of treatments that look promising when they start and turn out to be no more effective than placebo interventions," said Deyo, who also is deputy editor of the journal Spine. "We also have a history of treatments that, in some cases, turned out to be harmful. It's really too early to know if this is going to be effective or safe."

The study's authors agreed. They said they hope this pilot project will encourage more research.

"We hope it will get people thinking and hopefully promote a future controlled study," Meyer said.

WebMD News from HealthDay

SOURCES: Joseph Meyer Jr., M.D., Ph.D., anesthesiologist and pain-medicine specialist, Columbia Interventional Pain Center, St. Louis; Richard Deyo, M.D., M.P.H., Kaiser-Permanente endowed professor of evidence-based medicine, department of family medicine, Oregon Health and Sciences University, Portland, Ore.; April 11, 2013, presentation, American Academy of Pain Medicine annual meeting, Fort Lauderdale, Fla.

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Stem Cells, Fetal Tissue Research & Cloning – Oregon Right …

Posted: August 25, 2017 at 6:45 am

Stem cell research is a controversial issue that sharply divides people. There are two kinds of stem cell research: embryonic and adult. This distinction is imperative because of the ethical issues involved.

Embryonic stem cell research requires cells to be extracted from a human embryo. In the process of extracting the stem cells the embryo is destroyed and a life is ended. The embryonic stem cells are then isolated and theoretically coaxed into developing into just about any cell in the body. Embryonic stem cell transplants have not been shown concretely to have successfully helped a single patient.

Fetal tissue research requires the abortion of a living unborn child. The Center for Medical Progress, in a series of investigative videos (link to site), revealed that Planned Parenthoods affiliated clinics participate in the harvesting and sale of aborted baby body parts and placental tissue for financial gain. These are then used in research facilities around the country, including at Oregon Health and Science University. Fetal tissue research has also not successfully helped treat a single patient.

Adult stem cell research on the other hand, does not require the destruction of life. Adult stem cells are derived from sources like umbilical cords and organ tissue.

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Stem Cells, Fetal Tissue Research & Cloning - Oregon Right ...

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New Biomedical Engineering Grants Aim at Heart Failure and Resistant High Blood Pressure – Newswise (press release)

Posted: August 25, 2017 at 6:45 am

Newswise BIRMINGHAM, Ala. Biomedical engineering researchers will attack two banes of cardiovascular disease heart failure after heart attacks and the scourge of resistant high blood pressure with $4.8 million in National Institutes of Health grants that begin this fall.

One sign of the clinical significance of this research by the University of Alabama at Birmingham investigators are the percentile scores that Jianyi Jay Zhang, M.D., Ph.D., and Gangjian Qin, M.D., received in those two NIH grant applications.

Zhangs plan to dissect the mechanisms of electromechanical integration of a human heart-muscle patch to aid survival and stability of the patch garnered a 1 percentile score, the highest possible. Qins plan to dissect a novel molecular pathway in endothelial cells of arteries that appears to regulate contractile function and blood pressure has significant potential to improve human health from the disease and death caused by high blood pressure, NIH reviewers said, and Qin received a 2 percentile score.

Zhang, chair and professor of the UAB Department of Biomedical Engineering and holder of the T. Michael and Gillian Goodrich Endowed Chair of Engineering Leadership, will receive $2.5 million over four years. Qin, professor of biomedical engineering and director of the Molecular Cardiology Program, will receive $2.3 million over four years.

Zhang came to UAB in 2015 from the University of Minnesota Medical School with the goal of moving his work with engineered heart patches into human use within seven years. As chair of Biomedical Engineering, a joint department of the UAB School of Medicine and the UAB School of Engineering, Zhang has recruited top researchers, and he also was awarded $11 million of NIH funding in 2016 $8 million of which is shared in collaborations with University of Wisconsin and Duke University researchers.

One of the recent recruits to biomedical engineering is Qin, who serendipitously discovered a novel and fascinating line of research that may lead to new drugs for treatment-resistant high blood pressure, where existing blood pressure drugs are ineffective. People with resistant high blood pressure have increased risk of strokes, heart attacks, heart failure and arterial aneurysms, and high blood pressure is a leading cause of chronic kidney failure. Even moderately elevated arterial blood pressure shortens life expectancy.

At the time, Qin was interested in the often fatal heart failure that occurs months or years after heart attacks. He reasoned that growth of new blood vessels into the damaged heart tissue of the left ventricle could be boosted by altering the amounts of cell-cycle regulators in the E2F family of transcription factors, to speed division of cells in the endothelial tissue of arteries.

When he deleted one of the eight E2Fs that are found in mice and humans E2F2 it had no effect on cell growth. But unexpectedly, we found a striking function, Qin said. If you delete E2F2, the vessel is more contractile. It becomes rigid and hard, and this contributes to high blood pressure.

So we had a question: How does E2F2 interact with other molecules to regulate blood pressure? Qin did pull-down experiments with E2F2, where other proteins are flowed past tethered E2F2 molecules to see if any would bind. He found that a kinase enzyme called Sam68 did bind to the transcription factor.

When he knocked out the gene for Sam68 in mice, they had low blood pressure.

Ultimately, a series of experiments in Qins lab and observations of other laboratories suggested a previously unknown mechanism of blood pressure control that involves E2F2/Sam68 and the expression of endothelial converting enzyme 1b. ECE-1b affects the levels of peptides that constrict blood vessels and raise blood pressure. Dysregulation of this pathway may contribute to blood pressure disorders, especially hypertension.

Despite a strong correlation, the E2F2/Sam68-ECE-1b pathway has not explicitly been linked to blood pressure regulation, and the mechanisms of how Sam68/E2F2 signaling regulates ECE-1b expression and blood vessel function remain uncharacterized.

Qin will use his new grant to search for the link to blood pressure regulation and characterize the mechanisms. His research could provide the missing links between the results of large-scale genomewide association studies of human high blood pressure and its pathogenesis namely how dysregulation leads to refractory hypertension.

Detailed knowledge of those steps would offer new targets for potential new drugs, which are especially needed to prevent or treat resistant hypertension.

Qin says he was attracted to UAB by the strong focus of clinicians and basic scientists on solving the clinical problem of hypertension, as well as the depth and breadth of cardiovascular disease research in biomedical engineering, the UAB Department of Pathology and the UAB Division of Cardiovascular Disease. He also has great interest in Zhangs research, where Qins past work in stem cell biology and cardiovascular science can contribute.

As measured by NIH funding, the UAB Department of Biomedical Engineering is the fourth-ranked biomedical engineering department among all departments that are jointly led by schools of medicine and engineering, according to the 2016 Blue Ridge NIH database.

The joint biomedical engineering departments ahead of UAB are at Stanford University, Johns Hopkins University, and Oregon Health and Science University. Those trailing UAB in the funding ranking are at the University of North Carolina-Chapel Hill, Emory University, University of Virginia, Case Western Reserve University, University of Colorado-Denver, University of Rochester, the University of Illinois-Chicago, Wake Forest University Health Sciences and State University of New York-Stony Brook.

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New Biomedical Engineering Grants Aim at Heart Failure and Resistant High Blood Pressure - Newswise (press release)

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Gene editing used to repair diseased genes in embryos – NHSUK – NHS Choices

Posted: August 25, 2017 at 6:45 am

Deadly gene mutations removed from human embryos in landmark study, reports The Guardian. Researchers have used a gene-editing technique to repair faults in DNA that can cause an often-fatal heart condition called hypertrophic cardiomyopathy.

This inherited heart condition is caused by a genetic change (mutation) in one or more genes. Babies born with hypertrophic cardiomyopathy have diseased and stiff heart muscles, which can lead to sudden unexpected death in childhood and in young athletes.

In this latest study researchers used a technique called CRISPR-cas9 to target and then remove faulty genes. CRISPR-cas9 acts like a pair of molecular scissors, allowing scientists to cut out certain sections of DNA. The technique has attracted a great deal of excitement in the scientific community since it was released in 2014. But as yet, there have been no practical applications for human health.

The research is at an early stage and cannot legally be used as treatment to help families affected by hypertrophic cardiomyopathy. And none of the modified embryos were implanted in the womb.

While the technique showed a high degree of accuracy, its unclear whether it is safe enough to be developed as a treatment. The sperm used in the study came from just one man with faulty genes, so the study needs to be repeated using cells from other people, to be sure that the findings can be replicated.

Scientists say it is now important for society to start a discussion about the ethical and legal implications of the technology. It is currently against the law to implant genetically altered human embryos to create a pregnancy, although such embryos can be developed for research.

The study was carried out by researchers from Oregon Health and Science University and the Salk Institute for Biological Studies in the US, the Institute for Basic Science and Seoul University in Korea, and BGI-Shenzen and BGI-Quingdao in China. It was funded by Oregon Health and Science University, the Institute for Basic Science, the G. Harold and Leila Y. Mathers Charitable Foundation, the Moxie Foundation and the Leona M. and HarryB. Helmsley Charitable Trust and the Shenzhen Municipal Government of China. The study was published in the peer-reviewed journal Nature.

The Guardian carried a clear and accurate report of the study. While their reports were mostly accurate, ITV News, Sky News and The Independent over-stated the current stage of research, with Sky News and ITV News saying it could eradicate thousands of inherited conditions and the Independent claiming it opens the possibility for inherited diseases to be wiped out entirely. While this may be possible, we dont know whether other inherited diseases might be as easily targeted as this gene mutation.

Finally, the Daily Mail rolls out the arguably tired clich of the technique leading to designer babies, which seems irrelevant at this point. The CRISPR-cas9 technique is only in its infancy and (ethics aside) its simply not possible to use genetic editing to select desirable characteristics - most of which are not the result of one single, identifiable gene. No reputable scientist would attempt such a procedure.

This was a series of experiments carried out in laboratories, to test the effects of the CRISPR-Cas9 technique on human cells and embryos. This type of scientific research helps us understand more about genes and how they can be changed by technology. It doesnt tell us what the effects would be if this was used as a treatment.

Researchers carried out a series of experiments on human cells, using the CRISPR-cas9 technique first on modified skin cells, then on very early embryos, and then on eggs at the point of fertilisation by sperm. They used genetic sequencing and analysis to assess the effects of these different experiments on cells and how they developed, up to five days. They looked specifically to see what proportion of cells carrying faulty mutations could be repaired, whether the process caused other unwanted mutations, and whether the process repaired all cells in an embryo, or just some of them.

They used skin cells (which were modified into stem cells) and sperm from one man, who carried the MYBPC3 mutation in his genome, and donor eggs from women without the genetic mutation. This is the mutation known to cause hypertrophic cardiomyopathy.

Normally in such cases, roughly half the embryos would have the mutation and half would not, as theres a 50-50 chance of the embryo inheriting the male or female version of the gene.

The CRISPR-cas9 technique can be used to select and delete specific genes from a strand of DNA. When this happens, usually the cut ends of the strand join together, but this causes problems so cant be used in the treatment of humans. The scientists created a genetic template of the healthy version of the gene, which they introduced at the same time as using CRISPR-cas9 to cut the mutated gene. They hoped the DNA would repair itself with a healthy version of the gene.

One important problem with changing genetic material is the development of mosaic embryos, where some of the cells have corrected genetic material and others have the original faulty gene. If that happened, doctors would not be able to tell whether or not an embryo was healthy.

The scientists needed to test all the cells in the embryos produced in the experiment, to see whether all cells had the corrected gene or whether the technique had resulted in a mixture. They also did whole genome sequencing on some embryos, to test for unrelated genetic changes that might have been introduced accidentally during the process.

All embryos in the study were destroyed, in line with legislation about genetic research on embryos.

Researchers found that the technique worked on some of the stem cells and embryos, but worked best when used at the point of fertilisation of the egg. There were important differences between the way the repair worked on the stem cells and the egg.

Only 28% of the stem cells were affected by the CRISPR-cas9 technique. Of these, most repaired themselves by joining the ends together, and only 41% were repaired by using a corrected version of the gene.

67% of the embryos exposed to CRISPR-cas9 had only the correct version of the gene higher than the 50% that would have been expected had the technique not been used. 33% of embryos had the mutated version of the gene, either in some or all their cells.

Importantly, the embryos didnt seem to use the template injected into the zygote to carry out the repair, in the way the stem cells did. They used the female version of the healthy gene to carry out the repair, instead.

Of the embryos created using CRISPR-cas9 at the point of fertilisation, 72% had the correct version of the gene in all their cells, and 28% had the mutated version of the gene in all their cells. No embryos were mosaic a mixture of cells with different genomes.

The researchers found no evidence of mutations induced by the technique, when they examined the cells using a variety of techniques. However, they did find some evidence of gene deletions caused by DNA strands splicing (joining) themselves together without repairing the faulty gene.

The researchers say they have demonstrated how human embryos employ a different DNA damage repair system to adult stem cells, which can be used to repair breaks in DNA made using the CRISPR-cas9 gene-editing technique.

They say that targeted gene correction could potentially rescue a substantial portion of mutant human embryos, and increase the numbers available for transfer for couples using pre-implantation diagnosis during IVF treatment.

However, they caution that despite remarkable targeting efficiency, CRISPR-cas9-treated embryos would not currently be suitable for transfer. Genome editing approaches must be further optimised before clinical application can be considered, they say.

Currently, genetically-inherited conditions like hypertrophic cardiomyopathy cannot be cured, only managed to reduce the risk of sudden cardiac death. For couples where one partner carries the mutated gene, the only option to avoid passing it on to their children is pre-implantation genetic diagnosis. This involves using IVF to create embryos, then testing a cell of the embryo to see whether it carries the healthy or mutated version of the gene. Embryos with healthy versions of the gene are then selected for implantation in the womb.

Problems arise if too few or none of the embryos have the correct version of the gene. The researchers suggest their technique could be used to increase the numbers of suitable embryos. However, the research is still at an early stage and has not yet been shown to be safe or effective enough to be considered as a treatment.

The other major factor is ethics and the law. Some people worry that gene editing could lead to designer babies, where couples use the tool to select attributes like hair colour, or even intelligence. At present, gene editing could not do this. Most of our characteristics, especially something as complex as intelligence, are not the result of one single, identifiable gene, so could not be selected in this way. And its likely that, even if gene editing treatments became legally available, they would be restricted to medical conditions.

Designer babies aside, society needs to consider what is acceptable in terms of editing human genetic material in embryos. Some people think that this type of technique is "playing God" or is ethically unacceptable because it involves discarding embryos that carry faulty genes. Others think that its rational to use the scientific techniques we have developed to eliminate causes of suffering, such as inherited diseases.

This research shows that the questions of how we want to legislate for this type of technique are becoming pressing. While the technology is not there yet, it is advancing fast. This research shows just how close we are getting to making genetic editing of human embryos a reality.

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The Public Needs to Weigh In on the Ethics of Genetically Engineering Humans – Slate Magazine

Posted: August 17, 2017 at 3:45 am

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On Aug. 3, the scientific article in Nature finally gave us some facts about the much-hyped experiments that involved editing the genomes of human embryos at the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University. The story had broken in late July in Technology Review, spurring profuse hand-wringing and discussion. But until we saw the scientific paper, it was not clear what cells and methods were used, what genes were edited, or what the results were.

Now we know more, and while the paper demonstrates the possibility of genome editing of human embryos, it raises more questions than it answers. It is a useful demonstration of technical promise, though not an immediate prelude to the birth of a genome-edited baby. But the process by which the news emerged is also an ominous harbinger of the discombobulated way the debate about genetically altering human embryos is likely to unfold. We need open, vigorous debate that captures the many, often contradictory, moral views of Americans. Yet what we are likely to get is piecemeal, fragmented stories of breakthroughs with incomplete details, more sober publication in science journals that appear later, news commentary that lasts a few days, and very little systematic effort to think through what policy should be.

The science underlying this news cycle about human genome editing builds on a technique first developed six years ago by studying how bacteria alter DNA. CRISPR genome editing is the most recent, and most promising, way to introduce changes into DNA. It is faster, easier, and cheaper than previous methods and should eventually be more precise and controllablewhich is why it may one day be available for clinical use in people.

Though headlines about the study discussed designer babies, researchers prefer to emphasize how these techniques could help stop devastating genetic disorders. The Oregon experiments with human embryo cells corrected disease-associated DNA variants associated with heart muscle wasting that can cause heart failure. The treated embryos were alive for only a few days and were never intended to become a human baby. They were, however, human embryos deliberately created for the research.

U.S. guidance in this area is sparse and reflects the lack of societal consensus. In 1994, when the federal government was contemplating funding for research involving human embryos, the NIH Embryo Research Panel concluded that just this kind of experiment was ethically appropriate. But within hours of that reports release, then-President Bill Clinton announced he did not agree with creating embryos in order to do research on them.

The United States currently has just two policies relevant to genomic editing of human embryos. The first blocks federal funding: On April 28, 2015, Francis Collins, director of the National Institutes of Health, stated, NIH will not fund any use of gene-editing technologies in human embryos. This is not embedded in statute or formal executive order, but members of Congress are fully aware of it and it is, in effect, a federal policy. NIH can (and does) fund genome editing of nonembryonic cells that might be used to treat cancer and for other possible therapeutic purposes, but not embryonic cells that would have their effect by creating humans with germline alterations.

Second, Congress has prohibited the Food and Drug Administration from reviewing research in which a human embryo is intentionally created or modified to include a heritable genetic modification. This language comes from a rider to FDAs annual appropriations. Yet use of human embryonic cells for treatment should be subject to FDA regulation. So this language in effect means alterations of embryonic cells cannot be done in the United States if there is any intent to treat a human being, including implantation of an altered embryo into a womans uterus. This will remain true so long as the rider is included in FDAs annual appropriations. The federal government thus has two relevant policies, both of which take federal agencies out of the action: One removes NIH funding, and the other precludes FDA oversight of genome-edited human embryos.

This leaves privately funded research that has no direct therapeutic purpose, such as with the Oregon experiments. The funding came from OHSU itself; South Korean Basic Research Funds; the municipal government of Shenzhen, China; and several private philanthropies (Chapman, Mathers, Helmsley, and Moxie). The research complies with recommendations to study the basic cellular processes of genome editing, keeping an eye on possible future clinical use but only so long as the work does not attempt to create a human pregnancy.

By coincidence, on the same day the Nature paper came out, the American Journal of Human Genetics also published a thoughtful 10-page position statement about germline genome editing from the American Society for Human Genetics endorsed by many other genetic and reproductive medicine organizations from all over the world. It reviews recommendations of the National Academies of Sciences, Engineering, and Medicine, several international and U.S.-based organizations and commissions, and makes several recommendations of its own, concluding it is inappropriate to perform germline gene editing that culminates in human pregnancy, but also there is no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications. Indeed, the statement argues for public funding. Finally, it urges research to proceed only with compelling medical rationale, strong oversight, and a transparent public process to solicit and incorporate stakeholder input.

So is there a problem here? It is truly wonderful that medical and scientific organizations have addressed genome editing. It is, however, far from sufficient. Reports and scientific consensus statements inform the policy debate but cannot resolve it. All of the reports on genome editing call for robust public debate, but the simple fact is that embryo research has proven highly divisive and resistant to consensus, and it is far from clear how to know when there is enough thoughtful deliberation to make policy choices. Its significant that none of the reports have emerged from a process that embodied such engagement. The Catholic Church, evangelical Christians, and concerned civic action groups who view embryo research as immoral are not likely to turn to the National Academies of Sciences, Engineering and Medicine, the American Society for Human Genetics, the Hinxton Group, the Nuffield Council on Bioetics, or other scientific and medical organizations for their primary counsel. They may well listen to scientists, but religious and moral doctrine will get greater weight. Yet religious groups highly critical of embryo research are part of the political systemand whether we embrace this sort of genome editing in the United States is a political question, not a purely technical one.

Reports and scientific consensus statements inform the policy debate but cannot resolveit.

Addressing the political questions will be extremely difficult. The U.S. government is poorly positioned to mediate the policy debate in a way that recognizes and addresses our complex moral pluralism. NIH and FDA are two of the most crucial agencies, but current policies remove them from line authority, and with good reason, given that engaging in this debate could actually endanger the agencies other vital missions. International consensus about genome editing of human embryos remains no more likely than about embryo research in general: Some countries ban it while others actively promote and fund it. Private foundations dont have the mandate or incentive to mediate political debate about a controversial technology that rouses the politics of abortion. What private philanthropic organization would willingly take on such a thankless and politically perilous task, and what organization would be credible to the full range of constituencies?

So who can carry out the public engagement that everyone seems to agree we need? The likely answer is no one. This problem occurs with all debate about fraught scientific and technical innovations, but its particularly acute when it touches on highly ossified abortion politics.

The debate about genomic editing of human embryos is unlikely to follow the recommendations for systematic forethought proposed by illustrious research bodies and reports. Given the reactions weve seen to human embryonic stem-cell research in the past two decades, we have ample reason for pessimism. Rather, debate is more likely to progress by reaction to events as researchers make newsoften with the same lack of information we lived with for the last week of July, based on incomplete media accounts and quotes from disparate experts who lacked access to the details. Most of the debate will be quote-to-quote combat in the public media, leavened by news and analysis in scientific and medical journals, but surrounded by controversy in religious and political media. It is not what anyone designing a system would want. But the recommendations for robust public engagement and debate feel a bit vacuous and vague, aspirations untethered to a concrete framework.

Our divisive political system seems fated to make decisions about genomic editing of human embryos mainly amidst conflict, with experts dueling in the public media rather than through a thoughtful and well-informed debate conducted in a credible framework. As the furor over the Oregon experiments begins to dissipate, we await the event that will cause the next flare-up. And so it will continue, skipping from news cycle to news cycle.

History shows that sometimes technical advances settle the issues, at least for most people and in defined contexts. Furor about in vitro fertilization after Louise Brown, the first test tube baby, was born in 1978 gave way to acceptance as grateful parents gave birth to more and more healthy babies and welcomed them into their families. Initial revulsion at heart transplants gave way in the face of success. Anger about prospects for human embryonic stem-cell research might similarly attenuate if practical applications emerge.

Such historical examples show precisely why reflective deliberation remains essential, despite its unlikely success. Momentum tends to carry the research forward. Yet at times we should stop, learn more, and decide actively rather than passively whether to proceed, when, how, and with what outcomes in mind. In the case of genome editing of human embryos, however, it seems likely that technology will make the next move.

This article is part of Future Tense, a collaboration among Arizona State University, New America, and Slate. Future Tense explores the ways emerging technologies affect society, policy, and culture. To read more, follow us on Twitter and sign up for our weekly newsletter.

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The Public Needs to Weigh In on the Ethics of Genetically Engineering Humans - Slate Magazine

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Altering human heredity? Researchers repair disease-causing gene – TRT World

Posted: August 17, 2017 at 3:45 am

In this microscope photo provided by Oregon Health & Science University, human embryos grow in a laboratory for a few days after researchers used gene editing technology to successfully repair a heart disease-causing genetic mutation.

In a first, researchers safely repaired a disease-causing gene in human embryos, targeting a heart defect best known for killing young athletes a big step toward one day preventing a list of inherited diseases.

In a surprising discovery, a research team led by Oregon Health and & Science University reported on Wednesday that embryos can help fix themselves if scientists jump-start the process early enough. It's laboratory research only, nowhere near ready to be tried in a pregnancy.

The work, a scientific first led by researchers at Oregon Health & Science University, marks a step toward one day preventing babies from inheriting diseases that run in the family.

But it suggests that scientists might alter DNA in a way that protects not just one baby from a disease that runs in the family, but his or her offspring as well.

And that raises ethical questions.

"I for one believe, and this paper supports the view, that ultimately gene editing of human embryos can be made safe. Then the question truly becomes, if we can do it, should we do it?" said Dr George Daley, a stem cell scientist and dean of Harvard Medical School. He wasn't involved in the new research and praised it as "quite remarkable."

"This is definitely a leap forward," agreed developmental geneticist Robin Lovell-Badge of Britain's Francis Crick Institute.

A way to repair embryos?

Today, couples seeking to avoid passing on a bad gene sometimes have embryos created in fertility clinics so they can discard those that inherit the disease and attempt pregnancy only with healthy ones, if there are any.

Gene editing, in theory, could rescue diseased embryos. But so-called "germline" changes altering sperm, eggs or embryos are controversial because they would be permanent, passed down to future generations.

Critics worry about attempts at "designer babies" instead of just preventing disease, and a few previous attempts at learning to edit embryos, in China, didn't work well and, more importantly, raised safety concerns.

In a series of laboratory experiments reported in the journal Nature, the Oregon researchers tried a different approach.

They targeted a gene mutation that causes a heart-weakening disease hypertrophic cardiomyopathy that affects about 1 in 500 people. Inheriting just one copy of the bad gene can cause it.

Snipping away mutation

The team programmed a gene-editing tool named CRISPR-Cas9 that acts like a pair of molecular scissors to find that mutation a missing piece of genetic material.

Then came the test. Researchers injected sperm from a patient with the heart condition along with those molecular scissors into healthy donated eggs at the same time.

The scissors cut the defective DNA in the sperm.

Normally cells will repair a CRISPR-induced cut in DNA by essentially gluing the ends back together. Or scientists can try delivering the missing DNA in a repair package, like a computer's cut-and-paste program.

An intelligent repair mechanism

Instead, the newly-forming embryos made their own perfect fix without that outside help, reported Oregon Health & Science University senior researcher Shoukhrat Mitalipov.

We all inherit two copies of each gene, one from dad and one from mom and those embryos just copied the healthy one from the donated egg.

"The embryos are really looking for the blueprint," Mitalipov, who directs OHSU's Center for Embryonic Cell and Gene Therapy, said in an interview. "We're finding embryos will repair themselves if you have another healthy copy."

It worked 72 percent of the time, in 42 out of 58 embryos. Normally a sick parent has a 50-50 chance of passing on the mutation.

Previous embryo-editing attempts in China found not every cell was repaired, a safety concern called mosaicism.

Beginning the process before fertilization avoided that problem: until now, "everybody was injecting too late," Mitalipov said.

Nor did intense testing uncover any "off-target" errors, cuts to DNA in the wrong places, reported the team, which also included researchers from the Salk Institute for Biological Studies in California and South Korea's Institute for Basic Science.

The embryos weren't allowed to develop beyond eight cells, a standard for laboratory research.

The experiments were privately funded; US tax dollars aren't allowed for embryo research.

Genetics and ethics experts not involved in the work say it's a critical first step but just one step toward eventually testing the process in pregnancy, something currently prohibited by US policy.

The ethics behind editing embryos back to health

"This is very elegant lab work," but it's moving so fast that society needs to catch up and debate how far it should go, said Johns Hopkins University bioethicist Jeffrey Kahn.

And lots more research is needed to tell if it's really safe, added Britain's Lovell-Badge.

He and Kahn were part of a National Academy of Sciences report earlier this year that said if germline editing ever were allowed, it should be only for serious diseases with no good alternatives and done with strict oversight.

"What we do not want is for rogue clinicians to start offering treatments" that are unproven, as has happened with some other experimental technologies, he stressed.

Among key questions: Would the technique work if mom, not dad, harboured the mutation? Is repair even possible if both parents pass on a bad gene?

Mitalipov is "pushing a frontier," but it's responsible basic research that's critical for understanding embryos and disease inheritance, noted University of Pittsburgh professor Kyle Orwig.

In fact, Mitalipov said the research should offer critics some reassurance: If embryos prefer self-repair, it would be extremely hard to add traits for "designer babies" rather than just eliminate disease.

"All we did is un-modify the already mutated gene."

Source: AP

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Altering human heredity? Researchers repair disease-causing gene - TRT World

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First embryo gene-repair holds promise for inherited disease – HollandSentinel.com

Posted: August 14, 2017 at 2:46 am

Altering human heredity? In a first, researchers safely repaired a disease-causing gene in human embryos, targeting a heart defect best known for killing young athletes a big step toward one day preventing a list of inherited diseases.

WASHINGTON Altering human heredity? In a first, researchers safely repaired a disease-causing gene in human embryos, targeting a heart defect best known for killing young athletes a big step toward one day preventing a list of inherited diseases.

In a surprising discovery, a research team led by Oregon Health and & Science University reported Wednesday that embryos can help fix themselves if scientists jump-start the process early enough.

It's laboratory research only, nowhere near ready to be tried in a pregnancy. But it suggests that scientists might alter DNA in a way that protects not just one baby from a disease that runs in the family, but his or her offspring as well. And that raises ethical questions.

"I for one believe, and this paper supports the view, that ultimately gene editing of human embryos can be made safe. Then the question truly becomes, if we can do it, should we do it?" said Dr. George Daley, a stem cell scientist and dean of Harvard Medical School. He wasn't involved in the new research and praised it as "quite remarkable."

"This is definitely a leap forward," agreed developmental geneticist Robin Lovell-Badge of Britain's Francis Crick Institute.

Today, couples seeking to avoid passing on a bad gene sometimes have embryos created in fertility clinics so they can discard those that inherit the disease and attempt pregnancy only with healthy ones, if there are any.

Gene editing in theory could rescue diseased embryos. But so-called "germline" changes altering sperm, eggs or embryos are controversial because they would be permanent, passed down to future generations. Critics worry about attempts at "designer babies" instead of just preventing disease, and a few previous attempts at learning to edit embryos, in China, didn't work well and, more importantly, raised safety concerns.

In a series of laboratory experiments reported in the journal Nature, the Oregon researchers tried a different approach.

They targeted a gene mutation that causes a heart-weakening disease, hypertrophic cardiomyopathy, that affects about 1 in 500 people. Inheriting just one copy of the bad gene can cause it.

The team programmed a gene-editing tool, named CRISPR-Cas9, that acts like a pair of molecular scissors to find that mutation a missing piece of genetic material.

Then came the test. Researchers injected sperm from a patient with the heart condition along with those molecular scissors into healthy donated eggs at the same time. The scissors cut the defective DNA in the sperm.

Normally cells will repair a CRISPR-induced cut in DNA by essentially gluing the ends back together. Or scientists can try delivering the missing DNA in a repair package, like a computer's cut-and-paste program.

Instead, the newly forming embryos made their own perfect fix without that outside help, reported Oregon Health & Science University senior researcher Shoukhrat Mitalipov.

We all inherit two copies of each gene, one from dad and one from mom and those embryos just copied the healthy one from the donated egg.

"The embryos are really looking for the blueprint," Mitalipov, who directs OHSU's Center for Embryonic Cell and Gene Therapy, said in an interview. "We're finding embryos will repair themselves if you have another healthy copy."

It worked 72 percent of the time, in 42 out of 58 embryos. Normally a sick parent has a 50-50 chance of passing on the mutation.

Previous embryo-editing attempts in China found not every cell was repaired, a safety concern called mosaicism. Beginning the process before fertilization avoided that problem: Until now, "everybody was injecting too late," Mitalipov said.

Nor did intense testing uncover any "off-target" errors, cuts to DNA in the wrong places, reported the team, which also included researchers from the Salk Institute for Biological Studies in California and South Korea's Institute for Basic Science. The embryos weren't allowed to develop beyond eight cells, a standard for laboratory research. The experiments were privately funded; U.S. tax dollars aren't allowed for embryo research.

Genetics and ethics experts not involved in the work say it's a critical first step but just one step toward eventually testing the process in pregnancy, something currently prohibited by U.S. policy.

"This is very elegant lab work," but it's moving so fast that society needs to catch up and debate how far it should go, said Johns Hopkins University bioethicist Jeffrey Kahn.

And lots more research is needed to tell if it's really safe, added Britain's Lovell-Badge. He and Kahn were part of a National Academy of Sciences report earlier this year that said if germline editing ever were allowed, it should be only for serious diseases with no good alternatives and done with strict oversight.

"What we do not want is for rogue clinicians to start offering treatments" that are unproven, as has happened with some other experimental technologies, he stressed.

Among key questions: Would the technique work if mom, not dad, harbored the mutation? Is repair even possible if both parents pass on a bad gene?

Mitalipov is "pushing a frontier," but it's responsible basic research that's critical for understanding embryos and disease inheritance, noted University of Pittsburgh professor Kyle Orwig.

In fact, Mitalipov said the research should offer critics some reassurance: If embryos prefer self-repair, it would be extremely hard to add traits for "designer babies" rather than just eliminate disease.

"All we did is un-modify the already mutated gene."

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First embryo gene-repair holds promise for inherited disease - HollandSentinel.com

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

Posted: August 6, 2017 at 5:45 am

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

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