Stem Cell Clinic

SpaceX to launch heart, bone health experiments to space station – CU Boulder Today

By Stem Cell Clinic

Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch has been rescheduled for Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

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SpaceX to launch heart, bone health experiments to space station - CU Boulder Today

Trials of Embryonic Stem Cells to Launch in China

By Embryonic Stem Cells

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In the next few months, surgeons in the Chinese city of Zhengzhou will carefully drill through the skulls of people with Parkinsons disease and inject 4 million immature neurons derived from human embryonic stem cells into their brains. Then they will patch the patients up, send them home and wait.

This will mark the start of the first clinical trial in China using human embryonic stem (ES) cells, and the first one worldwide aimed at treating Parkinsons disease using ES cells from fertilized embryos. In a second trial starting around the same time, a different team in Zhengzhou will use ES cells to target vision loss caused by age-related macular degeneration.

The experiments will also represent the first clinical trials of ES cells under regulations that China adopted in 2015, in an attempt to ensure the ethical and safe use of stem cells in the clinic. China previously had no clear regulatory framework, and many companies had used that gap as an excuse to market unproven stem-cell treatments.

It will be a major new direction for China, says Pei Xuetao, a stem-cell scientist at the Beijing Institute of Transfusion Medicine who is on the central-government committee that approved the trials. Other researchers who work on Parkinsons disease, however, worry that the trials might be misguided.

Both studies will take place at the First Affiliated Hospital ofZhengzhouUniversity in Henan province. In the first, surgeons will inject ES-cell-derived neuronal-precursor cells into the brains of individuals with Parkinsons disease. The only previous trial using ES cells to treat Parkinsons began last year in Australia; participants there received stem cells from parthenogenetic embryosunfertilized eggs that are triggered in the lab to start embryonic development.

In the other Zhengzhou trial, surgeons will take retinal cells derived from ES cells and transplant them into the eyes of people with age-related macular degeneration. The team will follow a similar procedure to that of previous ES-cell trials carried out by researchers in the United States and South Korea.

Qi Zhou, a stem-cell specialist at the Chinese Academy of Sciences Institute of Zoology in Beijing, is leading both efforts. For the Parkinsons trial, his team assessed hundreds of candidates and have so far have picked ten who best match the ES cells in the cell bank, to reduce the risk of the patients bodies rejecting the cells.

The 2015 regulations state that hospitals planning to carry out stem-cell clinical work must use government-certified ES-cell lines and pass hospital-review procedures. Zhous team completed four years of work with a monkey model of Parkinsons, and has met the government requirements, he says.

Parkinsons disease is caused by a deficit in dopamine produced by brain cells. Zhous team will coax ES cells to develop into precursors to neurons, and will then inject them into the striatum, a central region of the brain implicated in the disease.

In their unpublished study of 15 monkeys, the researchers did not observe any improvements in movement at first, says Zhou. But at the end of the first year, the team examined the brains of half the monkeys and found that the stem cells had turned into dopamine-releasing cells. He says that they saw 50% improvement in the remaining monkeys over the next several years. We have all the imaging data, behavioural data and molecular data to support efficacy, he says. They are preparing a publication, but Zhou says that they wanted to collect a full five years worth of animal data.

Jeanne Loring, a stem-cell biologist at the Scripps Research Institute in La Jolla, California, who is also planning stem-cell trials for Parkinsons, is concerned that the Australian and Chinese trials use neural precursors and not ES-cell-derived cells that have fully committed to becoming dopamine-producing cells. Precursor cells can turn into other kinds of neurons, and could accumulate dangerous mutations during their many divisions, says Loring. Not knowing what the cells will become is troubling.

But Zhou and the Australian team defend their choices. Russell Kern, chief scientific officer of the International Stem Cell Corporation in Carlsbad, California, which is providing the cells for and managing the Australian trial, says that in preclinical work, 97% of them became dopamine-releasing cells.

Lorenz Studer, a stem-cell biologist at the Memorial Sloan Kettering Cancer Center in New York City who has spent years characterizing such neurons ahead of his own planned clinical trials, says that support is not very strong for the use of precursor cells. I am somewhat surprised and concerned, as I have not seen any peer-reviewed preclinical data on this approach, he says.

Studers and Lorings teams are part of an international consortium that coordinates stem-cell treatments for Parkinsons. In the next two years, five groups in the consortium plan to run trials using cells fully committed to becoming dopamine-producing cells.

Regenerative neurobiologist Malin Parmar, who heads one of the teams at Lund University in Sweden, says that the groups are all rapidly moving towards clinical trials, and this field will be very exciting in the coming years.

Source & Credits: ScientificAmerican

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Trials of Embryonic Stem Cells to Launch in China

First adult cured of sickle cell at a Kansas hospital – Idaho Statesman

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First adult cured of sickle cell at a Kansas hospital
Idaho Statesman
More public education about the cure and better recruitment of bone marrow donors could help more high-risk patients shed the disease, said Joseph McGuirk, medical director for blood and marrow transplant for the University of Kansas Health System.

and more »

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First adult cured of sickle cell at a Kansas hospital - Idaho Statesman

Stem Cell Therapy – Checkbiotech.org (press release)

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Stem Cell Therapy is poised to change the face of medicine.

Thousands of published studies and or testimonialscan be wrong! Regeneration or Regenerative Medicine has the ability to change almost all facets of medicine.

Doctors are using them on themselves to help with problems and or provide with a better quality of life, in-fact one doctor sais in a recent stem cell seminar that he would be doing them every year just for preventative maintenance.

Stem Cells have been studied for decades however in the past few years a real breakthrough in using Human UmbilicalCell Tissue (HUCT) being harvested from healthy mommy / healthy baby umbilical cords.

Studies have proved that the older you get the fewer stem cells in the body, ruling out the effectiveness of stem cells extracted from your aging body or your fat.

Statin drugs have been proven to diminishstem cells

Using Concentrated Umbilical-Cord Potentcy Stem Cells (CUP STEM CELLS) give you cell counts in the millions ffrom a newborn tht may allow stem cells to duplicate every 28 hours, over 65 or so cucles making CUP STEM CELLS very favorable to all the other options available in the past.

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Stem Cell Therapy - Checkbiotech.org (press release)

Trials of embryonic stem cells to launch in China : Nature …

By Embryonic Stem Cells

Jason Lee/Reuters

Former Chinese leader Deng Xiaoping had Parkinsons disease, one of the first targets of embryonic-stem-cell therapies being tested in China.

In the next few months, surgeons in the Chinese city of Zhengzhou will carefully drill through the skulls of people with Parkinsons disease and inject 4 million immature neurons derived from human embryonic stem cells into their brains. Then they will patch the patients up, send them home and wait.

This will mark the start of the first clinical trial in China using human embryonic stem (ES) cells, and the first one worldwide aimed at treating Parkinsons disease using ES cells from fertilized embryos. In a second trial starting around the same time, a different team in Zhengzhou will use ES cells to target vision loss caused by age-related macular degeneration.

The experiments will also represent the first clinical trials of ES cells under regulations that China adopted in 2015, in an attempt to ensure the ethical and safe use of stem cells in the clinic. China previously had no clear regulatory framework, and many companies had used that gap as an excuse to market unproven stem-cell treatments.

It will be a major new direction for China, says Pei Xuetao, a stem-cell scientist at the Beijing Institute of Transfusion Medicine who is on the central-government committee that approved the trials. Other researchers who work on Parkinsons disease, however, worry that the trials might be misguided.

Both studies will take place at the First Affiliated Hospital ofZhengzhouUniversity in Henan province. In the first, surgeons will inject ES-cell-derived neuronal-precursor cells into the brains of individuals with Parkinsons disease. The only previous trial using ES cells to treat Parkinsons began last year in Australia; participants there received stem cells from parthenogenetic embryosunfertilized eggs that are triggered in the lab to start embryonic development.

In the other Zhengzhou trial, surgeons will take retinal cells derived from ES cells and transplant them into the eyes of people with age-related macular degeneration. The team will follow a similar procedure to that of previous ES-cell trials carried out by researchers in the United States and South Korea.

Qi Zhou, a stem-cell specialist at the Chinese Academy of Sciences Institute of Zoology in Beijing, is leading both efforts. For the Parkinsons trial, his team assessed hundreds of candidates and have so far have picked ten who best match the ES cells in the cell bank, to reduce the risk of the patients bodies rejecting the cells.

The 2015 regulations state that hospitals planning to carry out stem-cell clinical work must use government-certified ES-cell lines and pass hospital-review procedures. Zhous team completed four years of work with a monkey model of Parkinsons, and has met the government requirements, he says.

Parkinsons disease is caused by a deficit in dopamine produced by brain cells. Zhous team will coax ES cells to develop into precursors to neurons, and will then inject them into the striatum, a central region of the brain implicated in the disease.

In their unpublished study of 15 monkeys, the researchers did not observe any improvements in movement at first, says Zhou. But at the end of the first year, the team examined the brains of half the monkeys and found that the stem cells had turned into dopamine-releasing cells. He says that they saw 50% improvement in the remaining monkeys over the next several years. We have all the imaging data, behavioural data and molecular data to support efficacy, he says. They are preparing a publication, but Zhou says that they wanted to collect a full five years worth of animal data.

Jeanne Loring, a stem-cell biologist at the Scripps Research Institute in La Jolla, California, who is also planning stem-cell trials for Parkinsons, is concerned that the Australian and Chinese trials use neural precursors and not ES-cell-derived cells that have fully committed to becoming dopamine-producing cells. Precursor cells can turn into other kinds of neurons, and could accumulate dangerous mutations during their many divisions, says Loring. Not knowing what the cells will become is troubling.

But Zhou and the Australian team defend their choices. Russell Kern, chief scientific officer of the International Stem Cell Corporation in Carlsbad, California, which is providing the cells for and managing the Australian trial, says that in preclinical work, 97% of them became dopamine-releasing cells.

Lorenz Studer, a stem-cell biologist at the Memorial Sloan Kettering Cancer Center in New York City who has spent years characterizing such neurons ahead of his own planned clinical trials, says that support is not very strong for the use of precursor cells. I am somewhat surprised and concerned, as I have not seen any peer-reviewed preclinical data on this approach, he says.

Studers and Lorings teams are part of an international consortium that coordinates stem-cell treatments for Parkinsons. In the next two years, five groups in the consortium plan to run trials using cells fully committed to becoming dopamine-producing cells.

Regenerative neurobiologist Malin Parmar, who heads one of the teams at Lund University in Sweden, says that the groups are all rapidly moving towards clinical trials, and this field will be very exciting in the coming years.

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Trials of embryonic stem cells to launch in China : Nature ...

World-first trials have been launched to treat Parkinson’s and blindness with embryonic stem cells – ScienceAlert

By Embryonic Stem Cells

In a world first, surgeons in the Chinese city of Zhengzhou are planning to inject stem cells derived from human embryos into the brains of patients with Parkinson's disease with the aim of treating their debilitating symptoms.

Meanwhile, another medical team in the same city is aiming to target vision loss using embryonic stem cells (ESC) to replace lost cells in the retina, marking a new direction in China in the wake of major changes in how the country regulates stem cell treatments.

While similar treatments on Parkinson's patients have already been tested in Australia, those trials relied on cells taken from eggs that were forced to divide without first being fertilised in an effort to circumvent any ethical concerns.

Stem cells are a little like blank slates that are yet to take on a specific task. If you rewind the clock on any of your body's tissues, its cells will become less specialised, until you're left with a cell with a lot of potential to become nearly anything.

In the case of both kinds of embryonic stem cells, divided egg cells are subjected to various treatments to encourage them to develop into replacement cells that could treat a condition in a recipient.

The symptoms of Parkinson's disease are largely caused by a loss of nervous tissue deep inside the brain in an area called the basal ganglia.

Losing those cells means a loss of a neurotransmitter called dopamine, and with it a lower ability to control nervous impulses that would prevent muscles in the extremities from activating.

In the case of a condition called macular degeneration, damage to a layer of tissue called the retinal pigment epithelium at the back of the eye causes the light-catching cells above it to die.

By turning ESC into cells that can naturally develop into the tissues that have deteriorated such as the precursors to neurons that can produce dopamine, or into retinal tissue and then injecting it into the target site, the researchers hope to improve the lost functions.

Not everybody is convinced of the success of trials such as those being done in China and last year in Australia.

A stem cell biologist from the Scripps Research Institute in California, Jeanne Loring, believes the choice of cell used in both Parkinson's disease trials won't be specialised enough to match expected results.

"Not knowing what the cells will become is troubling," Loring told David Cyranoski at Nature.

But the research team in China remains confident in its decision.

Qi Zhou from the Chinese Academy of Sciences Institute of Zoology in Beijing is the stem cell specialist leading both sets of ESC trials, and says four years of animal trials conducted on monkeys have so far showed promising results.

"We have all the imaging data, behavioural data, and molecular data to support efficacy," Zhou told Nature.

He also claims the team conducting the Parkinson's trial have been selective with their potential candidates, choosing patients who will have the least chance of rejecting the ESCs from the cell bank.

In 2015, China introduced tough new regulations to deal with the growing problem of 'rogue clinics' offering stem cell treatments without due record keeping or process, making it hard to evaluate safety, or even the types of cells used in the treatments.

The changes are set to improve the ethics and safety of stem cell treatments by enforcing the use of cells through a regulatory body, ensuring informed patient consent, and permitting treatments only through authorised hospitals.

Time will tell if the regulations can be enforced, but for stem cell researchers, the changes are positive.

"It will be a major new direction for China," stem cell scientist Pei Xuetaotold Nature.

If the results are as good as the teams in Australia and China predict, it could also set new standards for the world.

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World-first trials have been launched to treat Parkinson's and blindness with embryonic stem cells - ScienceAlert

From baby mice to designer humans? – Brnow

By Embryonic Stem Cells

Scientists in Japan are growing mice from skin cells. A team of researchers at Kyushu University last year converted tail cells from adult female mice into viable eggs, and then inseminated those eggs to produce embryos. They implanted the embryos in female mice who gave birth to healthy baby mice. The process, called in vitro gametogenesis (IVG), is a big leap from todays in vitro fertilization (IVF). With IVG, doctors can artificially create eggs and sperm by coaxing cells from other parts of the body into stem cells, and then into eggs and sperm. Researchers say it is only a matter of time before they can use the process for human reproduction. But experts warn of serious ethical, medical and legal consequences for using this new technology on humans. In a cautionary article published earlier this year in the journal Science Translational Medicine, a group of academics from Harvard and Brown universities noted the technology promises to transform the fields of reproductive and regenerative medicine, but also creates vast ethical and social policy challenges that must be addressed. With IVG, creating life no longer would require a man and a woman: Two men could make a baby biologically related to them both using the skin cells of one to make an egg, and the sperm of the other. A woman could make a baby by herself using her cell-turned-sperm and her egg, almost like cloning. A group of three or four people could create a baby by creating two embryos, and then taking an egg from one and a sperm from the other, creating another embryo with multiple parents. Such scenarios inevitably would affect the traditional understanding of parenting. The article also addressed the potential for unauthorized use of biomaterials: Someone retrieves a skin cell from a hotel room bed or bathroom, creating a baby biologically related to someone without their knowledge. Should the law criminalize such an action? If it takes place, should the law consider the source of the skin cells to be a legal parent to the child, or should it distinguish an individuals genetic and legal parentage? the authors wrote. They also raised the potential for bioethical issues on a massive scale. IVG may raise the specter of embryo farming on a scale currently unimagined, which might exacerbate concerns about the devaluation of human life, wrote the authors, pointing to the inevitable destruction of large numbers of embryos, the commercialization of egg production, the creation of an all but inexhaustible supply of embryonic stem cells for research and the open invitation for a couple to create designer babies due to limitless eggs. But significant scientific hurdles remain. People are a lot more complicated than mice, Susan Solomon, chief executive of the New York Stem Cell Foundation, told The New York Times. And weve often seen that the closer you get to something, the more obstacles you discover. Despite those hurdles, the articles authors warn IVG technology is moving faster than our conversations about the ethical questions it raises. We have come to realize that scientific developments are outpacing our ability to think through them, Eli Y. Adashi, a medical science professor at Brown, told The New York Times. Its a challenge for which we are not fully prepared. (EDITORS NOTE Kiley Crossland writes for WORLD News Service, a division of WORLD Magazine, worldmag.com, based in Asheville, N.C. Used by permission.)

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From baby mice to designer humans? - Brnow

How does the body process pain? Study sheds new light – Medical News Today

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Currently available pain medications have limited efficacy and numerous side effects. New research, however, provides deeper insights into how our bodies process pain, paving the way for an innovative, more effective way of targeting chronic pain.

According to recent estimates from the National Institutes of Health (NIH), as many as 25 million people in the United States live with daily pain, and 23 million of the country's adults have more severe pain.

Chronic pain is defined as any pain that lasts for longer than 12 weeks, although in most cases of chronic pain, the discomfort lasts for months on end.

The currently available treatment options for chronic pain aim to help the patients better manage the pain rather than "cure" it. To this end, medical professionals prescribe medication, electrical brain stimulation, or surgery.

Commonly prescribed medications for pain management include two categories of drugs: nonsteroidal anti-inflammatory drugs (NSAIDs), such as aspirin or ibuprofen, or opioids, such as codeine or morphine.

Both of these classes of medication can cause serious side effects, besides not always working. Opioids can cause addiction, especially if used over a longer period of time, and NSAIDs can lead to stomach ulcers and damage to the kidneys. For these reasons, people often turn to complementary or alternative medicine to ease their pain.

But now, researchers from Columbia University Medical Center in New York - led by Dr. Nigel Bunnett, Ph.D., a professor of surgery and pharmacology - have unveiled the mechanism behind chronic pain, in a study that could prove to be a game-changer for pain medication.

"Previous efforts to develop more effective analgesics have been stalled by our limited understanding of the mechanisms that allow nerves to sense and transmit pain signals," Dr. Bunnett says.

The new research - published in the journal Science Translational Medicine - reveals how pain occurs in a location that may be "hidden" away from the reach of widely available pain medication.

Some of the analgesics available target so-called G protein-coupled receptors (GPCRs) on the surface of the cell.

GPCRs are a group of membrane receptors shared by humans, animals, plants, and even fungi. Their role is to receive information from the environment and pass it on to the cells.

In the human body, GPCRs fulfill a wide range of functions - in fact, humans have almost 1,000 different GPCRs, attuned to very specific signals from the environment. So, the receptors are involved in almost all of the body's biological processes, including its response to pain.

As a result, between one third and 50 percent of all of the drugs available on the market work by targeting GPCRs. For instance, some pain medication works by activating opioid receptors, which are a kind of GPCR.

There is an additional type of GPCR called the neurokinin 1 receptor (NK1R). When activated, this receptor causes pain and inflammation.

New drugs that have attempted to target this pain receptor have been unsuccessful, however.

However, the new experiments carried out by Dr. Bunnett and his colleagues revealed that when activated by pain, NK1R tends to quickly shift away from the cell surface and into the endosomes of the nerve cells.

Endosomes are small compartments inside a cell. When NK1R is inside an endosome, it can continue to cause pain and inflammation.

Further experiments in rodents showed that attaching a fat molecule, or a lipid, to NK1R-blocking chemical compounds helps them to break through the membrane and into the endosomes, thus providing pain relief.

"From these experiments, we have demonstrated that designing NK1R inhibitors that are capable of reaching the endosomal network within nerve cells may provide much longer-lasting pain relief than currently available analgesics. More than a third of all currently available drugs act on GPCRs in some way. We think that modification of many existing compounds, as we did with NK1R inhibitors, may have the potential to enhance the effectiveness of many different classes of medications."

Dr. Nigel Bunnett

Given the limitations of NSAIDs and opioids, the research done by Dr. Bunnett and colleagues could bring some much-needed innovation to existing pain-targeting medication.

Learn how yoga and acupuncture may be effective for chronic pain management.

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How does the body process pain? Study sheds new light - Medical News Today

Philips, MSK Partner on Genome Analytics for Prostate Cancer Precision Medicine – Genetic Engineering & Biotechnology News

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The Memorial Sloan Kettering Cancer Center (MSK) and Philips will exploit the latters IntelliSpace Genomics platform through their research collaboration to develop new genome analysis methods and informatics approaches for diagnosing pancreatic cancer and aiding personalized therapeutics. The collaboration will employ large-scale next-generation sequencing to generate new insights into the drivers of pancreatic cancer at the single-cell level, with the goal of enabling more precise diagnosis so that patients can be prescribed optimum treatments that target the cause of their disease.

Philips' IntelliSpace Genomics platform has been developed to support the implementation and scaling of informatics-heavy precision medicine research. "Collaborating with MSK and its experts will allow us to take a unique approach to diagnosing and treating this devastating disease, commented Henk van Houten, Ph.D., CTO at Philips. Leveraging the advanced capabilities of the Philips IntelliSpace Genomics solution we can gain new insights into the origin, development, and optimal treatment of pancreatic cancer and share these insights broadly with care providers to help improve outcomes for patients. Our ultimate goal is to translate these findings into more precise diagnostics and therapeutics to battle this devastating disease."

Philips and MSK have previously established a research collaboration in the field of radiation oncology. Just yesterday, MSK and Mabvax Therapeutics signed a research agreement to develop chimeric antigen receptor (CAR) T-cellimmunotherapies for pancreatic and small-cell lung cancer.

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Philips, MSK Partner on Genome Analytics for Prostate Cancer Precision Medicine - Genetic Engineering & Biotechnology News

One gene closer to regenerative therapy for muscular disorders – Medical Xpress

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June 1, 2017 This microscopic image of fibroblast cells shows the induction of cell fusion by a newly described gene and its protein, called myomerger. Multi-nucleus cells expressing genes needed to form skeletal muscle can be seen in flower-like clumps forming as cells fuse together. Reporting results in Nature Communications, the researchers seek ways to develop regenerative therapies for muscle disorders by getting stem cells to fuse and form functioning skeletal muscle tissues. Credit: Cincinnati Children's

A detour on the road to regenerative medicine for people with muscular disorders is figuring out how to coax muscle stem cells to fuse together and form functioning skeletal muscle tissues. A study published June 1 by Nature Communications reports scientists identify a new gene essential to this process, shedding new light on possible new therapeutic strategies.

Led by researchers at the Cincinnati Children's Hospital Medical Center Heart Institute, the study demonstrates the gene Gm7325 and its protein - which the scientists named "myomerger" - prompt muscle stem cells to fuse and develop skeletal muscles the body needs to move and survive. They also show that myomerger works with another gene, Tmem8c, and its associated protein "myomaker" to fuse cells that normally would not.

In laboratory tests on embryonic mice engineered to not express myomerger in skeletal muscle, the animals did not develop enough muscle fiber to live.

"These findings stimulate new avenues for cell therapy approaches for regenerative medicine," said Douglas Millay, PhD, study senior investigator and a scientist in the Division of Molecular Cardiovascular Biology at Cincinnati Children's. "This includes the potential for cells expressing myomaker and myomerger to be loaded with therapeutic material and then fused to diseased tissue. An example would be muscular dystrophy, which is a devastating genetic muscle disease. The fusion technology possibly could be harnessed to provide muscle cells with a normal copy of the missing gene."

Bio-Pioneering in Reverse

One of the molecular mysteries hindering development of regenerative therapy for muscles is uncovering the precise genetic and molecular processes that cause skeletal muscle stem cells (called myoblasts) to fuse and form the striated muscle fibers that allow movement. Millay and his colleagues are identifying, deconstructing and analyzing these processes to search for new therapeutic clues.

Genetic degenerative disorders of the muscle number in the dozens, but are rare in the overall population, according to the National Institutes of Health. The major categories of these devastating wasting diseases include: muscular dystrophy, congenital myopathy and metabolic myopathy. Muscular dystrophies are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. The most common form is Duchenne MD.

Molecular Sleuthing

A previous study authored by Millay in 2014 identified myomaker and its gene through bioinformatic analysis. Myomaker is also required for myoblast stem cells to fuse. However, it was clear from that work that myomaker did not work alone and needed a partner to drive the fusion process. The current study indicates that myomerger is the missing link for fusion, and that both genes are absolutely required for fusion to occur, according to the researchers.

To find additional genes that regulate fusion, Millay's team screened for those activated by expression of a protein called MyoD, which is the primary initiator of the all the genes that make muscle. The team focused on the top 100 genes induced by MyoD (including GM7325/myomerger) and designed a screen to test the factors that could function within and across cell membranes. They also looked for genes not previously studied for having a role in fusing muscle stem cells. These analyses eventually pointed to a previously uncharacterized gene listed in the database - Gm7325.

Researchers then tested cell cultures and mouse models by using a gene editing process called CRISPR-Cas9 to demonstrate how the presence or absence of myomaker and myomerger - both individually and in unison - affect cell fusion and muscle formation. These tests indicate that myomerger-deficient muscle cells called myocytes differentiate and form the contractile unit of muscle (sarcomeres), but they do not join together to form fully functioning muscle tissue.

Looking Ahead

The researchers are building on their current findings, which they say establishes a system for reconstituting cell fusion in mammalian cells, a feat not yet achieved by biomedical science.

For example, beyond the cell fusion effects of myomaker and myomerger, it isn't known how myomaker or myomerger induce cell membrane fusion. Knowing these details would be crucial to developing potential therapeutic strategies in the future, according to Millay. This study identifies myomerger as a fundmentally required protein for muscle development using cell culture and laboratory mouse models.

The authors emphasize that extensive additional research will be required to determine if these results can be translated to a clinical setting.

Explore further: Researchers turn stem cells into somites, precursors to skeletal muscle, cartilage and bone

More information: Nature Communications (2017). DOI: 10.1038/NCOMMS15665

Adding just the right mixture of signaling moleculesproteins involved in developmentto human stem cells can coax them to resemble somites, which are groups of cells that give rise to skeletal muscles, bones, and cartilage ...

A team led by Jean-Franois Ct, researcher at the IRCM, identified a ''conductor'' in the development of muscle tissue. The discovery, published online yesterday by the scientific journal Proceedings of the National ...

Athletes, the elderly and those with degenerative muscle disease would all benefit from accelerated muscle repair. When skeletal muscles, those connected to the bone, are injured, muscle stem cells wake up from a dormant ...

Johns Hopkins researchers report they have inadvertently found a way to make human muscle cells bearing genetic mutations from people with Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy is a chronic disease causing severe muscle degeneration that is ultimately fatal. As the disease progresses, muscle precursor cells lose the ability to create new musclar tissue, leading to faster ...

Researchers at Sanford Burnham Prebys Medical Research Institute (SBP) have conclusively identified the protein complex that controls the genes needed to repair skeletal muscle. The discovery clears up deep-rooted conflicting ...

The human body runs according to a roughly 24-hour cycle, controlled by a "master" clock in the brain and peripheral clocks in other parts of the body that are synchronized according to external cues, including light. Now, ...

Cholesterol, a naturally occurring compound at the lung surface, has been shown to have a clear effect on the properties of this nanoscale film that covers the inside of our lungs. Cholesterol levels in this system may affect ...

A detour on the road to regenerative medicine for people with muscular disorders is figuring out how to coax muscle stem cells to fuse together and form functioning skeletal muscle tissues. A study published June 1 by Nature ...

Researchers from Monash University have developed a new drug delivery strategy able to block pain within the nerve cells, in what could be a major development of an immediate and long lasting treatment for pain.

Can a fish with a malformed jaw tell us something about hearing loss in mice and humans? The answer is yes, according to a new publication in Scientific Reports.

A newly developed tissue scanner allows looking under the skin of psoriasis patients. This provides clinically relevant information, such as the structure of skin layers and blood vessels, without the need for contrast agents ...

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One gene closer to regenerative therapy for muscular disorders - Medical Xpress

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