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

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

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

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

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

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

IPS Cell Therapy Comments Off on Daiichi Sankyo invests in Osaka University spin-off – The Pharma Letter (registration) | August 7th, 2017

Gary Campbell's partner, Leanne Crawford, with helpers Lauryn MacKenna (9) and Kye Crawford (12) at the home baking stall at Sunday's fun day.PICTURE IAIN FERGUSON, THE WRITE IMAGE

Two fundraising events have been held in Lochaber at the weekend to help send a local man halfway round the world for ground-breaking treatment.

Gary Campbell, from Caol, who is just 29, was diagnosed with progressive MS in April.

But family, friends and supporters are pulling out all the stops to raise 45,000 in order to send him to Mexico for stem-cell treatment.

Hematopoietic stem cell transplantation, HSCT, involves the intravenous infusion of stem cells collected from bone marrow or peripheral blood.

On Saturday, youngsters and their parents took park in a teddy bear toddle to raise cash for Mr Campbells cause, while yesterday, a large crowd attended a fun afternoon at An Aird in Fort William.

Leanne Crawford, Garys partner, said: The fun day went really well and there was a good turnout.

There were lots of fund-raising activities including a charity shinty match, bouncy castle, beat the goalie, a nail bar, home baking and raffles with loads of prizes.

Gary used to play shinty and two of his old teams from Caol and Banavie took part. Some people had obviously not played in a wee while and were falling about, but it was great fun.

Unfortunately, Gary wasnt well enough to come along which was a pity as he would have really enjoyed it.

He had a fall in the garden on Saturday night and I couldnt get him up. Fortunately his mum was there and between us we managed to get him back into the wheelchair.

His right leg was shaking constantly it was really stressful for him.

Ms Crawford said she has known Mr Campbell for nine years, and for the past five has noticed different symptoms.

He wasnt very good on his feet and sometimes his legs would give way. It was as though he was drunk when he hadnt been drinking.

Gary actually thought he had a brain tumour and, in a way, it was a relief when he was diagnosed with MS.

Ms Crawford said she is hoping by the time enough cash is raised, HSCT might be available closer to home.

I believe hospitals in Galway and Sheffield are looking into the treatment, but if he has to go to Mexico, Ill get him on the plane even though he is petrified of flying.

I dont know how much has been raised yet from these events, but we collected 663 from a recent baking sale and have 2,500 already on the just giving page. We also have 1,500 in a bank account collected from fund-raising events.

Gary is over the moon with the support he is receiving.

Another Lochaber resident with MS, Frances OConnell, received HSCT in Mexico last year.

Excerpt from:
Fun weekend activities will help send Lochaber man for ground-breaking MS treatment - Press and Journal

Bone Marrow Stem Cells Comments Off on Fun weekend activities will help send Lochaber man for ground-breaking MS treatment – Press and Journal | August 7th, 2017

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Cardiac Stem Cells Comments Off on Funding debate aside, this is why we need a new heart hospital – The Sydney Morning Herald | August 7th, 2017

A syrinx is a fluid-filled cavity within the spinal cord (syringomyelia) or brain stem (syringobulbia). Predisposing factors include craniocervical junction abnormalities, previous spinal cord trauma, and spinal cord tumors. Symptoms include flaccid weakness of the hands and arms and deficits in pain and temperature sensation in a capelike distribution over the back and neck; light touch and position and vibration sensation are not affected. Diagnosis is by MRI. Treatment includes correction of the cause and surgical procedures to drain the syrinx or otherwise open CSF flow.

Syrinxes usually result from lesions that partially obstruct CSF flow. At least half of syrinxes occur in patients with congenital abnormalities of the craniocervical junction (eg, herniation of cerebellar tissue into the spinal canal, called Chiari malformation), brain (eg, encephalocele), or spinal cord (eg, myelomeningocele). For unknown reasons, these congenital abnormalities often expand during the teen or young adult years. A syrinx can also develop in patients who have a spinal cord tumor, scarring due to previous spinal trauma, or no known predisposing factors. About 30% of people with a spinal cord tumor eventually develop a syrinx.

Syringomyelia is a paramedian, usually irregular, longitudinal cavity. It commonly begins in the cervical area but may extend downward along the entire length of the spinal cord.

Syringobulbia, which is rare, usually occurs as a slitlike gap within the lower brain stem and may disrupt or compress the lower cranial nerve nuclei or ascending sensory or descending motor pathways.

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Syrinx of the Spinal Cord or Brain Stem - Neurologic ...

Spinal Cord Stem Cells Comments Off on Syrinx of the Spinal Cord or Brain Stem – Neurologic … | August 7th, 2017

Dr. Gabe Mirkin

Sam Shepard was a prolific playwright, actor, screenwriter and director who:

acted in more than sixty films and was nominated for an Academy Award for Best Supporting Actor for his portrayal of pilot Chuck Yeager inThe Right Stuff;

wrote more than 55 plays, often focusing on the serious problems that occur in American family life;

won the most Obie Awards (10) for his off-Broadway writing and directing. In 1979 he received a Pulitzer Prize for his play, Buried Child, andNew York Magazinecalled him the greatest American playwright of his generation.

In his late sixties, he developed amyotrophic lateral sclerosis (ALS), the disease that killed baseball great Lou Gehrig at age 37. Shepard died from complications of ALS on July 27, 2017, at age 73.

A Difficult Life

Sam Shepard

He was born on November 5, 1943, in Fort Sheridan, Illinois. His dysfunctional family served as a basis for characters in many of his plays. His father was a United States Army Air Forces bomber pilot during World War II who was also an alcoholic and an abusive husband and father. His loving, supportive mother, a teacher, offset some of the pain and abuse he suffered from his father. In his early years, the family had to move every two years because of army transfers. Later his father left the service and bought an avocado farm in Duarte, Calif. Shepard briefly studied animal husbandry at nearby Mt. San Antonio College, but soon left school to move to New York City, where he worked as a busboy, played in a psychedelic folk band and tried to break into the theater.

At age 35, his acting career took off when he won a role in Terrence MalicksDays of Heaven, with Richard Gere and Brooke Adams. At the same time, he continued to write successful plays and in 1986 (age 43) he was elected to the American Academy of Arts and Letters.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrigs Disease)

In his last few years, Shepard suffered privately from ALS, but he described his experience in his last book, The One Inside. One of the characters said that he couldnt get up from bed in the morning and felt as though his limbs werent connected to the motor driving his body. They wont take direction wont be dictated to the arms, legs, feet, hands. Nothing moves. Nothing even wants to. The brain isnt sending signals.

ALS is a progressive disease that destroys the nerves that move voluntary muscles. More than 6,000 people in the United States are diagnosed with ALS each year. Nobody knows the cause and there is no cure. The brain is supposed to send messages to nerves in the spinal cord which transmit messages to the nerves that move muscles. When a muscle loses its nerve control, it starts to twitch and can waste away to nothing. Early symptoms of ALS include

muscle weakness

twitching

slurred speech

inability to chew food

tripping or stumbling.

The first sign could be difficulty buttoning a shirt, writing, or turning a key in a lock. The disease usually does not affect a persons ability to think and reason, so affected people are terribly disturbed by their lack of ability to control their voluntary muscles. As the disease progresses, a person loses the ability to speak, eat, walk, and eventually breathe. The most common cause of death is inability to breathe, which typically occurs about 3-5 years after symptoms start. Only about ten percent of affected people live more than ten years after first being diagnosed.

Risk Factors and Diagnosis

The disease usually starts between the ages of 55 and 75, but there are no known specific risk factors. Military veterans appear to be twice as likely as non-veterans to develop ALS. Possible causes could be exposure to occupational or environmental toxins such as lead or pesticides, infections or trauma. Family history does not appear to predict the disease.

There are no specific tests to diagnose ALS. It is usually diagnosed by a history of the symptoms, physical examination and ruling out other causes.

Current Treatments and Research

The U.S. Food and Drug Administration (FDA) has approved riluzole (Rilutek) and edaravone (Radicava) to treat ALS. These drugs offer no hope for a cure, but Riluzole appears to protect nerves by decreasing glutamate, the chemical messenger for nerves that innervate muscles. Intravenous edaravone possibly slows loss of muscle function, but it costs $1,086 per infusion or a yearly cost before government discount of $145,524. Another drug under European review is being developed by French drug maker AB Science SA (ABS.PA). Since there is no cure, all patients should receive physical therapy and speech therapy because inactivity itself causes loss of muscle function.

Since ALS is caused by the death of nerve cells that cause muscles to contract, the most promising line of research is through stem cells. Stem cells are young cells that can become any type of tissue. Treatment in the future may be to program stem cells to become nerve cells that innervate muscles and then inject them into areas where the nerve cells have already died.

Dr. Gabe Mirkin is a Villager. Learn more at http://www.drmirkin.com

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Sam Shepard and Amyotrophic Lateral Sclerosis - Villages-News

Spinal Cord Stem Cells Comments Off on Sam Shepard and Amyotrophic Lateral Sclerosis – Villages-News | August 7th, 2017

Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntingtons disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

By creating HD in a dish, the researchers say they have taken a major step forward in efforts to better understand what disables and kills the cells in people with HD, and to test the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

Although the autosomal dominant gene mutation responsible for HD was identified in 1993, there is no cure. No treatments are available even to slow its progression.

The research, published in the journal Cell Stem Cell, is the work of a Huntingtons Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups. The consortium studied several other HD cell lines and control cell lines in order to make sure results were consistent and reproducible in different labs.

The general midlife onset and progressive brain damage of HD are especially cruel, slowly causing jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and eventually death. In some cases (as in the patient who donated the material for the cells made at Johns Hopkins), the disease can strike earlier, even in childhood.

Having these cells will allow us to screen for therapeutics in a way we havent been able to before in Huntingtons disease, says Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the studys lead researchers. For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic.

Ross and his team, as well as other collaborators at Johns Hopkins and Emory University, are already testing small molecules for the ability to block HD iPSC degeneration. These small molecules have the potential to be developed into novel drugs for HD.

The ability to generate from stem cells the same neurons found in Huntingtons disease may also have implications for similar research in other neurodegenerative diseases such as Alzheimers and Parkinsons.

To conduct their experiment, Ross took a skin biopsy from a patient with very early onset HD. When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.

Her skin cells were grown in culture and then reprogrammed by the lab of Hongjun Song, Ph.D., a professor at Johns Hopkins Institute for Cell Engineering, into induced pluripotent stem cells. A second cell line was generated in an identical fashion in Dr. Rosss lab from someone without HD. Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

Scientists at Johns Hopkins and other consortium labs converted those cells into generic neurons and then into medium spiny neurons, a process that took three months. What they found was that the medium spiny neurons deriving from HD cells behaved just as they expected medium spiny neurons from an HD patient would. They showed rapid degeneration when cultured in the lab using basic culture medium without extensive supporting nutrients. By contrast, control cell lines did not show neuronal degeneration.

These HD cells acted just as we were hoping, says Ross, director of the Baltimore Huntington's Disease Center. A lot of people said, Youll never be able to get a model in a dish of a human neurodegenerative disease like this. Now, we have them where we can really study and manipulate them, and try to cure them of this horrible disease. The fact that we are able to do this at all still amazes us.

Specifically, the damage caused by HD is due to a mutation in the huntingtin gene (HTT), which leads to the production of an abnormal and toxic version of the huntingtin protein. Although all of the cells in a person with HD contain the mutation, HD mainly targets the medium spiny neurons in the striatum, part of the brains basal ganglia that coordinates movement, thought and emotion. The ability to work directly with human medium spiny neurons is the best way, researchers believe, to determine why these specific cells are susceptible to cell stress and degeneration and, in turn, to help find a way to halt progression of HD.

Much HD research is conducted in mice. And while mouse models have been helpful in understanding some aspects of the disease, researchers say nothing compares with being able to study actual human neurons affected by HD.

For years, scientists have been excited about the prospect of making breakthroughs in curing disease through the use of stem cells, which have the remarkable potential to develop into many different cell types. In the form of embryonic stem cells, they do so naturally during gestation and early life. In recent years, researchers have been able to produce induced pluripotent stem cells (iPSCs), which are adult cells (like the skin cells used in Rosss experiments) that have been genetically reprogrammed back to the most primitive state. In this state, under the right circumstances, they can then develop into most or all of the 200 cell types in the human body.

The other members of the research consortium include the University of Wisconsin School of Medicine, Massachusetts General Hospital and Harvard Medical School, the University of California, San Francisco, Cardiff University the Universita degli Studi diMilano and the CHDI Foundation.

Primary support for this research came from an American Recovery and Reinvestment Act (ARRA) grant (RC2-NS069422) from the National Institutes of Healths National Institute of Neurological Disorders and Stroke and a grant from the CHDI Foundation, Inc.

Other Johns Hopkins researchers involved in this study include Sergey Akimov, Ph.D.; Nicolas Arbez, Ph.D.; Tarja Juopperi, D.V.M., Ph.D.; Tamara Ratovitski; Jason H. Chiang; Woon Roung Kim; Eka Chighladze, M.S., M.B.A.; Chun Zhong; Georgia Makri; Robert N. Cole; Russell L. Margolis, M.D.; and Guoli Ming, M.D., Ph.D.

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Turning Skin Cells Into Brain Cells - 06/28/2012

Skin Stem Cells Comments Off on Turning Skin Cells Into Brain Cells – 06/28/2012 | August 7th, 2017

A proof-of-concept study in mice has demonstrated how skin grafts could deliver gene therapy for obesity and diabetes.

'We think this platform has the potential to lead to safe and durable gene therapy, in mice and we hope, someday, in humans, using selected and modified cells from skin,' said senior author Dr Xiaoyang Wu of the University of Chicago, Illinois.

The technique explores the potential of glucagon-like peptide 1 (GLP1), a hormone which could help to treat conditions like diabetes and obesity. GLP1 reduces appetite and stimulates the release of insulin to lowerblood sugar, butdoes not last long in the blood and is challenging to deliver orally.

The researchers used CRISPR to edit skin stem cellstaken from newborn mice. They inserted a modified version of the GLP1 gene, designed to increase the duration of the hormone, and a genetic'switch' to turn the gene on in the presence of an antibiotic.

They grew the skin stem cells into a skin organoids, and grafted them onto mice. When the mice were fed small amounts of antibiotic, theysuccessfully produced modified GLP1, which lasted for three months, and showed higher levels of insulin and lower levels of glucose.

The researchers also tested feeding the mice a high-fat diet. Compared to controls, the mice with modified GLP1 skin grafts put on less weight.

Dr Wu said the skin graft method could be safer than using engineered viral vectorsto edit genes in patient's own tyissues, as viruses 'may cause a very strong immune reaction and inflammation in vivo.' He added that lab-grown skin grafts have been used clinically for some time to treat burns, and have been proven safe.

Being able to control the gene expression using a drug would also allow doctors to calibrate how much of the enzyme enters a patients bloodstream.

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

Dr Jeffrey Millman of Washington University, St Louis, who was not involved in the study, told The Scientist that more research would be needed to ensure that neither the CRISPR editing nor the stem cell culturing method inadvertently introduce dangerous mutations.

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Gene therapy skin grafts for obesity and diabetes - BioNews

Skin Stem Cells Comments Off on Gene therapy skin grafts for obesity and diabetes – BioNews | August 7th, 2017

Stem cells have been cultured to treat many different of conditions

Lewis Houghton/Science Photo Library

By Andy Coghlan

Last week, two people with type 1 diabetes became the first to receive implants containing cells generated from embryonic stem cells to treat their condition. The hope is that when blood sugar levels rise, the implants will release insulin to restore them to normal.

About 10 per cent of the 422 million people who have diabetes worldwide have type 1 diabetes, which is caused by the bodys immune system mistakenly attacking cells in the pancreas that make insulin. For more than 15 years, researchers have been trying to find a way to use stem cells to replace these, but there have been several hurdles not least, how to get the cells to work in the body.

Viacyte, a company in San Diego, California, is trying a way to get round this. The firms thumbnail-sized implant, called PEC-Direct, contain cells derived from stem cells that can mature inside the body into the specialised islet cells that get destroyed in type 1 diabetes.

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The implant sits just below the skin, in the forearm, for example, and is intended to automatically compensate for the missing islet cells, releasing insulin when blood sugar levels get too high.

If it works, we would call it a functional cure, says Paul Laikind, of Viacyte. Its not truly a cure because we wouldnt address the autoimmune cause of the disease, but we would be replacing the missing cells.

The device has already been safety tested in 19 people with diabetes, using smaller numbers of stem cells. Once implanted, the progenitor cells housed in the device did mature into islet cells, but the trial didnt use enough stem cells to try to treat the condition.

Now Viacyte has implanted PEC-Direct packages containing more cells into two people with type 1 diabetes. A third person will also get the implant in the near future. Once inside the body, pores in the outer fabric of the device allow blood vessels to penetrate inside, nourishing the islet progenitor cells and exposing them to growth factors that push them to mature. Once these cells have matured which should take about three months the hope is that they will be able to monitor sugar levels in the blood, and release insulin as required.

If effective, it could free people with type 1 diabetes from having to closely monitor their blood sugar levels and inject insulin, although they would need to take immunosuppressive drugs to stop their bodies from destroying the new cells.

If successful, this strategy could really change the way we treat type 1 diabetes in the future, says Emily Burns of the charity Diabetes UK. A similar way to treat the condition with pancreas cells from organ donors has been in use for nearly 20 years, successfully freeing recipients from insulin injections, but a shortage of donors limits how many people are able to have this treatment.

This isnt a problem with stem cells. The embryonic stem cells used to make the progenitor cells originally came from a spare early stage embryo donated by a woman who was having IVF. Because embryonic stem cells, and the progenitor cells made from them, can be multiplied in limitless amounts, Laikand says that, if the treatment works, the method would be able to treat everyone who has the condition.

A limitless source of human insulin-producing cells would be a major step forward on the journey to a potential cure for diabetes, says James Shapiro at the University of Alberta, Canada, who has collaborated with Viacyte on this project, and who pioneered the donor pancreas method decades ago. For sure, this will in the end prove to be a durable landmark for progress in diabetes care.

More on these topics:

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First implants of stem-cell pouches to 'cure' type 1 diabetes - New Scientist

Skin Stem Cells Comments Off on First implants of stem-cell pouches to ‘cure’ type 1 diabetes – New Scientist | August 7th, 2017

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

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

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

With this technology we can convert skin cells into elements of any organ with just one touch, says co-author Chandan Sen. This process only takes less than a second and is non-invasive, and then you're off. The chip does not stay with you, and the reprogramming of the cell starts. Our technology keeps the cells in the body under immune surveillance, so immune suppression is not necessary."

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

The device works using a small electrical charge.

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

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

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

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

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

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

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

Skin Stem Cells Comments Off on Nano-chip promises to heal organs at a touch – Cosmos | August 7th, 2017

"By using our novel nanochip technology, injured or compromised organs can be replaced, said Dr Sen.

We have shown that skin is a fertile land where we can grow the elements of any organ that is declining.

TNT extends the concept known as gene therapy, which has been known about for some time, however the big difference is how the DNA is delivered into the body.

"The concept is very simple," said Professor James Lee, who co-led the research.

"As a matter of fact, we were even surprised how it worked so well.

In my lab, we have ongoing research trying to understand the mechanism and do even better.

So, this is the beginning, more to come."

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

The study is published in the journal Nature Nanotechnology.

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Penny-sized nanochip pad to regrow organs and heal injuries - Telegraph.co.uk

Skin Stem Cells Comments Off on Penny-sized nanochip pad to regrow organs and heal injuries – Telegraph.co.uk | August 7th, 2017

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

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

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

Doctors note that stem cell transplant is being proposed as an effective treatment for cancers such as leukaemia and lymphoma, and primary immune deficiency disorders. Stem cells do not develop normally in such patients and it affects the blood cells that they make. By a transplant, the patient gets new stem cells that can make new and healthy blood cells. Earlier, stem cells were collected from the bone-marrow. Now, it is being collected from blood cells.

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

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

Ajith Kumar V.T., professor, department of paediatrics, Government Medical College, Manjeri, said donors could not be found often from the same families because of the nuclear family system.

There are not many places where you can match the human leukocyte antigen (HLA) typing with donors. Another problem is the lack of stem cell registries in the State from where matching unrelated donors could be found.

Even if doctors suggest a stem cell transplant, many families dont opt for it because of the high cost involved. If the donor is from the same family, the cost is relatively low. But for unrelated donors, it is very high, Dr. Sidharthan said. The solution, Dr. Ajith Kumar said, was government intervention to set up HLA registries and bone marrow transplant centres. nestCare Foundation, a not-for-profit organisation based in the U.S., had recently approached us expressing interest to set up these facilities in the State. Talks are on, he said.

A.S. Jayanth

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Trust those cells to help cure cancer - The Hindu

Bone Marrow Stem Cells Comments Off on Trust those cells to help cure cancer – The Hindu | August 6th, 2017

Tom Parham remembers the time his Woodrow Wilson baseball team was playing Class AAA power Huntington East.

It was 1980, and the Flying Eagles were hosting the Pony Express at Harry Lewin Field. Not known to be a cavernous venue, the field lent itself to an offensive barrage and Huntington East was the last team standing.

A few weeks later, Parham led Woodrow to the state championship game and a rematch with the Express. Woodrow fell short again, but this time it was by the more purist-friendly score of 2-1.

It was then that Parham knew the Eagles needed a new field.

In stepped Doug Epling, Beckley businessman and community leader. He would later be known for refurbishing the old East Bank High School field for WVU Tech to use, as well as the construction of Linda K. Epling Stadium in Beckley, the home of the West Virginia Miners.

The latter, of course, bears the name of Epling's wife. The Tech field is named for Epling himself.The field he helped build on the Woodrow Wilson campus doesn't have an official name.

That will change Saturday.A ceremony will be held at 2 p.m. in the school cafeteria to officially rename the field for Thomas Parham.

The effort to honor the longtime coach was started by Sheila Brown.

"Words cannot describe how it feels," Parham said. "When Mrs. Brown started talking about it, I always told her, nah (modestly). I just thought, 'Let it go.' And finally she told me in April, 'Well, I'm going to the board. I'm going to ask them.' So she did and they told her what to do (at the next meeting)."

The Raleigh County School Board laid out a plan for Brown, and at the next meeting former coaches, colleagues and friends voiced their support.

Legendary boys basketball coach Dave Barksdale. State championship-winning football coach Pete Culicerto. Fellow New Hope Baptist Church member C.W. Claytor. Even Epling himself. They all showed up to see that Parham got the respect they feel he deserves.

"It was just touching to hear former coaches Coach Barksdale, Coach Culicerto, and I even heard from one of my coaching buddies from out of town, Ron Rose," Parham said. "He told Pete what (he wanted) to say. It was just touching, and a humbling experience."

Parham is being recognized for a career that spanned nearly three decades. He was hired as a biology teacher by Ross Hutchens before the start of the 1974-75 academic year.

"He said, 'I need a good biology teacher. I can get a coach anywhere,'" Parham said, laughing.

His first season as head baseball coach was 1975, and he remained there until his retirement in 2000. Along the way, his teams rolled up over 200 wins and appeared in the state tournament five times. Two of those trips resulted in runner-up finishes the 1980 meeting with Huntington East, and in 1983 against Martinsburg.

And the list of star players Parham coached seems endless Chuck Tate, Andy "Bam Bam" Wakefield, Larry Maiolo, Mason Basham, Larry Hickman, Joe Joe Maiolo, Larry Pat Farley, Phil Culicerto, Tim Epling, Phil Lane, Ronnie Fama, John O'Dell.

There were many others, and many of themare members of the Woodrow Wilson Baseball Hall of Fame.

"I was fortunate I came across some good ball players," Parham said. "You don't like to toot your own horn, but like a fella said, we put Woodrow Wilson baseball on the map."

Another was Ronnie Scott, who went on to work for NASA in Florida before returning to Beckley in 2010. Sadly, he passed away in May at age 59.

"He wanted to see baseball dominant again like it was when he played," Parham said.

When Parham retired from baseball in 2000 he stayed on as a biology teacher for one more year it was the emphatic end of an era at the school. Not only did Parham retire, but Culicerto retired after the 1999 football season, and Barksdale left the bench just months before Parham to take a coaching job in Aiken, S.C.

"Indeed it was," said Parham, now 74. "I enjoyed working with Coach Culicerto (as an assistant football coach). He was a great football coach, and he was a baseball supporter. He had seven sons play baseball for me. Three of them played in the state tournament."

After his retirement, Parham's was a familiar face in the stands at Woodrow baseball games. But in 2009, his ability to be a spectator slowed down when it was discovered that he had cancer.

Parham was diagnosed with multiple myeloma, a blood cancer that develops in the plasma cells located in bone marrow. The cancer did eventually go into remission, but Parham was still getting checkups when something told him he needed to go to Johns Hopkins in Baltimore.

It was there that he was introduced to autologous stem cell transplant. It's a procedure that involves collecting the patient's stem cells and following it up with high doses of chemotherapy or a combination of chemo and radiation. The process kills cancer cells while also killing blood-producing cells left in the bone marrow.

The collected stem cells are later transplanted back into the patient, allowing the marrow to produce new blood cells.

"I met a very interesting and a caring doctor up there, Dr. (Ivan) Borrello. He told me (about the transplant)," Parham said. "As a matter of fact, they have been doing this since 1980. He asked, 'What do you think about a stem cell transplant?' And I said yeah. Anything to get rid of this cancer.

"At that time my cancer was in remission, so he couldn't do anything. He said we would have to wait until it comes back. He hoped it didn't come back, but if it does ..."

It did, and in February he had the procedure performed.

"It came back in 2016, and when you're over 70 they don't usually do these things," Parham said. "But he felt like I was in good shape, which I think well, I know I am. I went through it, successful, no problems whatsoever.

"After teaching biology, I thought I knew some things. Now I know I know some things."

Just like baseball.

Email: gfauber@register-herald.com and follow on Twitter @GaryFauber

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Woodrow Wilson baseball field to be renamed for Tom Parham - Beckley Register-Herald

Bone Marrow Stem Cells Comments Off on Woodrow Wilson baseball field to be renamed for Tom Parham – Beckley Register-Herald | August 6th, 2017

What causes the body to age?

The Greek Philosopher Aristotle thought it was the hearta hot, dry organ at the seat of intelligence, motion and sensation.

Fast-forward a few centuries, and the brain has overthrown the heart as master of thought. But its control over bodily agingif anywas unclear. Because each organ has its own pool of stem cells to replenish aged tissue, scientists have long thought that the body has multiple aging clocks running concurrently.

As it turns out, thats not quite right.

This week, a study published in Nature threw a wrench into the classical theory of aging. In a technical tour-de-force, a team led by Dr. Dongsheng Cai from the Albert Einstein College of Medicine pinpointed a critical source of aging to a small group of stem cells within the hypothalamusan ancient brain region that controls bodily functions such as temperature and appetite.

Like fountains of youth, these stem cells release tiny fatty bubbles filled with mixtures of small biological molecules called microRNAs. With age, these cells die out, and the animals muscle, skin and brain function declines.

However, when the team transplanted these stem cells from young animals into a middle-aged one, they slowed aging. The recipient mice were smarter, more sociable and had better muscle function. Andget thisthey also lived 10 to 15 percent longer than mice transplanted with other cell types.

To Dr. David Sinclair, an aging expert at Harvard Medical School, the findings represent a breakthrough in aging research.

The brain controls aging, he says. I can see a day when we are implanted with stem cells or treated with stem cell RNAs that improve our health and extend our lives.

Its incredible to think that a tiny group of cells in one brain region could be the key to aging.

But to Cai, there are plenty of examples throughout evolution that support the theory. Experimentally changing a few of the 302 neurons in the nematode worm C. elegans is often sufficient for changing its lifespan, he says.

Of course, a mammalian brain is much more complicated than a simple worm. To narrow the problem down, Cai decided to zero in on the hypothalamus.

The hypothalamus has a classical function to regulate the whole bodys physiology, he says, so theres a natural logic for us to reason that the hypothalamus might be involved in aging, which was never studied before.

Even so, it was a high-risk bet. The hippocampusbecause of its importance in maintaining memory with ageis the most popular research target. And while the hypothalamus was previously somehow linked to aging, no one knew how.

Cais bet paid off. In a groundbreaking paper published in 2013, he found that a molecule called NF-kappaB increased in the hypothalamus as an animal grew older. Zap out NF-kappaB activity in mice, and they showed much fewer age-related symptoms as they grew older.

But heres the kicker: the effects werent limited to brain function. The animals also better preserved their muscle strength, skin thickness, bone and tendon integrity. In other words, by changing molecules in a single part of the brain, the team slowed down signs of aging in the peripheral body.

But to Cai, he had only solved part of the aging puzzle.

At the cellular level, a cornucopia of factors control aging. There is no the key to aging, no single molecule or pathway that dominates the process. Inflammation, which NF-kappaB regulates, is a big contributor. As is the length of telomeres, the protective end caps of DNA, and of course, stem cells.

Compared to other tissues in the body, stem cells in the brain are extremely rare. So imagine Cais excitement when, just a few years ago, he learned that the hypothalamus contains these nuggets of youth.

Now we can put the two threads together, and ask whether stem cells in the hypothalamus somehow regulate aging, he says.

In the first series of experiments, his team found that these stem cells, which line a V-shaped region of the hypothalamus, disappear as an animal ages.

To see whether declined stem cell function contributes to aging, rather as a result of old age, the researchers used two different types of toxins to wipe out 70 percent of stem cells while keeping mature neurons intact.

The results were striking. Over a period of four months, these mice aged much faster: their muscle endurance, coordination and treadmill performance tanked. Mentally, they had trouble navigating a water maze and showed less interest in socializing with other mice.

All of these physiological changes reflected an acceleration in aging, Cai and team concluded in their article.

And the consequences were dire: the animals died months earlier than similar transgenic animals without the toxin treatment.

If the decline in stem cell function is to blame for aging, then resupplying the aged brain with a fresh source of stem cells should be able to reinvigorate the animal.

To test this idea, the team isolated stem cells from the hippocampus of newborn mice, and tinkered with their genes so that they were more resilient to inflammation.

We know the aged hypothalamus has more inflammation and that hurts stem cells, so this step was necessary, explained the authors.

When transplanted into middle-aged mice, they showed better cognitive and muscular function four months later. Whats more, they lived, on average, 10 percent longer than mice transplanted with other cell types. For a human, that means extending an 85-year life expectancy into 93. Not too shabby.

But the best was yet to come. How can a few cells have such a remarkable effect on aging? In a series of follow-up experiments, the team found that the pool of biological molecules called microRNAs was to thank.

microRNAs are tiny molecules with gigantic influence. They come in various flavors, bearing rather unimaginative names like 106a-5p, 20a-5p and so on. But because they can act on multiple genes at the same time, they pack a big punch. A single type of microRNA can change the way a cell workswhether it activates certain signaling pathways or makes certain proteins, for example.

While most cells make microRNAs, Cai found that the hypothalamus stem cells have a unique, very strong ability to pack these molecules up into blobs of membrane and shoot them out like a bubble gun.

Once outside the cell, the microRNAs go on a fantastic voyage across the brain and body, where they tweak the biology of other tissues.

In fact, when the team injected purified little bubbles of microRNAs into middle-aged mice, they also saw broad rejuvenating effects.

Cai explains: we dont know if the microRNAs are pumped out to directly affect the rest of the body, or if they first act on different areas of the brain, and the brain goes on to regulate aging in the body.

Even so, the aging field is intrigued.

According to Dr. Leonard Guarente, an aging biologist at MIT, the study could lead to new ways to develop anti-aging therapies.

Whats more, its possible the intervention could stack with other known rejuvenating methods, such as metformin, young blood or molecules that clean out malfunctioning cells.

Its possible that stem-cell therapy could boost the hypothalamus ability to regulate aging. However, scientists still need to know how stem cells link with the hypothalamus other main role, that is, releasing hormones.

Of course, injecting cells into the brain isnt a practical treatment. The team is now working hard to identify which of the thousands of types of microRNAs control aging and what exactly they do.

Then the goal is to validate those candidate anti-aging microRNAs in primates, and eventually, humans.

Of course humans are more complex. However, if the mechanism is fundamental, you might expect to see effects when an intervention is based on it, says Cai.

Stock Media provided by digitalreflections / Pond5

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Breakthrough Stem Cell Study Offers New Clues to Reversing Aging - Singularity Hub

Skin Stem Cells Comments Off on Breakthrough Stem Cell Study Offers New Clues to Reversing Aging – Singularity Hub | August 6th, 2017

Very few people have ever heard ofthe concept of artificial skin transplants.That will change in the near future, though. Artificial skin transplants may be the one thing we need most to treat type 2 diabetes. The skin grafts based on CRISPR gene editing couldyield some very powerful results. Their first tests involving mice werepositive, butensuring the technology works for humans in the same way will besomething else entirely.

Alot of people may not like the sound of artificial skin transplants. It sounds a lot scarier than it really is, however. There is actually nothing to fear about them. In fact, we have been using artificial skin implants for several decades now.Burn patients often recover thanks to these implants, for example. Artificial skin implants have proven to be an invaluable tool in the world of healthcare so far, and it seems thenumber of use cases may be expanded upon. However,they havenever been deployed to treat diabetesup untilnow.

Scientists have now successfully used these implants to treat diabetes in mice. That is a major development in medicine. The researchers edited stem cells from newborn mice to control the release of ahormone stimulating insulin production. Once the cells were turned into skin grafts, they were given to mice suffering from diabetes.

The mice were not born with diabetes. Instead, researchers fed them high-fat diets to causeobesity. Acruel method, perhaps, thoughit is not uncommon to see this sort of thingin the medical sector. Obesity is still one of the main risk factors causing type 2 diabetes in humans. People with a high insulin resistance are particularly prone to developing thecondition. Diabeteswas induced in these mice usingsome modifications to create viable test criteria.

Once the mice received the artificial skin implants, their insulin resistance levels started to reverse. Additionally, they gained around half the weight as those not given the grafts. Thissuggests that people cantreat diabetes usingthese implants, although theywill not do much for anyone suffering from type 1 diabetes. Thosewho do suffer from that condition may soon have access to a cheap and efficient solution created from stem cells. The goal is to turn these stem cells into human skin over time.

There may be other clinical applicationsinvolving artificial skin implants we have yet to discover. Ever since doctors started treating burn patients with this technique, the quest to find other use cases has been in full effect. Thanks torecent breakthroughsin this field, one can now grow artificial skin in a lab. However, given the lack of human test subjects, finding other use cases has been pretty difficult. This is where the mice come into the picture, even though the results involving human subjects mightdiffer greatly.

This is not a cure for diabetes, but it is an approach to help people maintain their glucose levels. For now, it only works withtype 2 diabetes causedby obesity, but it is still an important breakthrough regardless. The bigger question is what other types of diseases may be treated through artificial skin implants.

Original post:
Scientists Use Artificial Skin Implants to Treat Type 2 Diabetes The ... - The Merkle

Skin Stem Cells Comments Off on Scientists Use Artificial Skin Implants to Treat Type 2 Diabetes The … – The Merkle | August 6th, 2017

Genetically engineered skin cells grafted onto mice can treat the animals diabetes and obesity, according to new research published August 2, 2017 in Cell Stem Cell.

Researchers edited skin stem cells from newborn mice using CRISPR-based technology so that the cells secreted a peptide that regulates blood sugar. Transplanting the cells onto mice showed the grafts increased insulin secretion and reversed weight gain from a high-fat diet, as well as overturned insulin resistance. The result is a small step toward developing a safe and durable gene therapy to treat diabetes in humans.

Weve had this idea for a long time, so its exciting to see that, indeed, it can work to deliver therapeutics, coauthor Xiaoyang Wu, a stem cell biologist at the University of Chicago, tells GEN.

In the study, Wu and colleagues worked with skin because it is a large organ and easily accessible. The cells multiply quickly and are easily transplanted. And, transplanted cells can be removed, if needed. Skin is such a beautiful system, Wu says, noting that its features make it a perfect medium for testing gene therapies.

The team worked with the gene that produces glucagon-like peptide 1 (GLP-1), a hormone that stimulates the pancreas to secrete insulin. The additional insulin takes excessive glucose out of the bloodstream, which regulates complications from diabetes. The hormone can also decrease appetite. Using the genetic engineering tool CRISPR, the team inserted a mutation, adding an antibody fragment to the gene that would make the GLP-1 last longer in the blood and an additional modification to the targeting vector that would also attach an inducible promoter. This switch turns the gene on, as needed, to make more GLP-1. The switch would be triggered by the administration of the antibiotic doxycycline.

Wu and colleagues then inserted the altered gene into skin cells and grew the cells in a culture. Once the skin cells had grown into multiple layers, the team transplanted the patches onto mice with intact immune systems. Surprisingly, the mice didnt reject the graftsa feat in itselfsince human skin transplants are far more advanced than mice grafts, partly due to the animals furry skin.

Next, the team fed the mice small amounts of doxycycline. As a result, the animals released GLP-1 into the blood and had higher levels of insulin and lower levels of glucose. When fed a high-fat diet, the mice gained weight and became obese. But when the mice also were fed doxycycline so they secreted GLP-1, they gained less weight, showing the gene therapy was successful.

This kind of therapy could be potentially effective for many metabolic disorders, Wu says. The grafts could be used in patients who cant process protein or in individuals with hemophilia. The team is now testing the gene-therapy technique in combination with other medications.

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CRISPR Gene Therapy via Skin Grafts Treats Obesity and Diabetes in Mice - Genetic Engineering & Biotechnology News

Skin Stem Cells Comments Off on CRISPR Gene Therapy via Skin Grafts Treats Obesity and Diabetes in Mice – Genetic Engineering & Biotechnology News | August 6th, 2017

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

Skin Stem Cells Comments Off on Early gene-editing holds promise for preventing inherited diseases – The Jerusalem Post | August 6th, 2017

If you look at your skin, most of what you will see is actually dead cells. This thin outermost sheet protects the living cells underneath as they develop.

Thats why people exfoliatebecause your skin cells are designed to mature and slough off, says Thierry Work, a wildlife disease specialist with the U.S. Geological Survey. In June, he and his colleagues reported that they successfully mimicked this process with sea turtle skin. No, they didn't give sea turtles a facial, but they did grow layers of turtle skin in the lab.

Work is studying the virus responsible for a deadly disease called fibropapillomatosis, which causes tumors to grow all over sea turtles skin and inside their bodies. In order to grow this virus we have to basically replicate skin in the lab, because this virus will only grow when skin cells are maturing, he says. Cultivating the elusive virus may help researchers save sea turtles and perhaps shed light on how herpes viruses replicate in people.

Works efforts represent the first time that scientists have engineered reptile skin, but weve been building our own lab-grown versions of mammalian skin for decades. Lab-made skin can help us find treatments for diseases, heal wounds, spare animals from cosmetics testing, and design leather that emulates the look and feel of real animal hides. Heres how were imitating skin to benefit humans and other creatures.

Turtle trouble

Fibropapillomatosis most often strikes endangered green turtles, although it sometimes shows up in other species of sea turtles. The illness can debilitate turtles by growing tumors that prevent the animals from seeing properly or eating, and by suppressing the immune system.

To combat this disease, scientists must examine how the virus (Chelonid herpesvirus 5, or ChHV5 for short) multiplies in living cells. So Work and his team collected skin samples from green turtles with fibropapillomatosis that had just died or had to be euthanized. They then made a gel from collagenthe same protein that gives skin its firmnessand seeded it with cells taken from deep within the donated skin. Its this layer that we see when we examine a leather handbag or pair of shoes, Work says. Finally, his team grew surface skin cells on top of this scaffold.

The fruits of their labors are little plugs of skin only about 5 to 6 millimeters wide. But under the microscope, they look just like actual turtle skin, Work says.

Chelonid herpesvirus 5 and some other viruses (such as the one that causes warts in people) cant be grown in a lab without an environment that mimics the shape of skin and is populated by live, maturing cells.

Other viruses are less demanding, including herpes simplex (cause of cold sores and genital herpes in humans). Normally, herpes simplex and other viruses are grown on a flat lawn of skin cells in a petri dish. But this setting doesnt really recreate the shape and structure of real skin. So there may be insights were missing by cultivating viruses in a dish. We really havent seen what the virus does in the actual three-dimensional structure of reconstructed skin, Work says.

When he and his colleagues grew ChHV5 in their turtle skin, the pathogen did not behave as expected. The viruses formed odd looking, sun-shaped structures to serve as factories in the cells where the virus settled in and made copies of itself. The way all these components were being put togetherwas quite different than what people saw with conventional herpes viruses, Work says.

It could be that herpes simplex, and other herpes viruses that infect people, also form similar structures to assemble fresh recruits. The replication of the herpes virus is actually a lot more complicated than what people thought, Work speculates. The more we know about how viruses actually interact with cells, the more effective drugs we can design.

Now that scientists can cultivate ChHV5 in the lab, the next step is to come up with a blood test for fibropapillomatosis. That would tip them off that the disease is on the loose in a particular area before turtles begin to die. Theyre in a world of hurt, and by the time theyre heavily tumored its really too late to do anything, Work says.

It wont be practical to vaccinate or treat all the turtles directly. But we may be able to discourage the diseases transmission, the same way we use insecticides and bed nets to thwart mosquitoes from passing on malaria.

And now that reptile skin has been successfully grown in the lab, the technique could be put to work investigating other reptile and amphibian skin diseases. Engineered hide might help us learn more about snake fungal disease, which threatens snakes in the eastern and Midwestern United States, or the chytrid fungus that has infected frogs around the globe.

Healing wounds

Turtle skin doesnt have hair follicles or sweat glands, Work says. So its a bit easier to engineer than human skin. Nevertheless, the technique he used to emulate sea turtle pelts was adapted from ones commonly used to grow our own version of human skin.

Skin substitutes are an alternative to using grafts transplanted from elsewhere on a patients body to cover burns or other chronic wounds. Theres going to be a limited number of times that patients will allow the grafts to be taken from their thighs. These products that are ready-made became very popular because of that, says Vincent Falanga, an emeritus professor in the Boston University School of Medicines department of dermatology, whose studies on living bioengineered skin led to the U.S. Food and Drug Administration approving it to help non-healing wounds close more quickly.

Unlike skin grafts, bioengineered skin does not stick around, and eventually disintegrates. It does, however, protect the wound and stimulate the skins natural healing processes. Using bioengineered skin is a less painful process than undergoing skin grafts and may cause fewer complications, although its also more costly in general.

Bioengineered skin is often grown using cells taken from newborns foreskins. However, the vigorous young cells may stimulate the damaged area so much that it requires more energy than it can supply, Falanga says. Bioengineered skin that relies on less-active adult cells might actually be more effective in helping wounds to heal.

Skin substitutes dont quite function like the real thing. Last year, though, researchers in Japan reported that theyd grown realistic mouse skin from stem cells and successfully transplanted it onto other rodents. Previously when scientists grew skin from stem cells, they only managed to make sheets of cells emulating the skins outermost layer. But the new skin recreated all three of the layers found in skin, as well as boasting hair follicles and sebaceous glands (which make a fatty secretion to lubricate the skin).

Falanga is skeptical that this kind of engineered skin will perform well in chronic wounds. People in the bioengineering field, they want to reproduce whats already in nature, he says. We want to have a product that looks exactly like skin. Yet lingering wounds lack proper blood supply or have other problems that prevent them from healing. So they may be unable to maintain normally functioning skin, with its high energy demands.

However, the team in Japan hopes their lab-grown skin will eventually help people with burns, scars, or skin diseases like alopecia. "Up until now, artificial skin development has been hampered by the fact that the skin lackedimportant organs, such as hair follicles and exocrine glands, coauthor Takashi Tsuji, of the RIKEN Center for Developmental Biology, said in a press release. With this new technique, we have successfully grown skin that replicates the function of normal tissue.

The realistic skin also brings scientists closer to their dream of growing whole organs that can be transplanted into people, Tsuji said.

Sparing animals

But medical treatments aren't the only uses for skin substitutes. Tsuji and his team also have another goal in mind for their creation: Eventually, the realistic skin could be used for testing out cosmetics in lieu of animals.

There are already several companies devoted to just this purpose. Boston-based MatTek sells their lab-grown skin to other companies that make laundry detergent, makeup, anti-aging creams, and other chemicals.

Like Works sea turtle skin, these nubbins of skin are tinyjust a fraction of a millimeter thick. They are grown from skin cells left over after surgical procedures like tummy tucks and circumcisions. This skin is a better proxy for actual human hide than animals are, one of MatTeks customers told Wired last year.

Lab-made skin will soon provide an alternative to leather, too. Brooklyn-based startup Modern Meadow genetically engineers animal cells to produce collagen, which they use to make leather that resembles actual animal pelts. The biofabricated leather takes two weeks to grow, as opposed to the years it takes to raise an animal, slaughter it, and tan its hide. And because the cell-spun leather lacks features like hair and fat, its more eco-friendly to treat than the real thing.

Could Works lab-grown reptile hide also be used instead of skin from actual snakes or alligators for bags and shoes? Youd need to optimize this technique quite a bit before you got to that scale, he says. But I certainly think its possible.

Also apparently on the table: using DNA from a deceased fashion icon to grow skin for leather jackets and bags. Designer Tina Gorjanc wants to create artificial skin using Alexander McQueens genetic material, harvested from hair he used in a 1992 collection. Shes filed a patent in the United Kingdom for the process, although for now her prototypes are made from pig leather treated to look like human skin.

Read more from the original source:
Growing skin in a lab has benefits for humans and turtles alike - Popular Science

Skin Stem Cells Comments Off on Growing skin in a lab has benefits for humans and turtles alike – Popular Science | August 5th, 2017

Most nights, Marcia Dunlap attaches seven or eight leeches around her husband John's eyes as part of an effort to restore some of his vision. Tom Bailey/The Commercial Appeal

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

At home most evenings, Memphis, Tennessee, attorney John Dunlap, 80, unbuttons and removes his white dress shirt and counting his steps and remembering which way to turn carefullywalks with a tall white canefrom the living room to the dining table, where his wife Marcia has a plastic container of leeches.

Twenty-six months ago,the couple's schizophrenic sonAndrewattacked them in theirhome. The injuries blinded Dunlap. He's in total darkness.

After drapinga large, peach-colored towel around John's neck, Marcia reaches into the water for the skinniest leeches. Those are the hungriest and most likely to latchonto John's face.

One at a time, she gently presses four leeches to the skin around John's left eye and three around the right. She waits patiently wait for eachto bite and stay connected to John's skin.

"You can feel a bite,'' he says. "A little, stinging bite... And then after awhile you don't feel anything.''

The Dunlaps have carried out this unusualroutine60 or so times since December. It's a type of therapy prescribed by a Los Angeles doctor who offers experimental stem cell therapy designed to regenerate tissue.

"In the beginning he made it very clear he's not anophthalmologist and not an eye surgeon but he had had some success with stem cells in treating blindness. It's experimental,'' Dunlap said.

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The doctor prescribed the leech therapy as a preliminary step because, Dunlap said, the leech enzymesenhance the blood supply to the eye and nourishthe eye tissue.

The left eye had atrophied, or withered. The idea wasto restore health to the eyebefore the stem cell treatment. There is no right eye, but the hope is that the leech enzymes will help revive that optic nerve in case a transplant is ever possible.

Since the leech therapy,the pressure in the right eye has improved significantly, Dunlapsaid, referring to follow-upexams. The retina, which had folded into an ice-cream cone shape after the trauma, has begun returning to its normal shape, he said.

Even though he still cannot see out of the left eye and the optic nerve remains severed from the retina, Dunlap said, "I now have a live eye.''

The Dunlaps decline to identify the California doctor, describing him as a"humble'' person whodoes not seek the publicity.

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

Andrew, the Dunlaps' mentally ill son, is charged with attempted murder and domestic assault, and remains in jail awaiting trial. Thecouplehave told authorities that they mainly want Andrew to receive mental health treatment.

The Dunlapshave experienced tragedy long before the 2015 assault.

Their son Jeff, one of four children, was a St. Jude Children's Research Hospital patient who died of cancer at age 10, in September 1974.

Dunlap recalls a return car tripfrom Knoxville, where he and Marcia had been visiting grandchildren shortly after he was released from rehab.

"As we were driving back I started thinking of all the things I won't get to do again. In my mind, I was going down the list,'' he said.

It would be a long list, including some leisure activities he loves. An avid Cubs fan, heenjoyed attending spring training games in Arizona. A passionate golfer, he enjoyedwatching how the ball flew when he struck it well.

But Dunlap stopped himself from completing the list of losses, telling himself, " 'You don't want to dwell on that'. . . It's as if the Lord sent me a message that hit me across my forehead, saying, 'John, get over it. It could be a whole lot worse.'

"Anytime I want to start thinking about the things I'm missing or not doing what I used to do, I think 'Get over it. Move on'.''

Sudden blindness is such a change in lifestyle. "I guess some people may feel the world has ended for them, but it hasn't,'' he said.

Marcia Dunlap gets special leeches for her husband John's nightly treatment from the laundry room where she keeps it out of sight. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

The stem cell and leech therapy is expensive and not covered by health insurance. Some have expressed their skepticism about the legitimacy of the experimental treatments.

"You have some people who are concerned for you, that your approach is not going to be effective,'' Dunlap said.

"Yet, several folks up herehave said, 'John, I'd take a shot at it. It is expensive but you're the one with the white cane and the one who is blind and has to live with it. You have everything to gain and nothing to lose.'''

While some might be concerned about the unusual treatments, many others are inspired by the Dunlaps,saidBlanche Tosh, a fellow church member and friend since high school.

"I have told them so many times, 'You just can't begin to know the lives you have affected,'' Tosh said.

"I know so many people who look at the way they are dealing with multiple things. How could anybody endure that and just go on and be pleasant and make it from day to day with the consistent attitude that the world sees.

"You are not going to find many people whoever see one of them without a smile,'' Tosh said.

She was inspired to start a gofundme account (gofundme.com/johndunlapvision) to help coverthe Dunlaps' expenses. As of midweek, $8,795 of the $100,000 goal had been raised.

Memphis lawyer John Dunlap and his wife Marcia continue to search for some medical procedure to restore at least partial vision after John was blinded a few years ago when their mentally ill son attacked him. (Photo: Jim Weber/The Commercial Appeal)

Since December, Dunlap has undergone two-and-a-half rounds of leech therapy and two series ofstem cell treatments. The couple traveled to California in June for the most recent stem cell procedures, and returned home with stem-cell eye drops and injections.

Nowthey are in the middle of the leech therapy they resumed this summer.

John has a follow-up exam next week, when he will learn if there's been continued progress from the stem cell and leech therapies.

The California doctor "indicated it would take two to three months to see if we were getting any results from stem cell therapy out there,'' Dunlap said. That time could come sometime this month or in September.

If the stem cell therapy has not worked by then, he said,"We'll just have to see what any third plan looks like, and the cost involved.''

Late in life, Dunlap has been forced to learn to type, work a computer, navigate with a cane, count the steps and memorize the turns from one spot to another, communicate with Siri, and smile as blood-sucking leeches dangle from his cheeks.

Asked about his sources of inner-strength, he responded, "I don't know I'd call it inner-strength.

"I can tell you I certainly believe in the Lord. We pray daily. I appreciate the prayers of others. I think it certainly is a faithissue.''

He also credits his late mother, Cora, a single parentwho managed a grocery. "She was a very optimistic, loving person,'' he recalled.

"And I've had Marcia's support. Marcia wasn't going to let me give up, just sit down and do nothing.''

The Dunlaps are starting to consider resuming their annual trips to Cubs spring training in Arizona. Maybe next spring.

"You may have your vision by then,'' Marcia told John.

"I might,'' he responded."We'll see.''

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BINGHAMTON (WBNG) -- Thursday, a cancer survivor met her bone marrow donor for the first time, just days before her wedding.

"They told me that without a transplant I really only had about six months to a year," said Vivian Nolan, a bone marrow transplant recipient.

In 2008, Vivian Nolan was diagnosed with a rare form of cancer called multiple myeloma. Later on, she was diagnosed with leukemia.

Doctors tried a bone marrow transplant with her own stem cells. When that didn't work, they said she needed a donor.

"The only cure or chance of holding it off at all is a bone marrow transplant," Nolan said.

Lucky for Nolan, doctors found a match.

A stranger volunteered to save her life. Scott Durbin is Nolan's donor. He lives in Kentucky, over 850 miles away.

Thursday, Durbin and Nolan met for the first time.

Nolan is getting married on Saturday.Durbin and his family flew in to support her in her next phase of life, a life that she wouldn't have without him.

"This is the man who gave me my life back. So I'm really happy," Nolan said.

For Durbin, the decision to help someone in need was second nature.

"I signed up. 7 months later I got that phone call saying they was gonna fly me to Atlanta," bone marrow donor Scott Durbinsaid.

Nolan was still in shock that someone would do something so kind for a person he had never met.

"I just couldn't believe that there was someone out there that I never knew that would go through that for me," she said.

After the transplant, Nolan wanted to meet the man who now is a part of her.

Today, she was able to introduce her family to its newest member.

"Now I've got this whole new life and he's got this whole big new family."

For Durbin, it's a choice he'd make over and over.

"I would do it again to give you a second chance," Durbin said.

Nolan remains forever grateful for that second chance.

Since her bone marrow transplant, Nolan's leukemia is virtually gone. She says she feels great, and can't wait for her new lease on life.

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In 2002, Ian Thompson, a specialist in facial reconstruction at Kings College, London, received an urgent phone call. A patient in his late 20s had been struck by an out-of-control car mounting the pavement. The impact had sent him catapulting over the bonnet of the car, smashing his face and shattering the fragile orbital floor the tiny bone, no more than 1mm thick, which holds the eyeball in place in the skull.

Without the orbital floor, your eye moves backwards into the skull, almost as a defensive mechanism, Thompson explains. But this results in blurred vision and lack of focus. This patient had also lost the ability to perceive colour. His job involved rewiring aircraft and as he could no longer detect a red wire from a blue one, hed barely been able to work in three years.

The accident had happened three years earlier. Since then, surgeons had desperately tried to reconstruct the bony floor and push the eye back into position, first using material implants and then bone from the patients own rib. Both attempts had failed. Each time, infection set in after a few months, causing extreme pain. And now the doctors were out of ideas.

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Thompsons answer was to build the worlds first glass implant, moulded as a plate which slotted in under the patients eye into the collapsed orbital floor. The idea of using glass a naturally brittle material to repair something so delicate may seem counterintuitive.

But this was no ordinary glass.

If you placed a piece of window glass in the human body, it would be sealed off by scar tissue, basically wobble around in the body for a while and then get pushed out, says Julian Jones, an expert in bioglass at Imperial College London. When you put bioglass in the body, it starts to dissolve and releases ions which kind of talk to the immune system and tell the cells what to do. This means the body doesnt recognise it as foreign, and so it bonds to bone and soft tissue, creating a good feel and stimulating the production of new bone.

Bioglass actually works even better than the patients own bone Ian Thompson

For Thompson, the results were immediate. Almost instantaneously, the patient regained full vision, colour and depth perception. Fifteen years on, he remains in full health.

Thompson has gone on to use bioglass plates to successfully treat more than 100 patients involved in car or motorcycle accidents. Bioglass actually works even better than the patients own bone, Thompson says. This is because weve found that it slowly leaches sodium ions as it dissolves, killing off bacteria in the local environment. So, quite by chance, you have this mild antibiotic effect which eliminates infections.

Cutting edge

Bioglass was invented by US scientist Larry Hench in 1969. Hench was inspired by a chance conversation on a bus with an army colonel who recently had returned from the Vietnam War. The colonel told Hench that while modern medical technology could save lives on the battlefield, it could not save limbs. Hench decided to shelve his research into intercontinental ballistic missiles and instead work on designing a bionic material which would not be rejected by the human body.

Hench ultimately took his research to London, and it has been in Britain where some of the most revolutionary bioglass innovations are being made in fields from orthopaedic surgery to dentistry.

Over the last 10 years, surgeons have used bioglass in a powdered form, which looks and feels like a gritty putty, to repair bone defects arising from small fractures. Since 2010, this same bioglass putty has hit the high street as the key component in Sensodynes Repair and Protect toothpaste, the biggest global use of any bioactive material. During the brushing process, the bioglass dissolves and releases calcium phosphate ions which bond to tooth mineral. Over time, they slowly stimulate regrowth.

But many scientists feel that the current applications of bioglass are barely scratching the surface of what could be possible. New clinical products are being developed which could revolutionise bone and joint surgery like never before.

Sitting in his office in Imperial Colleges Department of Materials, Jones is holding a small, cube-shaped object hes dubbed bouncy bioglass. Its similar to the current bioglass but with a slight twist: subtle alterations in the chemical composition mean its no longer brittle. Instead it bounces,like a kids power ball as Jones describes it, and its incredibly flexible.

The point of this is that it can be inserted into a badly broken leg and can support both the patients weight and allow them to walk on it without crutches, without requiring any additional metal pins or implants for support. At the same time, the bouncy bioglass also will stimulate and guide bone regrowth while slowly, naturally assimilating into the body.

To regenerate large pieces of bone, for example in a really big fracture, its very important to be able to put weight on your leg, Jones says. And its really important that the bio-implant in your leg is able to transmit the force from your weight to the bone cells, like a signal. Our body makes its own bone in the architecture that its in, because the cells feel the mechanical environment. So to grow back a big piece of bone you need to be able to transmit the right signals to them. The reason why astronauts in space lose bone mass is because without gravity, the cells arent receiving the same information as they do on Earth.

Further alterations to the chemical makeup of bioglass produce a different form which is much softer and has an almost rubbery feel. It feels almost like a piece of squid at a seafood restaurant. This bioglass is designed for possibly the holy grail of orthopaedic surgery: cartilage repair.

Right now, surgeons attempt to repair damaged cartilage in arthritic hips or damaged knee joints with a fiddly procedure called microfracture. This involves smoothing over the damaged area to expose the bone underneath, then pricking it to release stem cells from the bone marrow which stimulate repair. But this results in scar cartilage and within a few years, as many athletes have found, the original problem returns.

As a solution, Jones is looking to produce bioglass which can be 3D-printed and then slotted into any hole in the cartilage. For the cells to accept it, the material must retain all the natural properties of cartilage. To test its effectiveness, Jones uses a simulator that has human knee joints from cadavers donated for medical research.

We simulate the walking action, bending, all the things a knee would do, and make sure that the bioglass actually preserves the rest of the joint and behaves as it should do, he says. If that works then well proceed to animal and then clinical trials.

This same bioglass could find an additional use in aiding people with chronic back pain due to herniated discs. At the moment surgeons treat this by replacing the dysfunctional disc with a bone graft which fuses the vertebrae in the back together. But while this takes away the pain, it results in a considerable loss in mobility. Instead, a bioglass implant could be printed and simply inserted to replace the faulty disc.

It seems the obvious thing to do, Jones says. So far nobody has been able to replicate the mechanical properties of cartilage synthetically. But with bioglass, we think we can do it.

Weve just got to prove that we can. If all goes well and we pass all the necessary safety tests, it could reach the clinic in 10 years.

Using man-made materials which can fuse to the body may seem far-fetched but it is appearing to be a more and more likely component of future medicine. Already, millions of people brush their teeth with it. And that may just be the start.

This story is a part of BBC Britain a series focused on exploring this extraordinary island, one story at a time. Readers outside of the UK can see every BBC Britain story by heading to theBritain homepage; you also can see our latest stories by following us onFacebookandTwitter.

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