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Archive for April, 2017

Bone Marrow vs. Fat Derived Stem Cells Continued : Stem …

Whether your adult mesenchymal stem cells come from bone marrow or from fat probably does not make a difference in terms of clinical results. Although some centers claim that bone marrow derived cells are superior to fat derived cells, there is no evidence to substantiate that. Recent studies show that fat derived cells make bone tissue much better than the bone marrow derived cells. Some studies are showing different outcomes but it is important to realize that these studies are all done in petri dishes and may not relate to living organism. Also, it is important that one is not mislead in some marketing materials by the word bone in bone marrow, possibly implying that since this is an orthopedic source it must be better for treating orthopedic conditions such as cartilage regeneration. In fact, the bone marrow is part of the reticulo-endothelial system (makes blood cells) and just happens to be found in the center of bone. The truth is, both bone marrow derived and stromal (from fat) derived stem cells are both effective for regenerative therapy and both have the potential to differentiate into mature functional cartilage. However, stem cells from fat are 100 to 1000 times more plentiful and this makes same day procedures (allowed in the US) much more effective with fat derived cells. The higher numbers of cells in fat leads to better clinical outcomes. Also, the quality of bone marrow declines with age and it has less numbers of cells and less healthy cells compared to the fat. The diminution in quantity and quality of bone marrow cells related to age and chronic illness is well documented. Last but not least, the ease of removing fat from under the skin using a mini-liposuction under local anesthetic is much less invasive and MUCH LESS painful than undergoing bone marrow aspiration to obtain bone marrow cells.

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Scientists Can Now Turn Human Skin Cells Into Brain Cells – Wall Street Pit

Researchers at University of California, Irvine (UCI) have developed a method that can transform human skin cells into brain cells. With this amazing feat, scientists may be able to better understand what role inflammation plays in the progression of Alzheimers disease. And this knowledge could lay the groundwork towards developing more effective treatments and therapies to manage the condition.

Before this breakthrough, scientists relied mostly on mice microglia to study the immunology of Alzheimers. Microglia sometimes referred to as Hortega cells are a special kind of cell that can be found in the human brain and spinal cord. The primary role of these cells is to protect the brain and the spine from infections, disease and any invading microbe. They provide immune support for the entire central nervous system by removing dead cells, damaged cells and other debris.

Along this line, microglial cells also help keep healthy cells from degenerating managing inflammation as well as developing and maintaining the integrity of neural networks which is why they are believed to play a special role in delaying the progression of neurodegenerative conditions like Alzheimers.

While studying brain cells from mice is useful, studying the real thing is, of course, more preferable. And the method developed by the UCI team is a step in this direction.

Using skin cells donated by UCI Alzheimers Disease Research Center patients, the UCI team led by Edsel Abud, Mathew Blurton-Jones and Wayne Poon made use of a genetic process to reprogram the skin cells and turn them into induced pluripotent cells (iPSCs) adult cells that are modified to act like embryonic stem cells which can turn into any kind of cell or tissue. The iPSCs were then exposed to a series of differentiation factors which mimicked the developmental origin of microglia. This exposure resulted in cells that are pretty much like human microglial cells.

Instead of continuing to rely on mice microglial cells, scientists now have a more realistic model for studying human disease in order to develop new and better therapies. And they have now started on this new path. They are using the microglial-like cells in 3D brain models so they can study how these cells interact with other brain cells and understand how this interaction impacts the progression of Alzheimers and the development of other neurological conditions.

As explained by Professor Blurton-Jones in a statement they issued: Microglia play an important role in Alzheimers and other diseases of the central nervous system. Recent research has revealed that newly discovered Alzheimers-risk genes influence microglia behavior. Using these cells, we can understand the biology of these genes and test potential new therapies.

This latest breakthrough is once again proving how important stem cells are in helping understand biological processes, both under normal conditions and under disease-related conditions. Eventually, scientists are bound to stumble on that ultimate discovery that can hopefully be instrumental in combating diseases right at their source, so we can stop dealing with devastating diseases, especially those that affect the brain and threaten a persons life.

The study was recently published in the journal Neuron.

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Scientists Can Now Turn Human Skin Cells Into Brain Cells - Wall Street Pit

Stem cells edited to fight arthritis: Goal is vaccine that targets … – Science Daily


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Stem cells edited to fight arthritis: Goal is vaccine that targets ...
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Using CRISPR technology, a team of researchers rewired stem cells' genetic circuits to produce an anti-inflammatory arthritis drug when the cells encounter ...
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CRISPR-SMART Cells Regenerate Cartilage, Secrete Anti-Arthritis DrugGenetic Engineering & Biotechnology News

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Stem cells edited to fight arthritis: Goal is vaccine that targets ... - Science Daily

China Is Racing Ahead of the US in the Quest to Cure Cancer With CRISPR – Gizmodo

On Friday, a team of Chinese scientists used the cutting-edge gene-editing technique CRISPR-Cas9 on humans for the second time in history, injecting a cancer patient with modified human genes in hopes of vanquishing the disease.

In the US, the first planned trials to use CRISPR in people still have not gotten under way. But in China, things appear to be moving relatively quickly.

Last fall, a team at Sichuan Universitys West China Hospital used CRISPR for the first time on an adult with lung cancer. In the new trial, reported by The Wall Street Journal, altered genes were injected into a patient with a rare type of head and neck cancer, called nasopharyngeal carcinoma, at Nanjing Universitys Nanjing Drum Tower Hospital.

The aim is to use CRISPR, which allows scientists to snip out pieces of DNA with greater ease than older gene-editing techniques, to suppress the activity of a gene preventing the patients body from effectively fighting the disease. On Friday, the university announced that the first patient had received an infusion of altered cells, which are taken from their body and altered in a lab before being injected back in.

In all, 20 patients with gastric cancer, nasopharyngeal carcinoma and lymphoma are expected to participate in the trial. Its first phase is expected to conclude next year.

The other Chinese trial, in which scientists modified immune cells to attack lung cancer in 11 patients, expects to release results this year, according to the Journal.

The first US human CRISPR trial is slated to begin this summerat the University of Pennsylvania, after receiving a regulatory stamp of approval to proceed last year. In that trial, scientists plan to genetically alter patients immune cells to attack three different kinds of cancer.

Clearly, a race to cure cancer with CRISPR is underfoot. And right now at least, China seems to be winning.

[Wall Street Journal]

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China Is Racing Ahead of the US in the Quest to Cure Cancer With CRISPR - Gizmodo

Irish cardiologists pioneer new treatment for heart patients – Irish Times

Irish cardiologists have found a way to repair damaged cardiac muscle and reduce the risk of future heart failure by injecting a growth promoter into the hearts of heart attack sufferers. Photograph: Getty Images

A team of Irish cardiologists have shown that injecting an insulin-like growth promoter into the hearts of patients who have suffered a severe heart attack can repair damaged cardiac muscle and reduce the risk of future heart failure.

Prof Noel Caplice, Chair of Cardiovascular Sciences at University College Cork, and his cardiologist colleagues at Cork University Hospital successfully tested the growth factor in a clinical trial involving 47 patients who presented at the Cork hospital after experiencing heart attacks.

Prof Caplice said 20 per cent of people who suffer heart attacks have severe ongoing difficulties because of lasting damage to heart muscle even after the best current therapies.

After you have a heart attack, regardless whether you treat it with a stent or whatever, about 20 per cent of patients go on to have poor remodelling heart muscle cells die, you get scar tissue forming and the heart tends to expand and dilates, a bit like a balloon, and you get thinned-out heart muscle.

With that poor remodelling of the heart, the heart as a structure performs much worse, it doesnt work very well in terms of its function that leads to a substantial number of those patients going on to suffer heart failure with an increased risk of death, he said.

However, 10 years ago, Prof Caplice and his team began looking at using stem cells as a means of repairing damaged tissue and they found a protein within the stem cells, IGF 1, previously used to treat congenital dwarfism and growth problems, was leading to the repair of damaged heart muscle.

IGF 1 acts differently to insulin in that it acts on a different receptor in the body and when we inject it, it gets into the heart tissue and it basically stimulates receptors on the surface of the cardiac cells and in about 30 minutes, it sends a survival signal to the heart muscles cells, he said.

What we discovered from the stem cell study was that the concentration of the factor was extremely low so what we did was that we took the purified factor and in studies with pigs we injected them in the context of a heart attack and we found these major remodelling benefits.

Those animal tests were funded by Science Foundation Ireland but four years ago the Health Research Board came on board and the two bodies provided a 1 million grant to allow the treatment be trialled on humans.

Working with a 25-strong team incorporating cardiologists, radiologists, MRI specialists and nurses, Prof Caplice was able to incorporate the IGF 1 trials into the treatment of patients attending CUH with severe cardiac events and over the past three years have trialled it on 47 patients.

Patients received two different low-dose preparations of insulin-like growth factor or placebo in a randomised double-blinded clinical trial, with results showing those who received the higher dose had improved remodelling of their heart muscle in the two-month follow-up after their heart attack.

Prof Caplice said the CUH trials, the results of which he will present at a European Society of Cardiology conference in Paris on Saturday, were the first use of IGF 1 in human hearts and part of its attractiveness was its low dosage ensuring minimal side effects while improving cardiac structure.

Among the beneficiaries was John Nolan from New Ross who suffered a heart attack in December 2014. I feel I was blessed to be asked to be involved; I had confidence that good would come from it, in terms of how they explained it to me. Looking back on it now, I feel it was the right choice.

For Prof Caplice, the challenge now is to expand the trials to several hundred patients possibly across different countries and different healthcare systems to see if the IGF 1 treatment is globally applicable which, if proven to be the case, could lead to regulatory approval within five years.

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Irish cardiologists pioneer new treatment for heart patients - Irish Times

City of Hope brings together bone marrow transplant recipients and donors for first time – The San Gabriel Valley Tribune

DUARTE >> Evan Braggs, a 32-year-old from Rancho Cucamonga, could not contain his emotions Friday when, for the first time, he met the man who saved his life more than a decade ago.

Its a humbling experience, Braggs said of meeting and embracing his bone marrow donor, Mike Cook, a 49-year-old Marine from Virginia. Braggs and Cook were one of two pairs of donors and recipients meeting for the first time at City of Hopes 41st annual Bone Marrow Transplant Reunion at the hospitals Duarte campus.

Its overwhelming, said Cook. To think that just me saying yes to what I thought was a little thing, turned into such a big thing. I want to thank you (Evan), because you make me a better person.

Sergio Ramirez, 34, of Los Angeles also got to meet and thank in person his donor, Michael Palacios, a 27-year-old from Silver Lake.

My sons were also excited to meet him, especially my youngest who wrote him a poem, and they all have thank-you cards for him, Ramirez said.

Youre a blessing, he said to Palacios.

The yearly event celebrates the work of City of Hope doctors and staff, as well as the success of their transplant program, which has performed more than 13,000 bone marrow, cord blood and stem cell transplants.

More than 4,000 City of Hope transplant recipients, donors, their families and others reunited Friday and were treated to a picnic-style gathering at the campus.

Braggs was a strong young athlete competing in hurdles at Mt. San Antonio College when he was diagnosed with aplastic anemia. The disease prevents a persons blood marrow from making an adequate amount of new blood cells, and can eventually lead to severe heart problems.

The diagnosis was a shock; hed never so much as broken a bone in his life. After several treatments to try to boost his bone marrow production, as well as weekly blood transfusions, doctors determined he needed a bone marrow transplant.

A match was made and Braggs underwent the transplant operation in 2005, while he was on summer break.

Braggs wife, Melina Fregoso, is also a cancer survivor. The couple rode on the City of Hope Rose Parade float together in 2015. She said meeting her husbands donor was an emotional experience.

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Im grateful that I got to meet him today, and to give thanks, Braggs said.

Cook, after serving in the Marines for more than two decades, is now a reverend in charge of the mens ministry at Shiloh New Site Baptist Church in Virginia. He said meeting his recipient for the first time Friday allowed him to understand what his donation truly meant.

Cook was stationed at Marine Corps Base Quantico in Virginia when he attended a blood drive and was offered to register for bone marrow donation. When he signed up, Cook thought of his nephew, who was diagnosed with a brain tumor when he was just a toddler.

I thought, if I ever have the chance to save someone, I would, he said.

Just like Cook, the act of giving came naturally to Palacios. He was a volunteer at Childrens Hospital Los Angeles when representatives from Be The Match registry visited. He signed up as soon as he could.

Its just something I do, Palacios said. We dont feel like heroes. If were in a position to help, were happy to do it.

Sergio Ramirez was diagnosed with acute lymphoblastic leukemia. Even after three years of treatment, the disease returned with even greater strength.

Knowing the chances of survival for all patients after relapsing were slim, Ramirez was more concerned with what would happen to his family his wife and three sons.

Ramirez took part in an immunotherapy trial at City of Hope. He responded well to the treatment, and he went into remission. But a bone marrow transplant would be the only thing to prevent the cancer from returning.

Its an amazing feeling every morning I wake up, to hear my kids, to see the sunshine, Ramirez said.

Palacios said he will follow up to make sure he is still on the donor registration list with the hopes that he could have the chance to save another life in the future.

I want to continue to inspire others to donate, he said. After meeting (Ramirez), he can finally say he doesnt have to worry about cancer, and be with his family; that makes me happy and gives me hope.

For more information about City of Hope and bone marrow donor registry, go to http://www.cityofhope.org.

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City of Hope brings together bone marrow transplant recipients and donors for first time - The San Gabriel Valley Tribune

The irrefutable success of stem cell treatments Miami’s Community … – Miami’s Community Newspapers

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In my opinion, one of the hardest things to accept is a new type of medical treatment, particularly when it changes the philosophy, parameters and overall results that we are expecting and basically used to receiving. Stem cells are undoubtedly no exception to this rule.

About six weeks ago, Eduardo K (a Cuban doctor with a Masters Degree from the University of Pittsburg in internal medicine and nephrology), brought his wife Maria to our institute, in order to assess the possibility of using stem cells to cure the severe chronic pain in her ankle; a pain so severe that it was basically hindering her ability to walk. Dr. K also expressed his extreme hesitation and concerns about having his wife involved in an invasive ankle surgery at this stage of her adult life.

However, while conducting our usual examining process, reviewing her medical records and MRIs and thoroughly discussing my overall recommendations about a potential stem cell transplant, I quickly realized that Dr. K was not a true believer in Stem Cell therapies, since he thought that there was not much medical evidence of their actual effectiveness and he ultimately also confessed that his wife had basically dragged him to accompany her to this particular appointment.

As always, I respectfully explained the reality that stem cells actually repair the damaged cartilage in a microscopic type fashion and thus, while this repair process would not be clearly reflected immediately on future X-rays, I assured them that the pain she was suffering from will soon subside and possibly even completely disappear. In addition, I expressed that I was extremely confident that she would also regain her mobility skills after the procedure, even if this improvement could not be easily detected via a radiological image.

Since Marias options were somewhat limited, added to the fact that months of traditional physical therapy, injections, medications and previous surgeries had completely failed her, Dr. K finally agreed to grant his wifes wishes to have her stem cell transplant (from her own bone marrow and fat) performed, although he was still very skeptical about the process and was showing little enthusiasm.

This morning, both of them attended our follow up appointment (six weeks after the procedure) and surprisingly, Maria and Dr. K happily confirmed that she felt at least 60 percent better, something that no previous traditional medical treatments had been able to accomplish. It was then that I explained to them that her stem cells had acted much faster than expected (something that possibly taught Dr. K an interesting lesson).

As we began to say our goodbyes, the doctor told me (first in English, then in Spanish) that: in spite of my skepticism about stem cell therapies, I can personally attest that the successful results seen on my wife have been irrefutable, and with a smile on both of their faces, they gratefully thanked my staff and I for this amazing improvement.

As I continued to replay the words expressed by this doctor over and over in my mind, I quickly realized how truly incredulous human beings tend to be, with most of us often needing to fail several times at accomplishing something before being able to realize and accept that we were truly mistaken in the first place!

So if you, a friend or relative would like to receive Stem Cell or PRP treatments, please call us at 305-598-7777. For information visit: http://www.stemcellmia.com (available in both English and Spanish), or watch our amazing video-testimonies on our YouTube Chanel and also please follow us on Facebook and Twitter. If you would like to ask a question directly to Dr. Castellanos, please do so via his direct email: stemdoc305@gmail.com.

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The irrefutable success of stem cell treatments Miami's Community ... - Miami's Community Newspapers

Bone marrow donor forgot he’d registered – Jewish Chronicle


Jewish Chronicle
Bone marrow donor forgot he'd registered
Jewish Chronicle
My phone rang and when I answered they said someone needed my stem cells. They asked me would I still like to donate? I went in the next day for tests and when I was deemed fit and healthy they got me to come back in for the procedure. On Tuesday the ...

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Bone marrow donor forgot he'd registered - Jewish Chronicle

Fighting arthritis: Researchers edit stem cells to fight inflammation – Kasmir Monitor

In a major development, researchers have developed a cartilage that fights inflammation caused by arthritis and other chronic conditions, using the gene-editing technique called CRISPR. For the breakthrough, researchers at Washington University School of Medicine converted skin cells from the tails of mice into stem cells. They then used the gene-editing tool CRISPR to remove a gene involved in inflammation and replace it with one that produces anti-inflammatory drug. They called the resulting cells as SMART cells, which stands for Stem cells Modified for Autonomous Regenerative Therapy. "Our goal is to package the rewired stem cells as a vaccine for arthritis, which would deliver an anti-inflammatory drug to an arthritic joint but only when it is needed," said Farshid Guilak, Professor at Washington University School of Medicine, and senior author of a study published online in the journal Stem Cell Reports. "To do this, we needed to create a 'smart' cell," Guilak said. According to the study, SMART cells, develop into cartilage cells that produce a biologic anti-inflammatory drug that could replace arthritic cartilage and simultaneously protect joints and other tissues from damage that occurs with chronic inflammation. Many current drugs used to treat arthritis attack an inflammation-promoting molecule called tumour necrosis factor-alpha (TNF-alpha). But the problem with these drugs is that they are given systemically rather than targeted to joints. As a result, they interfere with the immune system throughout the body and can make patients susceptible to side effects such as infections. "We want to use our gene-editing technology as a way to deliver targeted therapy in response to localised inflammation in a joint, as opposed to current drug therapies that can interfere with the inflammatory response through the entire body," Guilak said. The research has been published in the journal Stem Cell Reports.

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Fighting arthritis: Researchers edit stem cells to fight inflammation - Kasmir Monitor

CRISPR-SMART Cells Regenerate Cartilage, Secrete Anti-Arthritis Drug – Genetic Engineering & Biotechnology News

We have anti-arthritis drugs. What we lack is the ability to deploy them when and where they are needed in the body. The drugs would be far more effective, and occasion fewer side effects, if they were to appear only in response to inflammation, and only in the joints. If the drugs could be delivered so painstakinglyso smartlythey wouldnt have to be administered systemically.

Although conventional drug delivery systems may be unable to respond to arthritic flares with such adroitness, cells may have better luckif they are suitably modified. Stem cells, for example, have been rewired by means of gene-editing technology to fight arthritis. These stem cells, known as SMART cells (Stem cells Modified for Autonomous Regenerative Therapy), develop into cartilage cells that produce a biologic anti-inflammatory drug. Ideally, the new cartilage cells will replace arthritic cartilage, and the biologic will protect against chronic inflammation, preserving joints and other tissues.

SMART cells of this sort were prepared by scientists based at Washington University School of Medicine in St. Louis. The scientists initially worked with skin cells taken from the tails of mice and converted those cells into stem cells. Then, using the gene-editing tool CRISPR in cells grown in culture, they removed a key gene in the inflammatory process and replaced it with a gene that releases a biologic drug that combats inflammation.

Details of this work appeared April 27 in the journal Stem Cell Reports, in an article entitled Genome Engineering of Stem Cells for Autonomously Regulated, Closed-Loop Delivery of Biologic Drugs. The article describes how modified stem cells grew into cartilage and produced cartilage tissue. The engineered cartilage, the scientists reported, was protected from inflammation.

Using the CRISPR/Cas9 genome-engineering system, we created stem cells that antagonize IL-1- [interleukin-1] or TNF-- [tumor necrosis factor-] mediated inflammation in an autoregulated, feedback-controlled manner, wrote the authors of the Stem Cell Reports article. Our results show that genome engineering can be used successfully to rewire endogenous cell circuits to allow for prescribed input/output relationships between inflammatory mediators and their antagonists, providing a foundation for cell-based drug delivery or cell-based vaccines via a rapidly responsive, autoregulated system.

Many current drugs used to treat arthritisincluding Enbrel (etanercept), Humira (adalimumab), and Remicade (infliximab)attack TNF-, an inflammation-promoting molecule. But the problem with these drugs is that they are given systemically rather than targeted to joints. As a result, they interfere with the immune system throughout the body and can make patients susceptible to side effects such as infections.

"We want to use our gene-editing technology as a way to deliver targeted therapy in response to localized inflammation in a joint, as opposed to current drug therapies that can interfere with the inflammatory response through the entire body," said Farshid Guilak, Ph.D., the paper's senior author and a professor of orthopedic surgery at Washington University School of Medicine. "If this strategy proves to be successful, the engineered cells only would block inflammation when inflammatory signals are released, such as during an arthritic flare in that joint."

Dr. Guilak's team encoded the stem/cartilage cells with genes that made the cells light up when responding to inflammation, so the scientists easily could determine when the cells were responding. Recently, the team began testing the engineered stem cells in mouse models of rheumatoid arthritis and other inflammatory diseases.

If the work can be replicated in animals and then developed into a clinical therapy, the engineered cells or cartilage grown from stem cells would respond to inflammation by releasing a biologic drugthe TNF- inhibitorthat would protect the synthetic cartilage cells that Dr. Guilak's team created and the natural cartilage cells in specific joints.

"When these cells see TNF-, they rapidly activate a therapy that reduces inflammation," Dr. Guilak explained. "We believe this strategy also may work for other systems that depend on a feedback loop. In diabetes, for example, it's possible we could make stem cells that would sense glucose and turn on insulin in response. We are using pluripotent stem cells, so we can make them into any cell type, and with CRISPR, we can remove or insert genes that have the potential to treat many types of disorders."

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Another CRISPR Trial Begins – GenomeWeb


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Another CRISPR Trial Begins
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Researchers in China have embarked on a new trial using the gene-editing tool CRISPR/Cas9 to modify human genes to treat cancer patients, the Wall Street Journal reports. For this trial, researchers at Nanjing University have injected the first set of ...
China Is Racing Ahead of the US in the Quest to Cure Cancer With CRISPRGizmodo
CRISPR Used To Modify Multiple Genes In Rice | Asian Scientist ...Asian Scientist Magazine
Insider Selling: Crispr Therapeutics AG (CRSP) Insider Sells ...Transcript Daily
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Out of his mind surgeon plans human head transplant, revival of frozen brain – Ars Technica

Italian neurosurgeon Sergio Canavero will undertake the first human head transplant later this year in China, the doctor told German magazineOoomin an article published Thursday. And, following that effort, he will revive a cryogenically frozen brain and transplant it into a donor body within the next three years.

The plans, completely disconnected from reality and the state of modern medicine, are at least in line with his previous outlandish goals and dubious animal research.

Canavero made headlines in the past few years by claiming that transplanting the whole head of a human onto a donor body is currently possible. A Russian man, suffering from a spinal muscular atrophy malady called Werdnig-Hoffmann Disease, even publicly volunteered for the procedure.

As proof that the transplant could work, Canavero published gruesome experiments in 2016, said to have repaired the severely injured spinal cords of mice, rats, and a dog. The experiments came complete with cringe-worthy video of recovering animals struggling to drag their limp bodies around. Yet, the study lacked controls, detailed methods, and data on the injuries and recoveries. Canavero claimed to perform a head transplant on a monkey but did not publish the experiment.

Mouse limping after experimental spinal cord repair.

Sergio Canavero giving a TEDX talk.

Sergio Canavero with his Chinese partner, Dr. Xiaoping Ren, who will lead the operation team onsite during an attempted head transplant procedure.

Experts decidedly consider his research on spinal cord repair, let alone whole head transplants, unconvincing. A medical ethicist dubbed Canavero out of his mind for sweeping past the currently insurmountable challenges of such feats. These include intricately repairing and reattaching thousands of delicate nerves and restoring function. Right now, doctors cant even convince the immune system to accept far simpler transplants consistently. Theres also the completely unknown effects of such a transplant on the powerful human psyche.

Canavero is carrying on, undeterred it seems. In his Ooom interview, he not only glided through the idea of successfully transplanting a head, he made an even more absurd claim: that he would revive a cryogenically frozen brain and transplant it into a donor body. Canavero said he would obtain a preserved brain from Alcor Life Extension Foundation, a cryonics company located in Scottsdale, Arizona, according to Gizmodo.

There is currently no way to revive and molecularly repair a frozen human brain. And such transplants havent even been attempted in animals. Thus, the surgical procedure is decades if not centuries away.

As Gizmodo also reports, Alcor said that Canavera hadnt even contacted the company. It distanced itself from the doctor, as did other cryonics leaders, and noted that his efforts are not realistic or even a shared goal.

In a statement, the company said:

The Alcor Life Extension has had no contact with Dr. Canavero. It is not yet possible to revive human brains cryopreserved with present methods. Revival of todays cryonics patients will require future repair by highly advanced future technology, such as molecular nanotechnology. Technology that is advanced enough to repair a cryopreserved brain would by its nature also be able to regrow new tissues, organs, and a healthy body for the revived person. Therefore Alcor does not expect body donations or transplants to ever be necessary for revival of cryonics patients. Until advanced tissue regeneration technology is developed, we wish Dr. Canavero well in his development of body transplant surgery for living patients today who might benefit.

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Out of his mind surgeon plans human head transplant, revival of frozen brain - Ars Technica

Regenxbio Inc (RGNX) Stock Rating Upgraded by Zacks Investment Research – The Cerbat Gem


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Regenxbio Inc (RGNX) Stock Rating Upgraded by Zacks Investment Research
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The Company focuses on the development, commercialization and licensing of recombinant adeno-associated virus gene therapy. Its products candidates include RGX-501, for the treatment of homozygous familial hypercholesterolemia which uses the AAV8 ...
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$10 million settlement over alleged misconduct in Boston heart stem cell lab – Science Magazine

Brigham and Women's Hospital in Boston.

BRIAN SNYDER/REUTERS/Newscom

By Kelly ServickApr. 27, 2017 , 5:00 PM

A research misconduct investigation of a prominent stem cell lab by the Harvard Universityaffiliated Brigham and Womens Hospital (BWH) in Boston has led to a massive settlement with the U.S. government over allegations of fraudulently obtained federal grants. As Retraction Watch reports, BWH and its parent health care system have agreed to pay $10 million to resolve allegations that former BWH cardiac stem cell scientist Piero Anversa and former lab members Annarosa Leri and Jan Kajstura relied on manipulated and fabricated data in grant applications submitted to the U.S. National Institutes of Health (NIH).

A statement from the U.S. Attorneys Office for the District of Massachusetts released today notes that it was BWH itself that shared the allegations against Anversas lab with the government. The hospital had been conducting its own probe into the Anversa lab since at least 2014, when a retraction published in the journal Circulation revealed the ongoing investigation. The hospital has not yet released any findings.

In 2014, Anversa and Leri sued Harvard and BWHalong with BWH President Elizabeth Nabel and Gretchen Brodnicki, Harvards dean for faculty and research integrityfor launching and publicizing the investigation that they claimed wrongfully damaged their careers. In their complaint, they acknowledged fabricated data in the Circulation paper and altered figures in a 2011 paper for whichThe Lancethas published an expression of concern. But they claimed that Kajstura had altered data without their knowledge. (Anversa and Leris recent papers list their institution as Swiss Institute for Regenerative Medicine, Retraction Watch notes.)

In July 2015, a federal district court judge dismissed the lawsuit, ruling that the plaintiffs had to first air their grievances with the federal Office of Research Integrity, which handles misconduct investigations at NIH-funded labs.

Grant fraud cases against universities rarely involve research misconduct, and most are brought by whistleblowers who stand to claim a share of any returned funds. Despite the high penalty, BWH gets praise from the Department of Justice in todays announcement for self-disclosing the allegations and for taking steps to prevent future recurrences of such conduct.

But the result is confusing and potentially discouraging, says Ferric Fang, a microbiologist at the University of Washington in Seattle, who has published several analyses of retractions, misconduct, and the scientific enterprise. It sounds as if the researchers themselves were found to have engaged in improper practices, but the institution is on the hook for the settlement. The decision deserves greater clarification, he says, or it could discourage other institutions from being as forthcoming in the future.

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$10 million settlement over alleged misconduct in Boston heart stem cell lab - Science Magazine

3D Printed Patches seeded with cells to repair cardiac tissue after heart attacks – BSA bureau (press release)

The patches may be effective at helping to restore the heart following a myocardial infarction, as the heart isnt able to restore lost cells on its own. The patches may be effective at helping to restore the heart following a myocardial infarction, as the heart isnt able to restore lost cells on its own.

A team of researchers from University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham have developed a technique for 3D printing cardiac patches seeded with living cells. The patches may be effective at helping to restore the heart following a myocardial infarction, as the heart isnt able to restore lost cells on its own.

The technology has already been tested on a mouse model following an induced heart attack in which cardiac function wa significantly improved in four weeks following the application of the patch.

The patch is structurally based on how proteins naturally assemble within cardiac tissue. A highly detailed technique called multiphoton-excited 3D printing was used to create an extracellular matrix that was then seeded with about 50,000 cardiomyocytes, smooth muscle cells, and endothelial cells obtained from human-induced pluripotent stem cells.

The patch began beating on its own only a day after placing the cells and calcium transients, which are intercellular signaling mechanisms, were detected and increased over the following week.

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3D Printed Patches seeded with cells to repair cardiac tissue after heart attacks - BSA bureau (press release)

VistaGen Therapeutics Announces Peer-Reviewed Publication in … – Yahoo Finance

SOUTH SAN FRANCISCO, CA--(Marketwired - April 27, 2017) - VistaGen Therapeutics Inc. (VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today the peer-reviewed publication of nonclinical studies of the effects of AV-101 (4-Cl-KYN), its CNS prodrug candidate, in four well-established nonclinical models of pain.

The publication, titled: "Characterization of the effects of L-4-chlorokynurenine on nociception in rodents," by lead author, Tony L. Yaksh, Ph.D., and co-authors, Robert Schwarcz, Ph.D. and H. Ralph Snodgrass, Ph.D., was recently published in The Journal of Pain (DOI: 10.1016/j.jpain.2017.03.014) and is available online at http://www.jpain.org/article/S1526-5900(17)30552-7/abstract.

"In these studies, AV-101 was found to have robust anti-nociceptive effects, similar to gabapentin, but with a better side effect profile in several pre-clinical models of hyperalgesia and allodynia, results suggest AV-101's potential for treating multiple hyperpathic pain states," reported Tony L. Yaksh, Ph.D., Professor in Anesthesiology at the University of California, San Diego (UCSD).

"In comparison to gabapentin and other agents commonly used by millions of patients battling chronic neuropathic pain, we believe AV-101 has the potential to reduce debilitating pain effectively without causing burdensome side effects. Many neuropathic pain treatments on the market today have side effects, including anxiety, depression, mild cognitive impairment and sedation. The positive results published in these studies fall in line with our goal of advancing Phase 2 clinical development of AV-101 across a broad range of CNS indications, including major depressive disorder, neuropathic pain and L-DOPA-induced dyskinesia associated with Parkinson's disease. We are optimistic that we will be able to bring to market a new generation CNS medication that would help millions of patients currently treated with therapies with inadequate efficacy and significant side effects and safety concerns," stated H. Ralph Snodgrass, Ph.D., VistaGen's President and Chief Scientific Officer.

Study Summary and Key Findings:

About AV-101

AV-101 (4-CI-KYN) is an oral CNS prodrug candidate in Phase 2 development in the U.S. as a new generation treatment for major depressive disorder (MDD). AV-101 also has broad potential utility in several other CNS disorders, including chronic neuropathic pain and epilepsy, as well as addressing symptoms associated with neurodegenerative diseases, such as Parkinson's disease and Huntington's disease.

AV-101 is currently being evaluated in a Phase 2 monotherapy study in MDD, a study being fully funded by the U.S. National Institute of Mental Health (NIMH) and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH, as Principal Investigator.

VistaGen is preparing to advance AV-101 into a 180-patient, U.S. multi-center, Phase 2 adjunctive treatment study in MDD patients with an inadequate response to standard FDA-approved antidepressants, with Dr. Maurizio Fava of Harvard University as Principal Investigator.

About VistaGen

VistaGen Therapeutics, Inc. (VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen's lead CNS product candidate, AV-101, is in Phase 2 development as a new generation oral antidepressant drug candidate for major depressive disorder (MDD). AV-101's mechanism of action is fundamentally differentiated from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with inadequate response to standard, FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Company's Phase 2 adjunctive treatment study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including chronic neuropathic pain, epilepsy, and symptoms of Parkinson's disease and Huntington's disease, where modulation of the NMDAR, AMPA pathway and/or key active metabolites of AV-101 may achieve therapeutic benefit.

Read More

VistaStem Therapeutics is VistaGen's wholly owned subsidiary focused on applying human pluripotent stem cell technology, internally and with collaborators, to discover, rescue, develop and commercialize proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases, and cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. In December 2016, VistaGen exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease.

For more information, please visit http://www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook.

Forward-Looking Statements

The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH's Phase 2 (monotherapy) and/or the Company's planned Phase 2 (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, including neuropathic pain and L-DOPA-induced dyskinesia associated with Parkinson's disease, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the Phase 2 clinical development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Therapeutics Announces Peer-Reviewed Publication in ... - Yahoo Finance

Transparent Bones Enable Researchers to Observe Stem Cells Inside – Laboratory Equipment

Ten years ago, the bones currently in your body did not actually exist. Like skin, bone is constantly renewing itself, shedding old tissue and growing it anew from stem cells in the bone marrow. Now, a new technique developed at Caltech can render intact bones transparent, allowing researchers to observe these stem cells within their environment. The method is a breakthrough for testing new drugs to combat diseases like osteoporosis.

The research was done in the laboratory of Viviana Gradinaru (BS '05), assistant professor of biology and biological engineering and a Heritage Medical Research Institute Investigator. It appears in a paper in the April 26 issue of Science Translational Medicine.

In healthy bone, a delicate balance exists between the cells that build bone mass and the cells that break down old bone in a continual remodeling cycle. This process is partially controlled by stem cells in bone marrow, called osteoprogenitors, that develop into osteoblasts or osteocytes, which regulate and maintain the skeleton. To better understand diseases like osteoporosis, which occurs when loss of bone mass leads to a high risk of fractures, it is crucial to study the behavior of stem cells in bone marrow. However, this population is rare and not distributed uniformly throughout the bone.

"Because of the sparsity of the stem cell population in the bone, it is challenging to extrapolate their numbers and positions from just a few slices of bone," says Alon Greenbaum, postdoctoral scholar in biology and biological engineering and co-first author on the paper. "Additionally, slicing into bone causes deterioration and loses the complex and three-dimensional environment of the stem cell inside the bone. So there is a need to see inside intact tissue."

To do this, the team built upon a technique called CLARITY, originally developed for clearing brain tissue during Gradinaru's postgraduate work at Stanford University. CLARITY renders soft tissues, such as brain, transparent by removing opaque molecules called lipids from cells while also providing structural support by an infusion of a clear hydrogel mesh. Gradinaru's group at Caltech later expanded the method to make all of the soft tissue in a mouse's body transparent. The team next set out to develop a way to clear hard tissues, like the bone that makes up our skeleton.

In the work described in the new paper, the team began with bones taken from postmortem transgenic mice. These mice were genetically engineered to have their stem cells fluoresce red so that they could be easily imaged. The team examined the femur and tibia, as well as the bones of the vertebral column; each of the samples was about a few centimeters long. First, the researchers removed calcium from the bones: calcium contributes to opacity, and bone tissue has a much higher amount of calcium than soft tissues. Next, because lipids also provide tissues with structure, the team infused the bone with a hydrogel that locked cellular components like proteins and nucleic acids into place and preserved the architecture of the samples. Finally, a gentle detergent was flowed throughout the bone to wash away the lipids, leaving the bone transparent to the eye. For imaging the cleared bones, the team built a custom light- sheet microscope for fast and high-resolution visualization that would not damage the fluorescent signal. The cleared bones revealed a constellation of red fluorescing stem cells inside.

The group collaborated with researchers at the biotechnology company Amgen to use the method, named Bone CLARITY, to test a new drug developed for treating osteoporosis, which affects millions of Americans per year.

"Our collaborators at Amgen sent us a new therapeutic that increases bone mass," says Ken Chan, graduate student and co-first author of the paper. "However, the effect of these therapeutics on the stem cell population was unclear. We reasoned that they might be increasing the proliferation of stem cells." To test this, the researchers gave one group of mice the treatment and, using Bone CLARITY, compared their vertebral columns with bones from a control group of animals that did not get the drug. "We saw that indeed there was an increase in stem cells with this drug," he says. "Monitoring stem cell responses to these kinds of drugs is crucial because early increases in proliferation are expected while new bone is being built, but long-term proliferation can lead to cancer."

The technique has promising applications for understanding how bones interact with the rest of the body.

"Biologists are beginning to discover that bones are not just structural supports," says Gradinaru, who also serves as the director of the Center for Molecular and Cellular Neuroscience at the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech. "For example, hormones from bone send the brain signals to regulate appetite, and studying the interface between the skull and the brain is a vital part of neuroscience. It is our hope that Bone CLARITY will help break new ground in understanding the inner workings of these important organs."

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Transparent Bones Enable Researchers to Observe Stem Cells Inside - Laboratory Equipment

Groundbreaking stem cell study kicks off in Northwest Florida – WEAR

A local stem cell study is changing the future of orthopedics.

A new study taking place at the Andrews Institute in Northwest Florida could shape the future of orthopedic surgery.

The goal of the study, spearheaded by Dr. Adam Anz and already eight years in the making, is to use stem cells to regrow cartilage.

If approved, it will be the first orthopedics study of its kind done in the United States and only the second in the entire world.

Stem cells are currently utilized most in cancer research and treatments, but Dr. Anz of the Andrews Institute wants to change that by putting regenerative medicine to the test, using stem cells to regrow knee cartilage.

The Andrews Institute already uses stem cells in certain therapies, but this new method could be a game changer.

"The bone marrow aspirate, which we're studying for knee arthritis and we can offer to patients, is the 1990's technology of stem cells," Dr. Anz said. "What we're studying is the modern way to harvest many more stem cells. That's the reason the FDA has said you need to bring this through our process before you just offer it to people."

Through a process called apheresis, stem cells are harvested from the patient with help from a synthetic hormone that promotes the body to generate more stem cells.

"Through this process we can collect millions of cells," Dr. Anz said. "Just 140 milliliters -- about a half of a coke can -- will have 140 million stem cells."

The stem cells will then be sorted, divided and injected into the patient's knee. Excess cells are stored in a nitrogen freezer at negative 181 degrees Celsius until the next round of injections, a process to be repeated over the next two years.

"If this study is successful, this will be the first approved in orthopedics in the United States," said Dr. Anz.

The study begins in May. Dr. Anz believes it will take about another five to seven years before the FDA can approve it for use in patients.

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Groundbreaking stem cell study kicks off in Northwest Florida - WEAR

Scientists turn bones transparent to let them see into marrow – Stat – STAT

N

ow you see it, now you dont: Scientists have used a chemical technique to make mouse bones turn transparent. The technique has been used in the past to make brains and kidneyssee-through, but this marks the first time its been used in hard tissues.

The ability to see within a bone couldhave implications for research into bone diseases, by letting researchers get a more accurate picture of bones internal structure.

The technique is called CLARITY, and since 2013, when it was first described, it has been deployed on a wide variety of mammalian tissues and inplants. Caltech neuroscientistViviana Gradinaru, an original developer of the technique, even cleared an entire mouses body in 2014 (except for its bones, which were unaffected, she said).

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The approach works by chemically locking proteins and DNA in place with a hydrogel, after which researchers wash away fats within the tissue. Lipids refract light, so this washing step makes CLARITY-treated tissues transparent.

Flexible 3-D printed scaffolds could mend broken bones

In this case, Gradinaruwanted to look at bone marrow and count the number of stem cells that could ultimately produce new bone cells.

Bone is not a static organ. It iscontinuously changed. The bones we have in our body, we didnt have them 10 years ago, she explained. Acontinuous process of bone cell death and bone cell growth ishappening, spurred by progenitor cells in a bones soft, spongy marrow.

But looking for these cells can bechallenging. There arent that many progenitor cells, soextrapolating the number and distribution based ona small sample isnt ideal. Researchers can slice the bone, but cuttingcan damage the edges. Putting images of the sliced bones back together into a coherent, 3-D picture is very difficult, too.A clear bone avoidsslicing altogether.

Doug Richardson, director of imaging at the Harvard Center for Biological Imaging, said the paper represented a step forward in bone clearing. (Richardson was not involved in this research.)

This technique has the potential to monitor bone health or disease progression over larger volumes with greater accuracy, he said.

Gradinarus team has already demonstrated one possible application. They found that a drug for osteoporosis, currently being developed by Amgen, triggered an increase in the number of stem cells in CLARITY-treated bone.Some Amgen scientists were coauthors of the paper.

Using CLARITY let the team more effectively measure the rate of this increase.This is very important, because you want a controlled increase too much of an increase can lead to tumors, Gradinarusaid.

Other uses could be on the horizon. Being able to make a mouse or rat skull see-through could be useful for Gradinarus fellow neuroscientists who use implants in their research and want to establish the exact position of the impact after experiments are done.

Theres still more work to be done. For instance, finding a way to tagthe samples with antibodies without having to cut a bone in half, as researchers did in this paper would be ideal.Gradinaru also wouldnt mind some speed improvements:In this case, the CLARITY process took nearly a month.

Its not a fast method, by any means, Gradinaru said. However, the result theres no substitute for getting 3-D access to the intact bone marrow.

Kate Sheridan can be reached at kate.sheridan@statnews.com Follow Kate on Twitter @sheridan_kate

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Family seeks ‘miracle’ for siblings, saves lives in the process – Monroe News Star

Caroline, 11, Elizabeth, 3, Jon Thomas, 13 and James Christopher Allums, 20, do everything as a family. James Christopher and Elizabeth both have a rare medical condition. Their mother, Ellen Allums, said they all go through the process together and support each other with faith and love.(Photo: Courtesy)

Learning that your child has a rare, life-threatening illness is difficult for any family. Everything changes. One family learned that two of their children share the same rare blood disorder.

"That news that we heard was the worst news that we could hear, but it was the best thing that's ever happened to us. It really changed our perspective. It changed our priorities," Chris Allums said.

"We're no longer the same people we were," Ellen Allums said.

Ellen and Chrishave four childrenJames Christopher, 20,Jon Thomas, 13,Caroline,11, andElizabeth, 3.

James Christopher and Elizabeth have Fanconi anemia, a disease that affects the bone marrow's ability to produce blood. Bone marrow or blood stem cell transplants are considered the best treatments, andthey have not yet found a match for either child.

James Christopher was diagnosed 12 years ago and told he had about 18 monthsto live. The family was told he must received a bone marrow transplant.

"We immediately decided that, first of all, we're going to pray and expect a miracle and grow our faith, and next, we're going to try and see if we can find him a bone marrow match and help others along the way, see how many lives that we can affect, that we can save both spiritually and physically," Ellen said.

More than 16,000 people have been added to the worldwide bone marrow registry as a result of drives held on behalf of the Allums. Ellen said they know of at least 41 lives that have been saved because of those efforts, and they're asking more people to commit to donate.

'Looking for a double miracle'

Ellen said a doctor said someone with FA can be like a duck gliding on the water the surface appearance is calm, but people can't see all the effort that goes into staying in motion.

It has a variety of symptoms such as fatigueand can lead to bone marrow or organ failure. Ellen and Chris said FA patients are 500 times more likely to develop some cancers, such as leukemia. James Christopher is subject to constant screenings.

The disease is genetic. According to the National Organization for Rare Disorders, the incidence rate is 1 in 136,000 births. Ellen said her children are two ofsix in Louisiana affected by FA.

Elizabeth's blood counts have been OK, but doctors have said James Christopher has an immediate need for a transplant. DNA needs to be close to an exact match, and many families find a relative who can donate.Elizabeth is a 100 percent match, but she's ineligible because of her FA.

One donor, once found, could help both.A bone marrow transplant won't cure someone with FA, but it can help prolong life.

"Just because you're having to wait doesn't mean the miracle's not going to come. We've been waiting 12 years, but we still have faith that that miracle's coming. Just because it hasn't happened doesn't mean it's not going to. The timing needs to be right," Ellen said. "In our lives, we're looking for an even greater miracle because we're looking for a double miracle, with two children."

FA patients can require blood and platelet transfusions, after which they may become dependent and need additional rounds, which would require a bone marrow transplant quickly.

James Christopher received his first blood transfusion three weeks ago.

"Chris gave. His daddy gave blood to him, and we felt like it was his heavenly father and his earthly father that gave him that blood, and now we're praying and believing that he never has to receive it again," Ellen said.

She said they've dealt with some scary bleeding issues "like Niagara Falls," and James Christopher has almost lost his life a few times. His parents call him a survivor, a warrior. He gets up and stays active daily, even with low blood counts that doctors thinkwould cause fatigue.

"I love to prove doctors wrong. If they give me a boundary, I want to cross it, definitely, when it comes to that," he said. He likes to tell people "keep calm and carry on," like the World War II posters.

Every bump, scratch, scrape and bruise for the siblings is noteworthy, and the whole family works to avoid germs. A simple cough or cold could be devastating, so they're all in tune to notice illness.They're very aware of the importance of handwashing and staying home if ill. Chris said during cold and flu season, they often come in, shower and change clothes before interacting with the others.

Ellen said they respect people who choose not to vaccinate, but all of her children have been vaccinated because measles or chicken pox can kill someone with FA.

All the children home school to help prevent illness. When James Christopher was diagnosed, doctors said it could help him live longer. Chris said all four have excelled fromthe one-on-one time, and they've enjoyed getting to know other families inthe Christian Homeschool Association.

The Allums know their lives are different than those of many other families, but they are running their own race.

"I have to tell you that we have a wonderful life. Sure it's full of hard work, but it's wonderful because of what the Lord has done with it," Chris said.

Read more:Mom says prayer pulled her through transplant|Facing the storm: Mother shares unbelievable story|Big brother to the rescue: Man gives sister half of liver|Man saves 10 in life, death

Joy in the journey

The couple did their homework on hospitals that specialize in the disease and settled on Memorial Sloan Kettering Hospital's cancer center in New York. It had the best survival rates, and they've been going for 12 years.

James Christopher's and Elizabeth's immunity is low, the family cannot travel with the general public. They either have to make the almost 20-hour drive or arrange for a private plane. Ellen said they've had to go there, at times, every three to six months.

The whole family travels to medical appointments.

"Although they don't have the disease, they go through it with them," Ellen said of Jon Thomas and Caroline. She said all of her children have gone to hospitals and played with and prayed for children were facing terminal diagnoses. It's been a blessing to them and a ministry to others.

James Christopher said they try to find fun in the journey. Ellen said they do something fun every time they go to the hospital and embrace John 10:10Jesus came that we might have life and have it abundantly.

James Christopher Allums, 20, holds his sister Elizabeth Allums, 3. The siblings both have a rare medical condition called Fanconi anemia.(Photo: Courtesy)

What happens if there's a match?

"We would be moving to New York for six to eight months for the bone marrow transplant," Ellen said.

Ellen said the a bone marrow recipient with FA will have to go through chemotheraphy for two weeks to kill off the patient's natural bone marrow.

"When the cells are dead, then they receive someone else's bone marrow. It's a liquid, it looks just like an IV, and they lie there and you just pray to God that it's going to take," she said.

After the transplant, the patient is in isolation for 30-40 days. They stay at the transplant hospital for six to eight months and keep a medical mask on for one year. Chris said you hope graph vs. host disease isn't an issue.

Saving lives

She said she used to look at missions that dig wells in other countries and wish they could go save lives, but, after prayer, she realized they are saving people. With the help of family and friends, efforts to add bone marrow donors have helped dozens of people.

"I like to tell people 'You could be the reason someone lives.' ... And I think those words are pretty powerful" Ellen said.

She said the process to donate blood stem cells, which is the most common donation method, involves a needle in each arm for four to six hours.

"It's not even a surgery. It's not like giving a kidney or a lung or a heart, even, but the benefits are that strong. It can truly save a life, but yet all you have to do is like giving blood," Ellen said.

To test for a match, she said, it's even less of a commitment. It takes about five minutes to fill out paperwork and provide a swap from inside the cheek. Anyone 18-55 in good health can register.

The community has come together to help organize a drive for May 1, National Fanconi Anemia Day. A massive drive will take place at more than a dozen locations across northeastern Louisiana, and CenturyLink will be registering employees on-site.Anyone anywhere can order testing kitsonline atdkms.orgorbethematch.org.

A month after testing, people will get a phone call to confirm their position on the registry. Ellen said they pray people will make the commitment.Previous drives for the Allumshave set national records for most registered in one day. Over three days, they tested 5,000 people.

"When people come, we want to educate them on the processin hopes thatwhetherthey are a match in a month or a match in 20 yearsthat they will be committed to beingon that registry to help somebody," Chris said.

They heard of a woman who registered with her family at a previous event andlater developed leukemia. Her sister was found as an instant bone marrow match because theyalreadyhad been tested.

Ellen and Chris said knowing that 41 lives were saved as a result of their family'sefforts makes it all worth it, even though it hasn't been easy.

"But we believe that God is going to heal them both because He told us He would, and we believe that. We hold on to those promises of God. ... and we focus on that. That gives us strength," Ellen said.

Follow Bonnie Bolden on Twitter@Bonnie_Bolden_and on Facebook athttp://on.fb.me/1RtsEEP.

Want to register?

May 1 is National Fanconi Anemia Day, and a more than a dozen locations across northeastern Louisiana will be part of a single registration drive. Times vary and new locations may be added. Check The Friends of James Christopher and Elizabeth Allumson Facebook or visitcaringbridge.organd searchJames Christopher Allums.

Or order a testing kit online at dkms.org or bethematch.org.

Testing sites and times are:

Monroe

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Family seeks 'miracle' for siblings, saves lives in the process - Monroe News Star

5 Stem Cell Innovations From The Past Year, From Cancer Treatment To Diabetes Therapy – Medical Daily

Ten years ago, the topic of stem cells was shrouded in mystery, but now they're at the forefront of some of the latest innovations in biologyand medicine. Stem cellshave yet to change into a specific type of cell, such as a brain or skin cell. As a result, doctors can manipulate them into, well, any type of cellthey want. However, the way that stem cells are being manipulated is anything but simple. Here is a run-downof fiveof the most fascinating stem cell innovations fromthe past year.

Teeth are necessary for helping uschew our food, but once they fall out. they're useless; or not? The practice of tooth saving, or cryopreserving, has gained popularity, and forgood reason. New research suggests the stem cells found in the pulp of teeth could be used to help people regrow their adult teeth (rather than needing a crown or dentures), and may even have other potentially life-saving regenerative medical benefits, CNN reported.

While still in its early stages, the idea behind tooth preservation is that no other stem cells work better than your own. By saving your baby teeth, oradult teeth that need to be removed through surgery, you may later harvest stem cells that may be used to fight certain cancers or even astherapy for brain injuries.

Read: Stem Cell Research: What Are Stem Cells And Why Is There So Much Controversy

Leukemia is a type of cancer of the blood, and it starts in the bone marrow, which is where our stem cells originate. Traditional leukemia treatment involves a combination of chemotherapy and radiotherapy, but earlier this year doctors at Londons Great Ormond Street hospital believe theycured two babies of leukemia using a new stem cell treatment, Technology Review reported.

The treatment involves taking stem cells from a donor and genetically altering them before injecting them into a patient. These cells are altered so that they are able toattack cancer.

Stem cells are at the forefront of many medical innovations. Photo Courtesy of Pixabay

According to Euro Stem Cell, in traditional stem cell treatments for leukemia patients, cells are taken from donors and then transformed into special cancer-fighting cells; however,this process takes time something many seriously illcancer patients do not have. The Great Ormond Street team hopes that taking stem cells from donors and genetically altering them into hundreds of doses of cancer-fighting cells will create a reserve oftreatments available toanyone who needs them.

According to a study released last year, researchers at Washington University School of Medicine in St. Louis and Harvard University were able to change stem cells derived from the skin of diabetes patients into insulin-secreting cells.

Type 1 diabeticscannot create insulin, which is why patients must inject themselves with this hormone throughout the day. Although this new treatment is still being researched, injecting these stem-cell derived insulin-secreting cells into diabetes patients could control blood sugar without the need formedication.

Stem cellstheoretically can be turned into any type of cell, and as suggested by a 2016 project, this includes brain cells. The project, headed by a team at Bioquark Inc and Revita Life Science India,intendsto regenerate the brain cells of 20 patients that have been declared brain dead from a traumatic brain injury to see whether or not their central nervous systems can be restored, The Telegraph reported.

The team hope the stem cells will grow into new brain cells to replace thedead cells in the brain. While the treatment wouldn't restore these brain-dead patients back to life, the research may lead the way tonew therapies for patients in vegetative states or with certain degenerative conditions.

Brain balls are basically what they sound like;tiny little brains in the shape of balls. According to Wired, they are one of the newest innovations in stem cell research and could hold the answer to treating a variety of medical conditions.

These brain balls are created by coaxing a bunch of stem cells into becoming brain cells, and then using these mini brains to better understand how different diseases affect the brain. For example, according to Wired, these brain balls are ideal for studying conditions such as the Zika virus as scientists can see what's actually happening in an infected brain, but on a much smaller scale.

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Stem Cells Of Type 1 Diabetes Patients Transformed Into Insulin-Secreting Beta Cells; Research May Lead To New Therapy

Scientists Discover Method To 'Expand' Stem Cells In The Laboratory That Could Lead To New Cancer Treatments

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5 Stem Cell Innovations From The Past Year, From Cancer Treatment To Diabetes Therapy - Medical Daily

Outcomes of Burn Patients Treated with Cell Spray Technology … – Business Wire (press release)

NEW YORK & PITTSBURGH--(BUSINESS WIRE)--RenovaCare, Inc., (OTCQB: RCAR), highlighted an analysis of treatment results on a variety of wide-area and severe burn injuries published in Burns, the peer-reviewed Journal of the International Society for Burn Injuries. The treatment method, which involved isolating and spraying the patients own skin stem cells on the burn wounds, is the technology underlying RenovaCares patented CellMist and SkinGun*.

The early cell spray technology which was used to successfully treat a wide spectrum of burn injuries to some of the largest body areas ever treated with stem cell transplantation, has been engineered into todays RenovaCare SkinGun device, explained Mr. Thomas Bold, President and CEO of RenovaCare, Inc.

The results, published in August 2016, report the retrospective analysis of outcomes in 45 severe second-degree burn patients who received skin stem cell spray grafting treatment under an innovative practice approach.

The patients suffered burn wounds such as gas and chemical explosions, as well as electrical, gasoline, hot water and tar scalding burns. Click here to see before-after photos

In the case of one patient with severe electrical burns to over one-third of his body, his wounds were sprayed with 23 million stem cells isolated from a tiny 2 x 2 sample of his own skin. Within five days of treatment, his chest and arms were already healed. Four days later, the patient was discharged from the hospital, said Mr. Bold.

These published analyses are especially encouraging to us because patients were successfully treated with the technology no matter what the source of the burn, concluded Mr. Bold.

Six Burn Causes: Patient Results and Photos Click here to see before-after photos

According to the Burns article authors, regardless of the burn type, cell spray showed quick healing (fast epithelialization), along with other benefits. "Cell-spray grafting is also especially suitable for hands and joint areas, where prolonged times to re-epithelization may significantly impact functionality and esthetic outcome," said the report.

Other findings in the article, following cell spray with the technology include:

Gas Explosion Patient

A gas explosion caused burns to the upper right arm and partial right chest area of a 43-year-old man who also suffered orthopedic injuries. "There was no evidence of hypertrophic (abnormalenlargementofanorgan) scarring throughout the prior burn area, and his only functional impairment ... was due to his wrist injury," concluded the report.

Chemical Explosion Burn

A 37-year-old patient suffered serious burns to his arms and hands as a result of a potassium nitrate explosion. The report observed the following outcome: "... the areas of autografts were noted to be almost indiscernible with the normal skin ... The patient maintained a full range of motion in all extremities without restriction."

Electrical Burn

After grabbing a live electrical wire a 35-year-old male received deep burns to the head, chest, abdomen, back, right hand and foot. Doctors indicated a full recovery to the affected areas in the article: ... all of the areas treated with cell spray grafting were noted as completely healed and re-epithelialized ... the patient had a functional range of motion in all extremities."

Gasoline Flame Burn

A gasoline flame injury to an 18-year-old male resulted in burns to the arms and legs. Forty-five million cells were obtained from the patient and used to spray the entire burn wound surface. "Wounds were completely healed by ... and there was no evidence of ... scarring or contractures, and the patient demonstrated a full range of motion in all extremities," the article said.

Hot Water Scalding

A hot water scalding injury covered a 43-year-old patients upper left arm, shoulder, back and torso. His post cell spray treatment provided the following results: "A 100% re-epithelialization was noted and the patient was discharged that day with instructions to apply Eucerin moisturizer to the wound ... the patient was also noted to have a full range of motion in his extremities, according to the article.

Hot Tar Scalding

Hot tar burned a 43-year-old mans right arm, right hand and midsection and within seven days of treatment the article concluded: all areas were noted to be healed and re-epithelialized and the patient was discharged.

The Burns article titled, Second-degree burns with six etiologies treated with autologous noncultured cell-spray grafting, by: Roger Esteban-Vives, Myung S. Choi, Matthew T. Young, Patrick Over, Jenny Ziembicki, Alain Corcos, and Jrg Gerlach, was published by Elsevier in the November 2016 issue of Burns (Nov;42(7):e99-e106. doi: 10.1016/j.burns.2016.02.020. Epub 2016 Aug 25).

Copies of the article are available to credentialed journalists upon request; please contact Elsevier's Newsroom at newsroom@elsevier.com or+31 20 485 2492.

Study authors, Dr. Roger Esteban-Vives and Dr. Jrg Gerlach currently have a financial interest in the SkinGun spray-grafting technology through payments from RenovaCare, Inc. Dr. Esteban-Vives, who currently Director of Cell Sciences at RenovaCare, Inc., was a postdoctoral fellow at the University of Pittsburgh when this work was conducted and did not have such financial interest at that time.

*RenovaCare products are currently in development.They are not available for sale in theUnited States. There is no assurance that the companys planned or filed submissions to the U.S. Food and Drug Administration, if any, will be accepted or cleared by the FDA.

AboutBurns

Burnsaims to foster the exchange of information among all engaged in preventing and treating the effects of burns. The journal focuses on clinical, scientific and social aspects of these injuries and covers the prevention of the injury, the epidemiology of such injuries and all aspects of treatment including development of new techniques and technologies and verification of existing ones. Regular features include clinical and scientific papers, state of the art reviews and descriptions of burn-care in practice.

About RenovaCare

RenovaCare, Inc. is developing first-of-its-kind autologous (self-donated) stem cell therapies for the regeneration of human organs. Its initial product under development targets the bodys largest organ, the skin. The companys flagship technology, the CellMist System, uses its patented SkinGun to spray a liquid suspension of a patients stem cells the CellMist Solution onto wounds. RenovaCare is developing its CellMist System as a promising new alternative for patients suffering from burns, chronic and acute wounds, and scars. In the US alone, this $45 billion market is greater than the spending on high-blood pressure management, cholesterol treatments, and back pain therapeutics.

For additional information, please call Drew Danielson at: 888-398-0202 or visit: http://renovacareinc.com

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Investors and others should note that we announce material financial information to our investors using SEC filings and press releases. We use our website and social media to communicate with our subscribers, shareholders and the public about the company, RenovaCare, Inc. development, and other corporate matters that are in the public domain. At this time, the company will not post information on social media that could be deemed to be material information unless that information was distributed to public distribution channels first. We encourage investors, the media, and others interested in the company to review the information we post on the companys website and the social media channels listed below:

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Legal Notice Regarding Forward-Looking Statements

No statement herein should be considered an offer or a solicitation of an offer for the purchase or sale of any securities. This release contains forward-looking statements that are based upon current expectations or beliefs, as well as a number of assumptions about future events. Although RenovaCare, Inc. (the Company) believes that the expectations reflected in the forward-looking statements and the assumptions upon which they are based are reasonable, it can give no assurance that such expectations and assumptions will prove to have been correct. Forward-looking statements, which involve assumptions and describe our future plans, strategies, and expectations, are generally identifiable by use of the words may, will, should, could, expect, anticipate, estimate, believe, intend, or project or the negative of these words or other variations on these words or comparable terminology. The reader is cautioned not to put undue reliance on these forward-looking statements, as these statements are subject to numerous factors and uncertainties, including but not limited to: the timing and success of clinical and preclinical studies of product candidates, the potential timing and success of the Companys product programs through their individual product development and regulatory approval processes, adverse economic conditions, intense competition, lack of meaningful research results, entry of new competitors and products, inadequate capital, unexpected costs and operating deficits, increases in general and administrative costs, termination of contracts or agreements, obsolescence of the Company's technologies, technical problems with the Company's research, price increases for supplies and components, litigation and administrative proceedings involving the Company, the possible acquisition of new businesses or technologies that result in operating losses or that do not perform as anticipated, unanticipated losses, the possible fluctuation and volatility of the Company's operating results, financial condition and stock price, losses incurred in litigating and settling cases, dilution in the Company's ownership of its business, adverse publicity and news coverage, inability to carry out research, development and commercialization plans, loss or retirement of key executives and research scientists, and other risks. There can be no assurance that further research and development will validate and support the results of our preliminary research and studies. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that the Company will be able to develop commercially viable products on the basis of its technologies. In addition, other factors that could cause actual results to differ materially are discussed in the Company's most recent Form 10-Q and Form 10-K filings with the Securities and Exchange Commission. These reports and filings may be inspected and copied at the Public Reference Room maintained by the U.S. Securities & Exchange Commission at 100 F Street, N.E., Washington, D.C. 20549. You can obtain information about operation of the Public Reference Room by calling the U.S. Securities & Exchange Commission at 1-800-SEC-0330. The U.S. Securities & Exchange Commission also maintains an Internet site that contains reports, proxy and information statements, and other information regarding issuers that file electronically with the U.S. Securities & Exchange Commission athttp://www.sec.gov. The Company undertakes no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

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Outcomes of Burn Patients Treated with Cell Spray Technology ... - Business Wire (press release)

SMART cells open door to arthritis vaccine – New Atlas

An artist's impression of the reengineered cell that produces an anti-inflammatory drug when it encounters inflammation (Credit: Ella Marushchenko)

Combining two cellular-editing processes, researchers have developed cartilage that fights inflammation. The scientists hope that the breakthrough could eventually lead to localized injections that combat arthritis or perhaps a vaccine that would eliminate the condition altogether.

Like many of the biology breakthroughs happening today, the WU researchers started with stem cells. To be more accurate, they actually started with skin cells from the tails of mice and converted them into stem cells. They then used a gene-editing technique called CRISPR to remove a gene involved in inflammation and replace it with one that releases an anti-inflammatory drug. The resulting cells are known as SMART cells, which stands for Stem cells Modified for Autonomous Regenerative Therapy.

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"Our goal is to package the rewired stem cells as a vaccine for arthritis, which would deliver an anti-inflammatory drug to an arthritic joint but only when it is needed," said Farshid Guilak, the senior author of a paper about the work and a professor of orthopedic surgery at Washington University School of Medicine. "To do this, we needed to create a 'smart' cell."

As part of the current fight against arthritis, there are several drugs that work to eliminate an inflammatory molecule called tumor necrosis factor-alpha (TNF-alpha). The issue with such drugs, however, is that they work throughout the entire body, rather than only at the site of inflammation, and can have an impact on the body's overall immune system.

To change this dynamic, the researchers replaced the gene that expresses TNF-alpha with one that inhibits it by releasing a drug, basically converting the cells from those that create inflammation to those that fight it. "We hijacked an inflammatory pathway to create cells that produced a protective drug," said Jonathan Brunger, a postdoctoral fellow in cellular and molecular pharmacology at the University of California, San Francisco. They then coaxed these cells to grow into cartilage in the lab which, they found, was successful in combating inflammation.

The hope is that injecting the cells into areas afflicted by arthritis, the new anti-inflammatory cartilage could replace the old cartilage. This would effectively create a vaccine against the condition, as the newly engineered cells would only release the anti-inflammatory drug when inflammation is present such as during an arthritic flare-up and turn off the release of the drug when the flare subsides.

Additionally, the researchers also engineered the new cells to light up when they responded to inflammation so that they could track their response in the body. The cells are now being tested in mice with rheumatoid arthritis and other inflammatory disorders and the researchers think that the method of combining stem cells with CRISPR could help fight other diseases as well.

"We believe this strategy also may work for other systems that depend on a feedback loop," said Guilak. "In diabetes, for example, it's possible we could make stem cells that would sense glucose and turn on insulin in response. We are using pluripotent stem cells, so we can make them into any cell type, and with CRISPR, we can remove or insert genes that have the potential to treat many types of disorders."

The paper is published in the journal Stem Cell Reports.

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SMART cells open door to arthritis vaccine - New Atlas

Current CRISPR Patent Dispute, Explained – CALIFORNIA

Current CRISPR Patent Dispute, Explained
CALIFORNIA
They invented CRISPR-Cas9, a gene editing tool that uses a protein found in Streptococcus bacteria to chop up and rearrange viral DNA with precision. The implications of the technology were immediately apparent, astonishing, and perhaps just a wee bit ...

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Current CRISPR Patent Dispute, Explained - CALIFORNIA

CEOs of top gene-editing firms got huge compensation hikes last year – Boston Business Journal


Boston Business Journal
CEOs of top gene-editing firms got huge compensation hikes last year
Boston Business Journal
It's no secret that the burgeoning field of gene-editing a method of cutting out and replacing part of a gene has generated serious buzz in the biotech world lately. New scientific tools like CRISPR/Cas9 have the potential to revolutionize the ...
CRISPR Therapeutics to Present at the 42nd Annual Deutsche Bank Health Care ConferenceGlobeNewswire (press release)
Intellia (NTLA), CRISPR Therapeutics (CRSP) Receive U.S. Patent for CRISPR/Cas9 Ribonucleoprotein ComplexesStreetInsider.com
Intellia Therapeutics and CRISPR Therapeutics Announce U.S. ...Yahoo Finance
Texas Tribune -The Cerbat Gem -Sports Perspectives
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CEOs of top gene-editing firms got huge compensation hikes last year - Boston Business Journal

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