"These new follow-up results based on MRI scans are very encouraging, and strongly suggest that AST-OPC1 cells have engrafted in these patients post-implantation and have the potential to prevent lesion cavity formation, possibly reducing long-term spinal cord tissue deterioration after spinal cord injury," said Dr. Edward Wirth, Chief Medical Officer of Asterias. "Moreover, these new results add to the overall body of data supporting AST-OPC1's safety, and are consistent with safety data from our previous Phase 1 study in thoracic spinal cord injury and our extensive preclinical studies in more than 3,000 animals."

Under the study protocol, patients are monitored by MRI scans at regular intervals over 12 months in order to assess status of the injection site and surrounding tissues.

The Company will discuss the MRI data in more detail on its first quarter 2017 conference call and webcast on May 11, 2017 at 4:30 p.m. Eastern / 1:30 p.m Pacific. For both "listen-only" participants and those participants who wish to take part in the question-and-answer session, the call can be accessed by dialing 800-533-7619 (U.S./Canada) or 785-830-1923 (international) five minutes prior to the start of the call and providing the Conference ID 7610291. To access the live webcast, go to http://asteriasbiotherapeutics.com/inv_events_presentations.php.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/new-mri-data-from-asterias-ongoing-scistar-clinical-study-indicates-ast-opc1-cells-prevent-formation-of-damaging-lesion-cavities-in-patients-suffering-severe-spinal-cord-injury-300455768.html

SOURCE Asterias Biotherapeutics, Inc.

http://www.asteriasbiotherapeutics.com

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New MRI Data from Asterias' Ongoing SCiStar Clinical Study Indicates AST-OPC1 Cells Prevent Formation of ... - PR Newswire (press release)

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South African patients suffering from Leukaemia and other blood disorders who need a life-saving stem cell transplant, rely on the South African Bone Marrow Registry (SAMBR) to find a donor who is a genetic match.

But, the untrue and frightening belief that donating blood stem cells, or bone marrow, involves drilling through bones is a common misconception and is one of the challenges faced in growing the SAMBR.

These misconceptions, often stopping people from registering to become donors and giving someone the hope of life.

The Sunflower Fund educates, raises funds and recruits potential blood stem cell donors to this registry and pays for the tissue typing test cost for each person who joins.

The fund also prioritises educating people on the truths of becoming a donor and works hard to debunk any myths that exist. Sometimes it is just the words themselves that act as barriers. The challenges faced in growing the SABMR lies in the sentence itself, Alana James, CEO of the Sunflower Fund said. Its called The South African Bone Marrow registry, so when people see the word bone marrow they go Oh no I cant do that, it will hurt!.

Blood stem cell donor and long-time supporter of the Sunflower Fund Carey Symons shares her own story at events and conferences, spreading the message that the process of potentially saving a life is not as scary as we might think, encouraging others to do the same.

The stats of a perfect match are 1 in 100 000 so you can only imagine my joy in being that 1 in 100 000 and that I was able to contribute to giving someone a second chance, Symons said, who was called ten years after joining the registry to donate her stem cells to a patient suffering from leukaemia.

The Durban mother travelled to Constantiaberg hospital in Cape Town, to begin a series of painless Neupogen injections which stimulate the production and release of blood stem cells.

After three days of injections, she was ready to begin the donation process: Two needles, similar to the ones used when donating blood, were inserted; one in each arm. Blood was drawn from one arm, circulated through a cell-separator machine where her stem cells were collected and the remaining blood was returned through the other arm.

After 4-6 hours, the life-saving stem cells were harvested and for Symons, the process was over.

She said she often thought about the person she donated her stem cells to and sometimes wonders who they were. I realised that the day I signed as a donor, I was only hoping to make a difference. I will never know whose life I made a difference to, and part of that mystery excites me. Its a blessing to give without knowing and without being thanked.

There are just under 74 000 donors on the registry, but at least 400 000 are need. We definitely still have a mountain to climb and are committedly doing so. Registering as a donor on the SABMR is a simple process and can be very rewarding, James explained. You could be someones perfect match.

Find out more about becoming a blood stem cell a donor by contacting The Sunflower Fund on toll-free number: 0800 12 10 82 or visit http://www.sunflowerfund.org.za.

About The Sunflower Fund:

The Sunflower Fund, a South African Non-Profit Company (NPC), is dedicated to creating awareness, educating the public and handling the registration process for people to join the South African Bone Marrow Registry (SABMR).

The Sunflower Fund pays for the test cost of people joining the SABMR. This is fundamental to saving the lives of thousands of South Africans each year. The chance of finding a matching donor is 1 in 100,000 and as ethnic origin plays a significant role in the search for a donor, South Africas rainbow nation is at a distinct disadvantage, requiring a large pool of prospective donors.

Should you wish to become a donor, support one of the fundraising projects or make a financial contribution, please contact The Sunflower Fund on toll-free number:

0800 12 10 82. Visit http://www.sunflowerfund.org.za to learn more or look out for the DONATE button to make a cash donation via the website.

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The simple truth of saving lives - Independent Online

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Men's Health

This Gun Sprays Stem Cells, Helps Burn Victims Grow Skin in Days Men's Health A revolutionary new technique is enabling burn victims to heal quicker, less painfully, and with more normal skin. And it's all thanks to a gun. The SkinGun sprays stem cells onto wounds and allows patients to grow a new, healthy layer of skin in as ...

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This Gun Sprays Stem Cells, Helps Burn Victims Grow Skin in Days - Men's Health

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Scientists unveil the UK's largest resource of human stem cells from healthy donors Science Daily The Human Induced Pluripotent Stem Cell Initiative (HipSci) project used standardised methods to generate iPSCs on a large scale to study the differences between healthy people. Reference sets of stem cells were generated from skin biopsies donated by ... and more »

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Stem cell therapy is a type of cell therapy wherein cells are introduced into the damaged tissue so as to treat the disorder or the injury. There are a number of medical researchers who believes that the stem cell therapy has the potential to change the treatment of human diseases and reduce the suffering people face when they have a disease. They believe that there are a lot of potential to replace the damaged and diseased tissues in the body without getting the risk of rejections.

The stem cells have the ability to self-renew and also give rise to further generation of cells that can multiply. There are a number of stem cell therapies that do exist but most of them are still in the experimental stages. The treatments are very costly with an exception of bone marrow transplant. However, researchers believe that one day they will be able to develop technologies from embryonic stem cells and also adult stem cells to cure type I diabetes, cancer, Parkinsons disease, cardiac failure, neurological disorders and many more such ailments.

The stem cell therapy however carries its own pros and cons and like any other therapy it cannot be said that the stem cell therapy is an advantageous package. Here are some of the pros and cons of the therapy.

Pros of the stem cell therapy include:

It offers a lot of medical benefits in the therapeutic sectors of regenerative medicine and cloning.

It shows great potential in the treatment of a number of conditions like Parkinsons disease, spinal cord injuries, Alzheimers disease, schizophrenia, cancer, diabetes and many others.

It helps the researchers know more about the growth of human cells and their development.

In future, the stem cell research can allow the scientists to test a number of potential medicines and drugs without carrying out any test on animals and humans. The drug can be tested on a population of cells directly.

The stem cell therapy also allows researchers to study the developmental stages that cannot be known directly through the human embryo and can be used in the treatment of a number of birth defects, infertility problems and also pregnancy loss. A higher understanding will allow the treatment of the abnormal development in the human body.

The stem cell therapy puts into use the cells of the patients own body and hence the risk of rejection can be reduced because the cells belong to the same human body.

The cons of the stem cell therapy include the following:

The use of the stem cells for research involves the destruction of the blastocytes that are formed from the laboratory fertilization of the human egg.

The long term side effects of the therapy are still unknown.

The disadvantage of adult stem cells is that the cells of a particular origin would generate cells only of that type, like brain cells would generate only brain cells and so on.

If the cells used in the therapy are embryonic then the disadvantage is that the cells will not be from the same human body and there are chances of rejection.

The stem cell therapy is still under the process of research and there are a number of things that needs to be established before it used as a treatment line.

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Pros and Cons of Stem Cell Therapy - Health Guidance

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Harvard Gazette

Researchers work to create kidney filtration barrier on a chip ... Harvard Gazette Researchers say their glomerulus-on-a-chip lined by human stem cell-derived kidney cells could help model patient-specific kidney diseases and guide ... and more »

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FinancialsTrend

Why Neuralstem Inc. (NASDAQ:CUR) can't be predicted? FinancialsTrend NEURALSTEM's patent-protected technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature ... Which Analysts Are Watching Neuralstem, Inc. (CUR)? - Fiscal ...Fiscal Standard Neuralstem, Inc. (CUR) Under Analyst SpotlightUK Market News all 15 news articles »

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Q: Ive been reading a lot about stem cells recently. Willstem cell research change the treatment of heart disease?

A: Theres some exciting early data where scientists have been able to use stem cells for regeneration of cardiac tissue, in particular certain parts of the heart or maybe even an entire heart in mice or rats.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services. Policy

However, its not been done yet in humans reliably and that would be the next step. If the research bears out, we may see this as an option for heart patients in perhaps five to 10 years.

The area where stem cells might first be used is in patients who have had damage to their heart because of a heart attack. These patients have scarring on the heart and that area of the heart is not beating anymore. If we can regenerate cardiac tissue to replace this scarred tissue, the hope is to get the heart fully working again.

Growing whole new hearts will likely be later down the line and will depend on the success of the research.

Preventive cardiologistHaitham Ahmed, MD, MPH

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Will Stem Cell Research Change Treatment of Heart Disease? - Health Essentials from Cleveland Clinic (blog)

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Lab-engineered bone (the outer layer) with functional bone marrow (the inner layer). Image: Varghese Lab at UC San Diego

Thanks to advances in medicine, bone marrow transplants are no longer the last resorts they once were. Every year, thousands of marrow transplants are performed, a common treatment for ailments from bone marrow disease to leukemia. But because they first require a patient undergo radiation to kill off any existing bone marrow stem cells, marrow transplants remain incredibly hard on a patient.

Now, engineers at the University of California San Diego have developed a synthetic bone implant with functional marrow able to produce its own blood cells. So far, researchers revealed in a paper published in the Proceedings of the National Academy of Sciencesthis week, they have successfully tested the engineered bone tissues in mice. But one day, those biomimetic bone tissues could provide new bone marrow for human patients in need of transplants, too.

The implant does away with the need for radiation by giving donor cells their own space in the body to grow. Inside the implant, there is no threat of those cells being overtaken by the bodys native stem cells.

In mice, the researchers implanted the synthetic bone tissues with functional marrow under the skin. After six months, those donor cells were still alive and had begun supplying the mice with new blood cells.

The implants were designed to replicate the long bones in the body, with an outer bone compartment containing calcium phosphate minerals to build bone cells, and an inner area for donor stem cells that produce blood cells.

When implanted, they grew into bone tissues with working blood vessel network and functional bone marrow that supplied the body new blood cells. After 24 weeks, researchers found a mix of host and donor blood cells was still circulating in the bloodstream of the mice.

A treatment based on this technology would only work for patients with non-malignant bone marrow diseases, like aplastic anemia, a condition where the body cant make enough platelets and blood cells. Thats because while the technique can replenish types of cells that are lacking, it cant doing anything to fight off cells that have mutated and are spreading. Cancer patients would still need need to undergo radiation therapy to have their cancerous cells wiped out.

Much more research is needed, of course, before these implants are ready to make their way into human patients. But whats exciting here is that the synthetic bone tissues were not only functional, they allowed donor marrow to grow and survive for many weeks in the presence of host cells, and for the products of that marrow to make their way into the bodys circulatory system. Pretty neat.

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This Synthetic Bone Implant Could Replace Painful Marrow Transplants - Gizmodo

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Be The Match BioTherapies LLC, a recently created subsidiary of the Minneapolis-based National Bone Marrow Program/Be The Match, this month emerged as a biotechnology venture investor with its participation in a $50 million financing round for a Massachusetts stem cell company. And, its leaders say, it likely wont be the last time the 17-month-old nonprofit spinoff will take part in venture funding to support the commercialization of biotech related to NBMP/Be The Matchs mission of advancing cell therapies for leukemia patients and others needing bone marrow/stem cell transplants. NMDP/Be The Match moved its 995 employees into a newly constructed headquarters building in the North Loop in December 2015. It runs a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries. Wholly-owned subsidiary Be The Match BioTherapies was among the Series B investors for Magenta Therapeutics of Cambridge, Massachusetts, a biotech company developing therapies to improve and expand the use of curative stem cell transplantation for more patients. Other participants in the oversubscribed round included new lead investor GV (formerly Google Ventures) and existing investors such as Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. A major feature of the Magenta deal was also Be The Match BioTherapies new involvement as a strategic partner for the company, under which the two sides will explore opportunities to work together across all of Magentas research efforts, from discovery through clinical development. Magentas lead drug candidate is MGTA-456, which it claims is capable of expanding the number of cord blood stem cells available for transplantation, thus achieving superior clinical outcomes compared to standard transplant procedures. John Wagner M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota is leading the research. The strategic agreement allows Magenta to leverage Be The Match BioTherapies capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management and patient outcomes data generated from the parent organization. According to NMDPs 2015 annual report, Be The Match BioTherapies was established on Dec. 4, 2015, and authorized to do business as a nonprofit limited liability company. The report said it was anticipated the subsidiary would conduct certain business in the field of cellular therapy consistent with the nonprofit mission of its parent corporation, National Marrow Donor Program, but outside the scope of NMDPs customary core business. Led by NMDP Chief Financial Officer Amy Ronneberg, Be The Match BioTherapies says it is making the parent organizations capabilities available to commercial entities developing new allogeneic and autologous cellular therapies. For example, it says it is collaborating with an unnamed biotech company to design a donor identification and cell harvest strategy for white blood cells from donors with specific human leukocyte antigen types. When asked if the subsidiarys venture investment into Magenta Therapies was a sign that it is staking out ground as a stem cell industry investment player, company spokeswoman Melissa Neill told TCB its indeed a scenario that could play out again. We are continually looking for ways to advance science and research in new cellular therapies, she said in an email. In the future, this might mean investments in or additional partnerships with companies whose goals align with our goal of developing and delivering cellular therapies to positively impact patients lives.

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Be The Match Subsidiary Emerges as a Biotech Venture Player - Twin Cities Business Magazine

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Technology Networks

Stems Cells Could Help Treat Slipped Discs Technology Networks The study on the sick German shepherds was organized as follows: With the permission of the dog owners, neurologist Frank Steffen and his team removed stem cells from the marrow of the pelvic bone of the affected animals. After the cleaning and ... and more »

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A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia.

A public sector stem cell bank is set to come up at UP's King George's Medical University. A project of the university's transfusion medicine department, the stem cell bank would roll out stem cell therapy to patients of thalassemia and sickle cell anaemia. The proposal is awaiting clearance from state department of medical education.

Stem cells are found in human bone marrow and can be derived from the umbilical cord which contains blood vessels that connect baby in the womb to the mother to ingest nutrition required for development.

Research on the therapeutic use of stem cells is underway in US, Europe, China, South East Asia besides India. In UP, Sanjay Gandhi Post Graduate Institute of Medical Sciences (SGPGIMS) and KGMU are both trying to explore the potential of stem cells to treat various health problems. SGPGI has, so far, restricted itself to use of allogenic (stem cells derived from bone marrow of a person), while KGMU has used stem cells derived from the umbilical cord.

KGMU has sustained access to umbilical cord because of a very developed obstetrics and gynaecology department. The proposal is worth Rs 9 crore including infrastructure cost. Stem cell bank promises to become financially self-sustaining within 2-3 years of inception.

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Stem cell bank to come up at KGMU - BSI bureau (press release)

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Sensational 8-Year-Old Violinist Living With Painful Disease

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

We only listen to classical music at home, said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of two.*

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

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All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

Every month, Caesar and his family travel to Charlotte for blood transfusions to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.*

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

*This story has been updated to correct information. Lessons for Caesar started at the age of 2 and 300ml of his blood is replaced every month during his blood transfusions.

5 Facts About Sickle Cell Disease (CDC)

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Get burned over the weekend? RenovaCare has got your back. The New York-based biotech company has expertise in stem cells and organ regeneration, and has brought these skills to bear on wound care. One of the companys most promising methods uses a literal skin gun to spray skin stem cells on a burn or chronic wound to promote rapid healing. The healing is so rapid that you can walk into the hospital with a burn on a Friday night and return on Monday largelyhealed.

The skin gun process uses a patients stem cells, which are collected from healthy skin. The stem cells are isolated from the skin sample and suspended in a water solution that makes them easy to spray. Thecomputer-controlled skin gun works like the air brushes that are used by painters, but with much more precision.

The treatment is stupidly simple just spray the stem cells on the burned skin and wait for them to regrow. It is also extremely fast, taking only 1.5 hours to isolate the cells and and spray the skin. Once the skin cells are applied, it takes only a few days for the treatment to be effective. When state trooper Matthew Uram was burned in an unfortunate bonfire accident, he chose this experimental treatment and was entirely healed from his second-degree burns in four days.

This skin gun approach offers a significant improvement over the current methods of in-lab skin growth and surgical grafting that takes weeks and sometimes even months to be effective. Those who undergo these conventional skin graft techniques often suffer from infections and other setbacks, rendering the treatment far from optimal. A technology like the skin gun that could promote complete healing in a matter of days would represent a clear advance.

RenovaCares skin gun is still in the developmental stage and has not been approved by the FDA for sale in the United States, so you wont be able to find it on the shelves of burn units quite yet. The company is making progress towards that goal, however, and has recently announceda successful round of testing that shows its gun is capable of dispersing the skin cell liquid in a very uniform and dense manner.

Recent experiments conducted at Stem Cell Systems GmbH (Berlin, Germany) show that the gun can spray more than 20,000 evenly distributed droplets in a test area as compared to a conventional needle and syringe which produced only 91. The gun is not only capable of even dispersal, but it also is gentle on the skin stem cells, which retain 97.3 percent viability after SkinGun spraying. RenovaCare is continuing its research and development as it moves towards FDA approval and eventual commercial rollout. The company recently a filed a 510(k) submission with the FDA, which is a notice of intent to market a device and often is the first step before clinical trials.

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New Burn Healing Method uses Skin-Gun Stem-Cell Therapy ...

Skin Stem Cells Comments Off on New Burn Healing Method uses Skin-Gun Stem-Cell Therapy … | May 10th, 2017

A molecular switch has been identified that converts skin cells into cells found in blood vessels, raising hopes of aiding heart disease sufferers.

This technique boosts levels of an enzyme that keeps cells young and could also potentially help cells avoid ageing as they are grown in the lab. Although this technique has been used before, this is the first time it has been understood by scientists.

Some techniques to convert mature skin cells into pluripotent stem cells use a cocktail of chemicals to ensure they turn into designated cell types. Other methods modify cells, skippingthe stem cell state completely. Recently, researchers have been exploring rewinding skin cells so they lose some of their mature cell identity.

Dr Jalees Rehman, who led the study at the University of Illinois at Chicago, said: They dont revert all the way back to a pluripotent stem cell, but instead turn into intermediate progenitor cells. Even though they only differentiate into a few different cell types, progenitor cells can be grown in large quantities, making them suitable for regenerative therapies.

Rehmans research group discovered that progenitor cells could be converted into blood vessel endothelial cells or erythrocytes depending on the level of a gene transcription factor called SOX17. When SOX17 levels were increased, progenitor cells were five times as likely to become endothelial cells. When this process was reversed, fewer endothelial cells but more erythrocytes were produced.

Dr Rehman said: It makes a lot of sense that SOX17 is involved because it is abundant in developing embryos when blood vessels are forming. When human progenitor cells were embedded into a gel implanted into mice, the cells formed functional human blood vessels. Mice that were suffering from heart damage formed functional human blood vessels in their hearts even interlinking with existing murine vessels to improve heart function.

During the course of the research, the scientists observed increased levels of telomerase the anti-ageing enzyme responsible for telomeres on the ends of chromosome in progenitor cells. The increase in telomerase we see in the progenitor cells could be an added benefit of using this partial de-differentiation technique for the production of new blood vessels for patients with cardiac disease, especially for older patients, said Dr Rehman. The process of converting and expanding these cells in the lab could make them age even further and impair their long-term function. But if the cells have elevated levels of telomerase, the cells are at lower risk of premature ageing.

Increased levels of telomerase are also observed in cancer cells, enabling cell division to occur at avery high rate. However, the scientists didnt observe any tumour formation during their research and their next steps will involve further research over a longer time period in larger animals. The study was published in Circulation.

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Protein enables scientists to convert skin to blood vessels - Lab News

Skin Stem Cells Comments Off on Protein enables scientists to convert skin to blood vessels – Lab News | May 10th, 2017

Jonathan Pitre was exhausted on Tuesday, but he found some strength while watching the Ottawa Senators close out the New York Rangers in Game 6 of their second-round playoff series. -

His white blood cell count rising slowly, Jonathan Pitre will have a medical test Thursday to answer a crucial question: Are the new cells in his bloodstream genetically different?

The answer will reveal whether his second stem cell transplant has taken root in his bone marrow.

I want to be excited but Im holding back until we know for sure, said Pitres mother, Tina Boileau, who has been at his side in Minnesota since the transplant one month ago. Once we know, it seems like well be able to put one foot in front of the other and move on.

The family is taking a cautious approach since Pitres first transplant ended in disappointment in October when doctors learned that his own stem cells had recolonized his bone marrow.

Thursdays test will determine the source of Pitres new cells by isolating his white blood cells and examining the DNA they contain. All of Pitres cells will have a pair of X and Y chromosomes, but doctors will be hoping to find cells with a pair of X chromosomes since those cells can only come from his mother.

Such a discovery would provide evidence that the stem cells donated by Boileau have taken root in her sons bone marrow, and have started to produce new blood cells.

Im really hoping for a positive outcome; I think were due for good news, said Boileau, who expects to learn the results on Monday.

Pitre, who turns 17 next month, has seen his white blood cell count climb recently from 0.0 to 0.4, which remains well below the normal range of 4.0 to 11.0. He continues to suffer fevers, pain and profound exhaustion.

On Tuesday night, he watched the Ottawa Senators close out the New York Rangers while his mother applied fresh dressings and gauze after his bath. It was the first time in his life, Boileau said, that her son did not have the strength to stand during the procedure.

We had the game on and I have to say it really helped us get through it, said Boileau. Jonathan got a bit of strength from the excitement, and it was just enough to help me finish his dressings.

Pitre told his mother Tuesday night that hes not sure if he can see this one through.

I said, Youre going to have to because theres no way Im going home without you,' Boileau said. He managed to crack a little smile and said, OK, mom.

The University of Minnesota Masonic Childrens Hospital is theonly facility in the world that offers a blood and marrow transplant as a treatment for those with severe epidermolysis bullosa (EB). If Pitres transplant is successful, his new stems cells will have the power to deliver to his injured skin cells that can secrete a missing protein essential to the development of collagen.

Collagen is the glue that gives skin its strength and structure, and those with Pitres disease, recessive dytstrophic EB, are missing it. The treatment holds the potential to dramatically improve Pitres skin and make his disease more manageable.

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An exhausted Jonathan Pitre will soon learn if his stem cell transplant has worked - Ottawa Citizen

Skin Stem Cells Comments Off on An exhausted Jonathan Pitre will soon learn if his stem cell transplant has worked – Ottawa Citizen | May 10th, 2017

May 10, 2017 Eye stem cells. Credit: University of Southampton

Reported in Nature today, one of the largest sets of high quality human induced pluripotent stem cell lines from healthy individuals has been produced by a consortium involving the Wellcome Trust Sanger Institute. Comprehensively annotated and available for independent research, the hundreds of stem cell lines are a powerful resource for scientists studying human development and disease.

With collaborative partners from King's College London, the European Bioinformatics Institute, the University of Dundee and the University of Cambridge, the study also investigates in unprecedented detail the extensive variation between stem cells from different healthy people.

Technological advancements have made it possible to take an adult cell and use specific growth conditions to turn back the clock - returning it to an early embryonic state. This results in an induced pluripotent stem cell (iPSC), which can develop into any type of cell in the body. These iPSCs have huge scientific potential for studying the development and the impact of diseases including cancer, Alzheimer's, and heart disease.

However, the process of creating an iPSC is long and complicated and few laboratories have the facilities to characterise their cells in a way that makes them useful for other scientists to use.

The Human Induced Pluripotent Stem Cell Initiative (HipSci) project used standardised methods to generate iPSCs on a large scale to study the differences between healthy people. Reference sets of stem cells were generated from skin biopsies donated by 301 healthy volunteers, creating multiple stem cell lines from each person.

The researchers created 711 cell lines and generated detailed information about their genome, the proteins expressed in them, and the cell biology of each cell line. Lines and data generated by this initiative are available to academic researchers and industry.

Dr Daniel Gaffney, a lead author on the paper, from the Wellcome Trust Sanger Institute, said: "We have created a comprehensive, high quality reference set of human induced pluripotent stem cell lines from healthy volunteers. Each of these stem cell lines has been extensively characterised and made available to the wider research community along with the annotation data. This resource is a stepping stone for researchers to make better cell models of many diseases, because they can study disease risk in many cell types, including those that are normally inaccessible."

By creating more than one stem cell line from each healthy individual, the researchers were able to determine the similarity of stem cell lines from the same person.

Prof Fiona Watt, a lead author on the paper and co-principal investigator of HipSci, from King's College London, said: "Many other efforts to create stem cells focus on rare diseases. In our study, stem cells have been produced from hundreds of healthy volunteers to study common genetic variation. We were able to show similar characteristics of iPS cells from the same person, and revealed that up to 46 per cent of the differences we saw in iPS cells were due to differences between individuals. These data will allow researchers to put disease variations in context with healthy people."

The project, which has taken 4 years to complete, required a multidisciplinary approach with many different collaborators, who specialised in different aspects of creating the cell lines and characterising the data.

Dr Oliver Stegle, a lead author on the paper, from the European Bioinformatics Institute, said: "This study was only possible due to the large scale, systematic production and characterisation of the stem cell lines. To help us to understand the different properties of the cells, we collected extensive data on multiple molecular layers, from the genome of the lines to their cell biology. This type of phenotyping required a whole facility rather than just a single lab, and will provide a huge resource to other scientists. Already, the data being generated have helped to gain a clearer picture of what a typical human iPSC cell looks like."

Dr Michael Dunn, Head of Genetics and Molecular Sciences at Wellcome, said: "This is the fantastic result of many years of work to create a national resource of high quality, well-characterised human induced pluripotent stem cells. This has been a significant achievement made possible by the collaboration of researchers across the country with joint funding provided by Wellcome and the MRC. It will help to provide the knowledge base to underpin a huge amount of future research into the effects of our genes on health and disease. By ensuring this resource is openly available to all, we hope that it will pave the way for many more fascinating discoveries."

Explore further: Stem cell consortium tackles complex genetic diseases

More information: Helena Kilpinen et al, Common genetic variation drives molecular heterogeneity in human iPSCs, Nature (2017). DOI: 10.1038/nature22403

http://www.yourgenome.org/facts/what-is-a-stem-cell

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Scientists unveil the UK's largest resource of human stem cells from healthy donors - Medical Xpress

Skin Stem Cells Comments Off on Scientists unveil the UK’s largest resource of human stem cells from healthy donors – Medical Xpress | May 10th, 2017

Shinya Yamanaka ( , Yamanaka Shin'ya?, born September 4, 1962) is a Japanese Nobel Prize-winning stem cell researcher.[1][2][3] He serves as the director of Center for iPS Cell (induced Pluripotent Stem Cell) Research and Application and a professor at the Institute for Frontier Medical Sciences(ja) at Kyoto University; as a senior investigator at the UCSF-affiliated J. David Gladstone Institutes in San Francisco, California; and as a professor of anatomy at University of California, San Francisco (UCSF). Yamanaka is also a past president of the International Society for Stem Cell Research (ISSCR).

He received the 2010 BBVA Foundation Frontiers of Knowledge Award in Biomedicine category. Also he received the Wolf Prize in Medicine in 2011 with Rudolf Jaenisch;[6] the Millennium Technology Prize in 2012 together with Linus Torvalds. In 2012 he and John Gurdon were awarded the Nobel Prize for Physiology or Medicine for the discovery that mature cells can be converted to stem cells.[7] In 2013 he was awarded the $3 million Breakthrough Prize in Life Sciences for his work.

Yamanaka was born in Higashisaka Japan in 1962. After graduating from Tennji High School attached to Osaka Kyoiku University,[8] he received his M.D. at Kobe University in 1987 and his PhD at Osaka City University Graduate School in 1993. After this, he went through a residency in orthopedic surgery at National Osaka Hospital and a postdoctoral fellowship at the Gladstone Institute of Cardiovascular Disease, San Francisco.

Afterwards he worked at the Gladstone Institutes in San Francisco, USA and Nara Institute of Science and Technology in Japan. Yamanaka is currently a Professor at Kyoto University, where he directs its Center for iPS Research and Application. He is also a senior investigator at the Gladstone Institutes as well as the director of the Center for iPS Cell Research and Application(ja).[9]

Between 1987 and 1989, Yamanaka was a resident in orthopedic surgery at the National Osaka Hospital. His first operation was to remove a benign tumor from his friend Shuichi Hirata, a task he could not complete after one hour when a skilled surgeon would have taken ten minutes or so. Some seniors referred to him as "Jamanaka", a pun on the Japanese word for obstacle.[10]

From 1993 to 1996, he was at the Gladstone Institute of Cardiovascular Disease. Between 1996 and 1999, he was an assistant professor at Osaka City University Medical School, but found himself mostly looking after mice in the laboratory, not doing actual research.[10]

His wife advised him to become a practicing doctor, but instead he applied for a position at the Nara Institute of Science and Technology. He stated that he could and would clarify the characteristics of embryonic stem cells, and this can-do attitude won him the job. From 19992003, he was an associate professor there, and started the research that would later win him the 2012 Nobel Prize. He became a full professor and remained at the institute in that position from 20032005. Between 2004 and 2010, Yamanaka was a professor at the Institute for Frontier Medical Sciences.[11] Currently, Yamanaka is the director and a professor at the Center for iPS Cell Research and Application at Kyoto University.

In 2006, he and his team generated induced pluripotent stem cells (iPS cells) from adult mouse fibroblasts.[1] iPS cells closely resemble embryonic stem cells, the in vitro equivalent of the part of the blastocyst (the embryo a few days after fertilization) which grows to become the embryo proper. They could show that his iPS cells were pluripotent, i.e. capable of generating all cell lineages of the body. Later he and his team generated iPS cells from human adult fibroblasts,[2] again as the first group to do so. A key difference from previous attempts by the field was his team's use of multiple transcription factors, instead of transfecting one transcription factor per experiment. They started with 24 transcription factors known to be important in the early embryo, but could in the end reduce it to 4 transcription factors Sox2, Oct4, Klf4 and c-Myc.[1]

Yamanaka practiced judo (2nd Dan black belt) and played rugby as a university student. He also has a history of running marathons. After a 20-year gap, he competed in the inaugural Osaka Marathon in 2011 as a charity runner with a time of 4:29:53. He also took part in the 2012 Kyoto Marathon to raise money for iPS research, finishing in 4:03:19. He also ran in the second Osaka Marathon on November 25, 2012.[12]

In 2007, Yamanaka was recognized as a "Person Who Mattered" in the Time Person of the Year edition of Time Magazine.[13] Yamanaka was also nominated as a 2008 Time 100 Finalist.[14] In June 2010, Yamanaka was awarded the Kyoto Prize for reprogramming adult skin cells to pluripotential precursors. Yamanaka developed the method as an alternative to embryonic stem cells, thus circumventing an approach in which embryos would be destroyed.

In May 2010, Yamanaka was given "Doctor of Science honorary degree" by Mount Sinai School of Medicine.[15]

In September 2010, he was awarded the Balzan Prize for his work on biology and stem cells.[16]

Yamanaka has been listed as one of the 15 Asian Scientists To Watch by Asian Scientist magazine on May 15, 2011.[17][18] In June 2011, he was awarded the inaugural McEwen Award for Innovation; he shared the $100,000 prize with Kazutoshi Takahashi(ja), who was the lead author on the paper describing the generation of induced pluripotent stem cells.[19]

In June 2012, he was awarded the Millennium Technology Prize for his work in stem cells.[20] He shared the 1.2 million euro prize with Linus Torvalds, the creator of the Linux kernel.

In October 2012, he and fellow stem cell researcher John Gurdon were awarded the Nobel Prize in Physiology or Medicine "for the discovery that mature cells can be reprogrammed to become pluripotent."[21]

The 2012 Nobel Prize in Physiology or Medicine was awarded jointly to Sir John B. Gurdon and Shinya Yamanaka "for the discovery that mature cells can be reprogrammed to become pluripotent."[22]

There are different types of stem cells

. These are some types of cells that will help in understanding the material.

totipotency remains through the first few cell divisions ex. the fertilised egg

The early embryo consists mainly of pluripotent stem cells

ex) blood multipotent cells can develop into various blood cells

Theoretically patient-specific transplantations possible

Much research done Immune rejection reducible via stem cell bank

Pluripotent

Abnormal aging

No immune rejection Safe (clinical trials)

The prevalent view during the early 20th century was that mature cells were permanently locked into the differentiated state and cannot return to a fully immature, pluripotent stem cell state. They thought that cellular differentiation can only be a unidirectional process. Therefore, non-differentiated egg/early embryo cells can only develop into specialized cells. However, stem cells with limited potency (adult stem cells) remain in bone marrow, intestine, skin etc. to act as a source of cell replacement.[23]

The fact that differentiated cell types had specific patterns of proteins suggested irreversible epigenetic modifications or genetic alterations to be the cause of unidirectional cell differentiation. So, cells progressively become more restricted in the differentiation potential and eventually lose pluripotency.[24]

In 1962, John B. Gurdon demonstrated that the nucleus from a differentiated frog intestinal epithelial cell can generate a fully functional tadpole via transplantation to an enucleated egg. Gurdon used somatic cell nuclear transfer (SCNT) as a method to understand reprogramming and how cells change in specialization. He concluded that differentiated somatic cell nuclei had the potential to revert to pluripotency. This was a paradigm shift during the time. It showed that a differentiated cell nucleus has retained the capacity to successfully revert to an undifferentiated state, with the potential to restart development (pluripotent capacity).

However, the question still remained whether an intact differentiated cell could be fully reprogrammed to become pluripotent.

Shinya Yamanaka proved that introduction of a small set of transcription factors into a differentiated cell was sufficient to revert the cell to a pluripotent state. Yamanaka focused on factors that are important for maintaining pluripotency in embryonic stem (ES) cells. Knowing that transcription factors were involved in the maintenance of the pluripotent state, he selected a set of 24 ES cell transcriptional factors as candidates to reinstate pluripotency in somatic cells.

First, he collected the 24 candidate factors. When all 24 genes encoding these transcription factors were introduced into skin fibroblasts, few actually generated colonies that were remarkably similar to ES cells. Secondly, further experiments were conducted with smaller numbers of transcription factors added to identify the key factors, through a very simple and yet sensitive assay system. Lastly, he identified the four key factors. They found that 4 transcriptional factors (Myc, Oct3/4, Sox2 and Klf4) were sufficient to convert mouse embryonic or adult fibroblasts to pluripotent stem cells (capable of producing teratomas in vivo and contributing to chimeric mice).

These pluripotent cells are called iPS (induced pluripotent stem) cells; they appeared with very low frequency.

iPS cells can be selected by inserting the b-geo gene into the Fbx15 locus. The Fbx15 promoter is active in pluripotent stem cells which induce b-geo expression, which in turn gives rise to G418 resistance; this resistance helps us identify the iPS cells in a culture.

Moreover, in 2007, Yamanaka and his colleagues found iPS cells with germ line transmission (via selecting for Oct4 or Nanog gene). Also in 2007, they were the first to produce human iPS cells.

However, there are some difficulties to overcome. The first is the issue of the very low production rate of iPS cells, and the other is the fact that the 4 transcriptional factors are shown to be oncogenic.

Nonetheless, this is a truly fundamental discovery. This was the first time an intact differentiated somatic cell could be reprogrammed to become pluripotent. This opened up a completely new research field.

In July 2014, a scandal regarding the research of Haruko Obokata was connected to Yamanaka. He could not find the lab notes from the period in question [25] and was made to apologise.[26][27]

Since the original discovery by Yamanaka, much further research has been done in this field, and many improvements have been made to the technology. Here we[who?] discuss the improvements made to Yamanaka's research as well as the future prospects of his findings.

1. The delivery mechanism of pluripotency factors has been improved. At first retroviral vectors, that integrate randomly in the genome and cause deregulation of genes that contribute to tumor formation, were used. However, now, non-integrating viruses, stabilised RNAs or proteins, or episomal plasmids (integration-free delivery mechanism) are used.

2. Transcription factors required for inducing pluripotency in different cell types have been identified (e.g. neural stem cells).

3. Small substitutive molecules were identified, that can substitute for the function of the transcription factors.

4. Transdifferentiation experiments were carried out. They tried to change the cell fate without proceeding through a pluripotent state. They were able to systematically identify genes that carry out transdifferentiation using combinations of transcription factors that induce cell fate switches. They found trandifferentiation within germ layer and between germ layers, e.g., exocrine cells to endocrine cells, fibroblast cells to myoblast cells, fibroblast cells to cardiomyocyte cells, fibroblast cells to neurons

5. Cell replacement therapy with iPS cells is a possibility. Stem cells can replace diseased or lost cells in degenerative disorders and they are less prone to immune rejection. However, there is a danger that it may introduce mutations or other genomic abnormalities that render it unsuitable for cell therapy. So, there are still many challenges, but it is a very exciting and promising research area. Further work is required to guarantee safety for patients.

6. Can medically use iPS cells from patients with genetic and other disorders to gain insights into the disease process. - Amyotrophic lateral sclerosis (ALS), Rett syndrome, spinal muscular atrophy (SMA), 1-antitrypsin deficiency, familial hypercholesterolemia and glycogen storage disease type 1A. - For cardiovascular disease, Timothy syndrome, LEOPARD syndrome, type 1 and 2 long QT syndrome - Alzheimers, Spinocerebellar ataxia, Huntingtons etc.

7. iPS cells provide screening platforms for development and validation of therapeutic compounds. For example, kinetin was a novel compound found in iPS cells from familial dysautonomia and beta blockers & ion channel blockers for long QT syndrome were identified with iPS cells.

Yamanaka's research has opened a new door and the world's scientists have set forth on a long journey of exploration, hoping to find our cells true potential.[28]

In 2013, iPS cells were used to generate a human vascularized and functional liver in mice in Japan. Multiple stem cells were used to differentiate the component parts of the liver, which then self-organized into the complex structure. When placed into a mouse host, the liver vessels connected to the hosts vessels and performed normal liver functions, including breaking down of drugs and liver secretions. [29]

General references:

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Shinya Yamanaka - Wikipedia

IPS Cell Therapy Comments Off on Shinya Yamanaka – Wikipedia | May 10th, 2017

Induced pluripotent stem cells have revolutionized stem cell science in the decade since their invention. Theyre yielding clues into the nature of diseases such as cancer and Alzheimers, and are also being tapped for therapy.

But creating these IPS cells is lengthy, complicated and tricky, and the facilities equipped to make them cant accommodate all the scientists whod like to get their hands on them.

A UK-led consortium has removed that bottleneck, by producing 711 lines of ready-to-go IPS cells from healthy individuals. These lines are meant to help scientists understand the normal variations between healthy individuals and those involved in disease, as well as to understand normal human biology and development.

The IPS lines are available for research purposes to academic scientists and industry by contacting the Human Induced Pluripotent Stem Cell Initiative (HipSci), at http://www.hipsci.org and the European Bank for induced Pluripotent Stem Cells at https://www.ebisc.org.

The accomplishment was announced in a study published in Nature. It can be found online at j.mp/711ips.

While many other efforts have generated IPS cells to address rare diseases, this study produces them from healthy volunteers to plumb common genetic variation, Fiona Watt, a lead author on the paper and co-principal investigator of HipSci, from King's College London, said in a statement.

"We were able to show similar characteristics of iPS cells from the same person, and revealed that up to 46 per cent of the differences we saw in iPS cells were due to differences between individuals, Watt said in the statement. These data will allow researchers to put disease variations in context with healthy people."

Andrs Bratt-Leal, director of the Parkinson's Cell Therapy Program at The Scripps Research Institute in La Jolla, agreed.

This kind of study is extremely important because it leads to a deeper understanding of the differences between normal genetic variation and genetic changes that could negatively impact cell behavior, said Bratt-Leal, who was not involved in the study.

This data will help scientists using induced pluripotent stem cells to model diseases as well as scientists developing cell therapies, said Bratt-Leal, who works in the lab of stem cell researcher Jeanne Loring.

Because DNA sequencing has become a routine tool in the lab, enormous amounts of data have been produced, he said. Not only have we have observed a high level of genetic diversity between different people, but also a more subtle variation exists among the cells from an individual person. The next step is a better understanding of how this diversity translates to function and behavior of stem cells and mature cells derived from stem cells.

Loring and Bratt-Leal are studying the use of induced pluripotent stem cells to relieve symptoms of Parkinsons disease. They are in the process of translating the research into a therapy, aided with a grant from the California Institute for Regenerative Medicine.

The work was the product of a large-scale collaboration of scientists from various institutions in the United Kingdom, including the European Molecular Biology Laboratory in Cambridge; Wellcome Trust Sanger Institute in Cambridge; the University of Dundee in Dundee; and the University of Cambridge. Also participating was St Vincent's Institute of Medical Research in Victoria, Australia.

bradley.fikes@sduniontribune.com

(619) 293-1020

UPDATES:

1:00 p.m.: This article was updated with additional details.

This article was originally published at 10:00 a.m.

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Hundreds of new stem cell lines ready to help research - The San Diego Union-Tribune

IPS Cell Therapy Comments Off on Hundreds of new stem cell lines ready to help research – The San Diego Union-Tribune | May 10th, 2017