Palm Beach Post (blog)

Blinded by science: Women go blind after stem-cell treatment at Florida clinic Palm Beach Post (blog) In 2010, for example, a woman with the autoimmune disease lupus died after her own bone marrow cells were injected into her kidneys at a clinic in Thailand. In 2013, the Florida Department of Health revoked the medical license of Zannos Grekos over the ... Stem cells seem speedier in spacePhys.Org Stem cell therapy is safe for stroke patients, study showsScience Daily Borrowing from nature: UW-Madison scientists use plants to grow stem cellsMadison.com American Council on Science and Health -Medgadget (blog) -The Republic of East Vancouver all 34 news articles »

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Blinded by science: Women go blind after stem-cell treatment at Florida clinic - Palm Beach Post (blog)

Science Daily

First patient cured of rare blood disorder Science Daily The transplant technique is unique, because it allows a donor's cells to gradually take over a patient's bone marrow without using toxic agents to eliminate a patient's cells prior to the transplant. ... treatment options have been limited because they ... Stem Cell Transplant Cures a Man With Rare Blood DisorderNature World News Doctors cure first patient with rare blood disorderIANS all 3 news articles »

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First patient cured of rare blood disorder - Science Daily

The study consists of donor and patient pairs for allogeneic hematopoietic cell transplantation. The first part of the study, which is nearing completion, is intended to enroll an initial cohort of 10 donor and recipient pairs, consisting of patients with advanced hematological malignancies and their HLA-matched sibling donors. Interim results show that a single injection of BL-8040 mobilized sufficient amounts of cells required for transplantation at a level of efficacy similar to that achieved by using 4-6 injections of G-CSF, the current standard of care. Furthermore, all recipients transplanted so far have experienced a successful neutrophil engraftment. The recipients will be followed for one year to assess acute and chronic GVHD events. As for the donors, BL-8040 treatment was safe and well tolerated.

Philip Serlin, Chief Executive Officer of BioLineRx, stated, "We are very encouraged by these initial results of the Phase 2 clinical trial for assessing BL-8040, our lead oncology and hematology platform, as a single agent for hematopoietic stem cell mobilization for allogeneic transplantation. Hematopoietic stem cells are increasingly used as part of the treatment regimen for certain types of hematological cancers, as well as for severe anemia and immune deficiency disorders. These results, supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen, for rapid mobilization of substantial amounts of stem cells, represent a significant improvement over the current standard of care, which requires four-to-six daily injections of G-CSF and one-to-four apheresis sessions.If there are no safety concerns regarding graft failure or rejection after the interim safety review of donor-recipient pairs participating in Part 1 of the study, we will continue with Part 2 of the study, which will permit enrollment of recipients with either matched sibling or haploidentical donors, up to a total enrollment in the study of 24 donor-recipient pairs. We are looking forward to the topline results expected by the end of 2017."

"We continue our efforts to maximize the potential of our unique BL-8040 oncology platform, with multiple clinical studies for additional indications up and running or expected to start in 2017, including several combination studies with immune checkpoint inhibitors and a registration study in stem-cell mobilization for autologous transplantation," added Mr. Serlin.

The Phase 2 open-label study is conducted in collaboration with the Washington University School of Medicine, Division of Oncology, and will enroll up to 24 donor/recipient pairs, aged 18-70. The trial is designed to evaluate the ability of BL-8040, as a single agent, to promote stem cell mobilization for allogeneic hematopoietic cell transplantation. On the donor side, the primary endpoint of the study is the ability of a single injection of BL-8040 to mobilize sufficient amounts of cells for transplantation following up to two apheresis procedures. On the recipient side, the study aims to evaluate the time to engraftment rate following transplantation of the BL-8040 collected graft.

The study will also evaluate the safety and tolerability of BL-8040 in healthy donors, as well as graft durability, the incidence of grade 2-4 acute and chronic GVHD, and other recipient related parameters in patients who have undergone transplantation of hematopoietic cells mobilized with BL-8040.

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and certain hematological indications. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Stem Cell Mobilization

High-dose chemotherapy followed by hematopoietic cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Modern peripheral stem-cell harvesting often replaces the use of traditional surgical bone marrow stem-cell harvesting. In the modern method, stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), often with the addition of a mobilizing agent such as Plerixafor (Mozobil), harvested from the donor's peripheral blood by apheresis, and infused to the patient after chemotherapy ablation treatment.

An allogeneic hematopoietic cell transplant involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related or unrelated donor. HLA proteins are found on all cells of our body and are the main way the immune system tells the difference between our own cells and foreign cells. The closer the HLA match between a donor and recipient, the greater the chance a transplant will be successful. If the HLA match is not close enough, the donor's immune system, which accompanies the donated stem cells, recognizes the HLA mismatch, and will attack the recipient's tissues. This process is known as graft versus host disease (GVHD).

Approximately 70% of people with a hematological malignancy or bone marrow failure syndrome who need an allogeneic transplant have an HLA-identical sibling or unrelated donor available. For patients who need a stem cell transplant but do not have an HLA-matched related or unrelated donor, recent medical advances have made possible the use of a partially matched or haploidentical related donor. A haploidentical related donor is usually a 50% match to the recipient and may be the recipient's parent, sibling or child.

The advantage of having a haploidentical transplant is thatit increases the chance offinding a donoras almost everyone has at least one haploidentical relative. Relatives can usually be asked to donate stem cells much more quickly than unrelated volunteer donors, particularly when the volunteer donors live in other countries, thereby allowing transplants to be done in a more timely manner.

With improvements in medical treatment, complications of a haploidentical transplant, such as GVHD, rejection of the graft and slow recovery of the immune system appear not to be increased compared to transplants using HLA-matched related or unrelated donors. Since this is a relatively new approach to stem cell transplantation, a haploidentical transplant is a treatment option that is not offered at all treatment centers, but is becoming more common.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on oncology and immunology. The Company in-licenses novel compounds, primarily from academic institutions and biotech companies based in Israel, develops them through pre-clinical and/or clinical stages, and then partners with pharmaceutical companies for advanced clinical development and/or commercialization.

BioLineRx's leading therapeutic candidates are: BL-8040, a cancer therapy platform, which has successfully completed a Phase 2a study for relapsed/refractory AML and is in the midst of a Phase 2b study as an AML consolidation treatment and a Phase 2 study in stem cell mobilization for allogeneic transplantation; and BL-7010 for celiac disease and gluten sensitivity, which has successfully completed a Phase 1/2 study. In addition, BioLineRx has a strategic collaboration with Novartis for the co-development of selected Israeli-sourced novel drug candidates; a collaboration agreement with MSD (known as Merck in the US and Canada), on the basis of which the Company has initiated a Phase 2a study in pancreatic cancer using the combination of BL-8040 and Merck's KEYTRUDA; and a collaboration agreement with Genentech, a member of the Roche Group, to investigate the combination of BL-8040 and Genentech's Atezolizumab in several Phase 1b studies for multiple solid tumor indications and AML.

For additional information on BioLineRx, please visit the Company's website athttp://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated onFacebook,Twitter, andLinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 10, 2016. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contacts: PCG Advisory Vivian Cervantes Investor Relations +1-212-554-5482 vivian@pcgadvisory.com

or

Tsipi Haitovsky Public Relations +972-52-989892 tsipihai5@gmail.com

SOURCE BioLineRx Ltd.

Excerpt from:
BioLineRx Provides Update on Phase 2 Open-Label Study for BL-8040 as Novel Stem Cell Mobilization Treatment - PR Newswire (press release)

To grow clusters of human stem cells that mimic organs in the lab and might be used someday in tissue implants, Bill Murphy, a UW-Madison professor of biomedical engineering, creates tiny scaffolds made of plastic or rubber.

The three-dimensional scaffolds must support the cells and feed them, help them organize and allow them to communicate.

One spring day in 2014, Murphy looked out his office window near UW Hospital, onto the universitys Lakeshore Nature Preserve, and saw a structure that does those very things naturally: plants specifically, cellulose, the main component of the cell walls of green plants.

Now, Murphy and Gianluca Fontana, a UW-Madison post-doctoral fellow with help from Olbrich Botanical Gardens have grown skin, brain, bone marrow and blood vessel cells on cellulose from plants such as parsley, spinach, vanilla and bamboo.

Plants could be an alternative to artificial scaffolds for growing stem cells, the researchers reported Monday in the journal Advanced Healthcare Materials.

Rather than having to manufacture these devices using high-tech approaches, we could literally pick them off of a tree, said Murphy, co-director of the UW-Madison Stem Cell and Regenerative Medicine Center.

The strength, porosity and large surface area of plants could prove superior to making scaffolds using current methods, such as 3-D printing and injection molding, Murphy said.

Plants have a huge capacity to grow cell populations, he said. They can deliver fluids very efficiently to their leaves ... At the microscale, theyre very well organized.

In addition, there are many plants to chose from. After Murphys inspirational gaze out the window, he and Fontana tested plants as scaffolds for stem cells using varieties they could easily obtain: parsley, spinach, jewelweed, water horsetail, summer lilac and, from the UW Arboretum, softstem bulrush.

Then Fontana asked John Wirth, Olbrichs conservatory curator, about other species that might work. Wirth invited Fontana to walk through the tropical greenhouse and take samples back to his lab.

I had never had a request like this before; it made me look at plant material in a different way, Wirth said. I think its a fantastic way of using these pieces of living tissue, to grow human tissue.

Olbrich plants that proved useful include vanilla, bamboo, wasabi, elephant ear, zebra plant and various orchids.

To use plants as scaffolds, the scientists strip away all of the cells, leaving husks of cellulose. Since human cells have no affinity for plants, they add peptides as biological fasteners.

Theyre like grappling hooks for the cells to attach to the plant, Murphy said.

To determine if plant scaffolds could really replace those made of plastic or rubber, the researchers hope to test the cellulose models in animal studies this year.

A major goal of tissue engineering is to develop implants that could regenerate tissue in people to repair bone or muscle damage after traumatic injuries, for example.

It is likely the human body wouldnt reject tissue implants formed on plant scaffolds because the plant cells would be removed, Murphy said.

Were crossing kingdoms, he said. But were optimistic that these materials would be well-tolerated.

Originally posted here:
Borrowing from nature: UW-Madison scientists use plants to grow stem cells - Madison.com

Neuroscientists at the Wisconsin-based Wicab Inc have developed a device called BrainPort that helps blind people see with their tongues. According to the late co-founder of the company, neuroscientist Paul Bach-y-Rita, we see with our brains and not with our eyes, so it should be possible to develop devices that allow the blind to see.

BrainPort involves collecting visual data using a small digital camera that the blind person wears on a pair of sunglasses. The digital optical signals are then converted by a central processing unit (CPU) about the size of a cell phone that the blind person carries in his/her pocket into electrical signals, simulating and replacing the function of the retina. The CPU then sends the signals to sensors on the surface of a lollipop-like device that the blind person carries in the mouth. The nerves on the tongue receive these signals and transmit them to the brain, thereby creating the images of the object being viewed. With a little learning, the user can distinguish between a knife and a fork on the dining table and read letters and numbers and decipher them on the buttons in an elevator.

The device originally announced in 2009 has been tested extensively at the University of Pittsburgh Medical Centres UPMC Eye Centre and is now commercially available. In a subsequent development, the images can be transferred to the brain by an arm band through the nervous system in the arms. This avoids the use of lollipop devices.

Another way to restore vision for the blind has been developed by Prof. Michael Beauchamp at the University of Texas. He is exploring the possibility of electrically stimulating the visual cortex of the brain by means of electrical implants. He also believes that we see not with our eyes but with our brains and if electrical images generated from the visual objects can be transferred to the correct region of the brain, vision can be restored. About 10 percent of the blind people experience vivid hallucinations. This is attributed to the hyperactivity of the visual cortex of the brain and the images produced can be seen in exquisite detail. It is envisaged that a webcam fitted on the glasses of the blind person could be connected to an implant in the brain to restore vision.

The ability to record brain activity while seeing an image, and then play it back to reconstruct that image has been a matter of pure science fiction until now. Scientists working at the University of California, Berkeley, have succeeded in reconstructing visual images after recording the brain activity of people watching movie trailers. The scientists were able to see what peoples brains were seeing. They used a functional Magnetic Resonance Imaging (fMRI) scanner to record the flow of blood in certain parts of the brain. Using powerful computing techniques, it was possible to correlate the visual images with corresponding brain activities. This allowed the images to be reconstructed. The researchers hope to eventually read the thoughts of patients in a coma or those suffering from a paralysis after a stroke. They can even apply these techniques on spies who are trying to hide information. Researchers have now also succeeded in reconstructing words by detecting peoples corresponding brain activity.

Another area of intense research activity is that of regenerative medicine that involves the growth of human cells and tissues. Indeed stem cell therapy is heralding the advent of a revolution in medicine to repair damaged kidney and heart cells and to treat diabetes and other diseases. Stem cells can be differentiated into different types of specialised cells (heart, kidney, pancreas etc). Adult stem cell therapies have been used for a long time to treat leukaemia and other cancers by bone marrow transplants.

Now a special bandage, seeded with stem cells, has been developed by scientists at the Bristol University in the UK to repair cartilage tears that are otherwise difficult to heal. The bone marrow is extracted from the hip of the patient with a needle. Stem cells are obtained from it and multiplied separately before being embedded into a special membrane/bandage which is inserted into the torn cartilage. The stem cells present on the membrane are expected to help the healing process. The procedure can help repair meniscus tears that are particularly common in athletes.

Ink jet printers are commonly used for printing documents. An astounding breakthrough has been made by doctors at the Wake Forest Institute of Regenerative Medicine in the US where a device that resembles an ink jet printer can be used to spray new skin cells on to burn wounds. This method results in rapid healing and can eventually replace the need for having skin grafts. The device resembles a colour ink jet printer and comprises a tank that contains skin cells, stem cells and nutrients. These are sprayed by a computer controlled nozzle directly on to the burnt area. In animal experiments, the wounds in mice were fully healed within two weeks using this technique compared to the five-week period that skin graft procedures took. The ink jet printer method also showed less scarring and better hair regeneration. The technology is being employed by the US army to print-shut bullet wounds and blast damage.

Magicians have been practising the art of making objects disappear for centuries. Now, science can take on that role. In 2006, Prof. John Pendry and his colleagues proposed the design of a cloak that could steer light around an object, making it invisible. Soon thereafter Dr David Smith at Duke University made a cloaking device that used certain metamaterials that had unusual electromagnetic properties. The invisible threads of these metamaterials are made of components smaller than the wavelength of light. This allows them to bend light waves and impart optical properties that are not present in normal substances. Computer models indicate that such threads should not be thicker than a micrometre. When these carpet cloaks are placed over an object, the object becomes invisible.

The technology has applications in defence: it may allow soldiers, weapons, warships and planes to appear invisible. Harry Potters cloak of invisibility is fast becoming a reality. Invisible armies, ships, planes and submarines cloaked by metamaterials seem like a possibility in the near future.

Countries investing in these cutting edge researches are making billions of dollars through such entrepreneurial ventures. If Pakistan is to prosper, we must give the highest national priority to education, science, technology, innovation and entrepreneurship. This requires a visionary government that understands the critical role of a knowledge-based economy in the rapidly changing world of today.

The writer is chairman of UN ESCAP Committee on Science Technology & Innovation and former chairman of the HEC.

Email: [emailprotected]

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Disruptive innovations - The News International

Testing the efficacy of new gene therapies more efficiently ... Science Daily Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and ... and more »

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Testing the efficacy of new gene therapies more efficiently ... - Science Daily

By Staff

Electroacupuncture triggers a neurological response that releases stem cells that can relieve injury-induced pain, and help promote tissue repair, says a study in the journal Stem Cells led by Indiana University School of Medicine.

The school outlined its findings in a media release:

Electroacupuncture is a form of acupuncture that uses a small electrical current to augment the ancient Chinese medical practice of inserting fine needles into the skin at pre-determined points throughout the body.

For the study, a team of more than 40 scientists at institutions in the United States and South Korea was led by four senior authors including IU School of Medicines Maria B. Grant, MD, Marilyn Glick Professor of Ophthalmology and co-corresponding author; Mervin C. Yoder, MD, IU Distinguished Professor, Richard and Pauline Klingler Professor of Pediatrics, associate dean for entrepreneurial research at IU School of Medicine, director of the Herman B Wells Center for Pediatric Research and co-corresponding author; and Fletcher A. White, PhD, Vergil K. Stoelting Chair ofAnesthesia, professor of anesthesia, pharmacology and toxicology.

This work is a classic example of the power of team science, where investigators in different institutions with specific expertise worked together to unravel the complexity of how electroacupuncture works to help the body respond to stressors, said Dr. Yoder.

The researchers performed a series of lab tests involving humans, horses and rodents that follow the effects of electroacupuncture from the stimulus of the needle all the way to the brain, resulting in the release of reparative mesenchymal stem cells (MSCs) into the bloodstream.

Depending on the species, electroacupuncture led to activation of the hypothalamusa part of the brain that controls the nervous system and involuntary bodily functions such as heart rate and digestionwithin nine to 22 minutes. The stem cells were mobilized within two hours.

The acupuncture stimulus were giving these animals has a rapid effect on neuroanatomical pathways that connect the stimulus point in the arm to responsive neurons in the spinal cord and into a region in the brain called the hypothalamus. In turn, the hypothalamus directs outgoing signals to stem cell niches resulting in their release, said Dr. White, who is a neuroscientist at the Richard L. Roudebush VA Medical Center in Indianapolis.

The researchers found electroacupuncture treatments resulted in higher thresholds for injury-induced pain, as well as considerable increases in the presence of a type of collagen that promotes tendon repair and anti-inflammatory cells known to be predictors of faster healing time.

Dr. White said these findings could lead to new strategies for tissue repair and pain management related to injuries.

We could potentially capture the MSCs from an individuals blood following electroacupuncture and save the cells for future re-introduction in the patient post-surgery or to treat chronic pain due to an injury, he said.

The horses used in the study had been injured during training for international dressage competitions, and the six people who took part were healthy volunteers, who still showed activation of their hypothalamus through brain imaging.

See more here:
Electroacupuncture Releases Stem Cells to Relieve Pain and More, Study Finds - National Pain Report

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Cesca Therapeutics Inc. (NASDAQ: KOOL), a market leader in automated cell processing and point-of-care, autologous cell-based therapies, today announced the publication in a peer reviewed journal of data from its Critical Limb Ischemia (CLI) feasibility study utilizing the Companys innovative point-of-care technology. The report was published in the Stem Cells International and is available online at https://www.hindawi.com/journals/sci/2017/4137626/ref/.

Results from the seventeen patient clinical study titled, Safety and Effectiveness of Bone Marrow Cell Concentrate in the Treatment of Chronic Critical Limb Ischemia Utilizing a Rapid Point-of-Care System, (the Study) were obtained using Cescas automated point-of-care technology. The single treatment procedure was performed at the patients bedside and took less than 60 minutes. The Study results showed significant improvement in wound healing, rest pain and six-minute walking distance, along with significant reduction in intermittent claudication pain following the treatment.

Dr. Venkatesh Ponemone, Study Director and Executive Director of TotipotentRX, a Cesca subsidiary and the corresponding author of the article commented, We are targeting difficult to treat or life threatening conditions such as CLI with our autologous, cell-based therapies. We believe our innovative point-of-care cell processing systems, such as those used in the Study, can play an important role in optimizing the quality and quantity of target cells used to improve patient outcomes.

Dr. Xiaochun "Chris" Xu, Cesca's Interim CEO added, We are pleased that the Study was recognized and published in a peer reviewed journal. The encouraging data highlights Cescas capability to develop effective automated cellular processing systems. We welcome strategic partners to help us further refine their use in larger clinical settings.

About Cesca Therapeutics Inc.Cesca Therapeutics Inc. (www.cescatherapeutics.com) is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics. These include:

Forward-Looking StatementThe statements contained herein may include statements of future expectations and other forward-looking statements that are based on managements current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

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Cesca Therapeutics (KOOL) Announces CLI Feasibility Study Published in Stem Cells International - StreetInsider.com

The right bone marrow donor match has been hard to find for one 2-year-old.

Shern-Min Chow, KHOU 6:49 PM. CDT March 20, 2017

The right bone marrow donor match for this 2-year-old has been hard to find. (Photo: KHOU)

HOUSTON - Roman Shen is a happy 2-year-old. You wouldn't know it, but he suffers from Shwachman Diamond Syndrome, or SDS. That means his bone marrow doesn't work properly and doesnt make its own white blood cells.

The right bone marrow donor match has been hard to find.

Mostly because he is mixed race, Chinese-Italian, and they are underrepresented in the bone marrow registry," said Nicole Shen, his mother.

Mixed race people are 4 percent of the bone marrow registry. So for the past 2 years, Romans parents have been actively involved with the registry, launching and participating in Be the Match registration drives.

It takes about 10 minutes. You swab the inside of your cheek. You could save my son's life or someone else's life," Nicole Shen said.

Why is race so important to marrow donation?

You're matching antigens that are produced by your stem cells and those are in many cases matched to ethnicity," saidBe The Match spokeswoman FeliciaGann.

One in 540 registrants will end up being a match. Donors give either with platelets, which are given much like a blood donation or with actual bone marrow, removed from the hip bone via needle. Bone marrow transplants are used to treat over 70 diseases.

The Gulf Coast Regional Blood Center houses the Gulf Coast Marrow Donor Program (GCMDP) and is an accredited donor center for the National Marrow Donor Program (NMDP) which operates the Be The Match Registry. For more information, contact the GCMDP at (713)-791-6697.

To register as a potential marrow donor, you must be 18 to 44 years old and in general good health. Registration takes 10 minutes and involves completing a form and a cheek swab with a Q-tip.

You may register at any of the Gulf Coast Regional Blood Center Locations onlineor arrange for representatives come to you by emailing fgann@giveblood.org.

2017 KHOU-TV

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Family of 2-year-old hopes to find bone marrow donor - KHOU.com

Tri-City Herald

Retired Richland sergeant battling rare blood disease, awaiting marrow transplant Tri-City Herald People who are healthy, between 18 and 44, and want to register as a bone marrow/stem cell donor can do it at join.bethematch.org/hope4sarge or join.bethematch.org/hopeforsarge. A swab kit will be mailed to their home with instructions and a confirmation.

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Retired Richland sergeant battling rare blood disease, awaiting marrow transplant - Tri-City Herald

At SXSW, One Panel Discussion Centered On Potentially Life-Saving Mission Patch.com Many patients fighting blood cancer and other blood diseases like sickle cell anemia can be saved with a bone marrow or stem cell transplant. Thirty percent of all patients needing transplants find a compatible donor within their family, but 70 ...

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At SXSW, One Panel Discussion Centered On Potentially Life-Saving Mission - Patch.com

To grow clusters of human stem cells that mimic organs in the lab and might be used someday in tissue implants, Bill Murphy, a UW-Madison professor of biomedical engineering, creates tiny scaffolds made of plastic or rubber.

The three-dimensional scaffolds must support the cells and feed them, help them organize and allow them to communicate.

One spring day in 2014, Murphy looked out his office window near UW Hospital, onto the universitys Lakeshore Nature Preserve, and saw a structure that does those very things naturally: plants specifically, cellulose, the main component of the cell walls of green plants.

Now, Murphy and Gianluca Fontana, a UW-Madison post-doctoral fellow with help from Olbrich Botanical Gardens have grown skin, brain, bone marrow and blood vessel cells on cellulose from plants such as parsley, spinach, vanilla and bamboo.

Plants could be an alternative to artificial scaffolds for growing stem cells, the researchers reported Monday in the journal Advanced Healthcare Materials.

Rather than having to manufacture these devices using high-tech approaches, we could literally pick them off of a tree, said Murphy, co-director of the UW-Madison Stem Cell and Regenerative Medicine Center.

The strength, porosity and large surface area of plants could prove superior to making scaffolds using current methods, such as 3-D printing and injection molding, Murphy said.

Plants have a huge capacity to grow cell populations, he said. They can deliver fluids very efficiently to their leaves ... At the microscale, theyre very well organized.

In addition, there are many plants to chose from. After Murphys inspirational gaze out the window, he and Fontana tested plants as scaffolds for stem cells using varieties they could easily obtain: parsley, spinach, jewelweed, water horsetail, summer lilac and, from the UW Arboretum, softstem bulrush.

Then Fontana asked John Wirth, Olbrich's conservatory curator, about other species that might work. Wirth invited Fontana to walk through the tropical greenhouse and take samples back to his lab.

I had never had a request like this before; it made me look at plant material in a different way, Wirth said. I think its a fantastic way of using these pieces of living tissue, to grow human tissue.

Olbrich plants that proved useful include vanilla, bamboo, wasabi, elephant ear, zebra plant and various orchids.

To use plants as scaffolds, the scientists strip away all of the cells, leaving husks of cellulose. Since human cells have no affinity for plants, they add peptides as biological fasteners.

Theyre like grappling hooks for the cells to attach to the plant, Murphy said.

To determine if plant scaffolds could really replace those made of plastic or rubber, the researchers hope to test the cellulose models in animal studies this year.

A major goal of tissue engineering is to develop implants that could regenerate tissue in people to repair bone or muscle damage after traumatic injuries, for example.

It is likely the human body wouldn't reject tissue implants formed on plant scaffolds because the plant cells would be removed, Murphy said.

Were crossing kingdoms, he said. But were optimistic that these materials would be well-tolerated.

Read more:
Borrowing from nature: UW-Madison scientists use plants to grow ... - La Crosse Tribune

March 20, 2017 by Melissa Gaskill Cultured stem cells. Credit: BioServe Inc., University of Colorado

Growing significant numbers of human stem cells in a short time could lead to new treatments for stroke and other diseases. Scientists are sending stem cells to the International Space Station to test whether these cells proliferate faster in microgravity without suffering any side effects.

Therapeutic uses require hundreds of millions of stem cells and currently no efficient way exists to produce such quantities. Previous research suggests that microgravity could help, and the space station is home to the nation's only national lab in microgravity.

Some types of stem cells grow faster in simulated microgravity, according to Abba Zubair, a researcher at the Mayo Clinic in Jacksonville, Florida. Zubair is principal investigator for the Microgravity Expanded Stem Cells investigation, which is cultivating human stem cells aboard the space station for use in clinical trials back on Earth. He holds a doctor of medicine degree in transfusion medicine and cell therapy and a doctorate of philosophy in tumor immunology.

Human stem cells are cells that have not yet specialized in function and can divide into a spectrum of cell types, rejuvenating and repairing tissue throughout a person's lifetime. Stem cells in every organ of the body, including skin and bones, maintain those organs and repair tissue by dividing and differentiating into specialized cells.

Harvesting a person's stem cells and growing enough of them for use in therapies has proven difficult, though. Researchers have successfully grown mesenchymal stem cells, found in bone marrow, but growing sufficient quantities takes weeks. That could be too late for treatment of some conditions.

"Stem cells are inherently designed to remain at a constant number," Zubair explains. "We need to grow them faster, but without changing their characteristics."

The first phase of the investigation, he adds, is answering the question: "Do stem cells grow faster in space and can we grow them in such a manner that they are safe to use in patients?"

Investigators will examine the space-grown cells in an effort to understand the mechanism behind microgravity's effects on them. The long-term goal is to learn how to mimic those effects and develop a safe and reliable way to produce stem cells in the quantities needed.

The second phase will involve testing clinical application of the cells in patients. Zubair has been studying treatment of stroke patients with lab-grown stem cells and plans to compare those results with use of the space-grown stem cells.

"What is unique about this investigation is that we are not only looking at the biology of the cells and how they grow, but focusing on application, how we can use them to treat patients," he says.

The investigation expands existing knowledge of how microgravity affects stem cell growth and differentiation as well as advances future studies on how to produce large numbers of stem cells for treating stroke and other conditions.

The faster that happens, the better for those who could benefit from stem cell therapies.

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An international team of researchers, funded by Morris Animal Foundation, has shown that adipose (fat) stem cells might be the preferred stem cell type for use in canine therapeutic applications, including orthopedic diseases ...

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Stem cells seem speedier in space - Phys.Org

In Brief

Macular degeneration affects more than 10 million people in the U.S., and is the most common cause of vision loss. It is caused by the deterioration of the middle of the retina, called the macula. The macula focuses central vision and controls our ability to see objects in fine detail, read, recognize colors and faces, and drive a car. Until now, the disease has been considered incurable.

An octogenarian with the condition is now the first person to receivesuccessful treatmentwith induced pluripotent stem (iPS) cells. The progression of the womans macular degenerationwas arrested by new retinal cells made in the lab.Unlike embryonic stem cells, iPS cells can be created from regular adult cells.In this case, the cells used to repair the damaged retina from macular degeneration came from the womansskin.

The team at Kobe, Japans RIKEN Laboratory for Retinal Regeneration, led by Masayo Takahashi, created iPS cells from the patients skin cells. Then, theyencouraged them to form cells to patch the retinal pigment epithelium. These cells help nourish and support the retina, allowing it to capture the light the eye needs to see.

Once the cells were transformed, the team used them to make a slither measuring 1 by 3 millimeters. This was the patch they used to replace the diseased tissue removed from the patients retina. Their aim was to stop the degeneration and save her sight. The results show that the procedure was technically a success: although her vision did not improve, the degeneration stopped.

A possible concern about this treatment, however, is that creating new tissues from stem cells could cause genetic mutations, which might in turnlead to cancer. While more research in this area and its possible applications is needed, in the case of the patient at RIKEN, therehave been no signs of cancer or any other complications.

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A Groundbreaking Stem Cell Treatment Just Prevented a Woman From Going Blind - Futurism

A discovery, several years in the making, by a University at Buffalo research team has proven that adult skin cells can be converted into neural crest cells (a type of stem cell) without any genetic modification, and that these stem cells can yield other cells that are present in the spinal cord and the brain.

The applications could be very significant, from studying genetic diseases in a dish to generating possible regenerative cures from the patients own cells.

Its actually quite remarkable that it happens, says Stelios T. Andreadis, PhD, professor and chair of UBs Department of Chemical and Biological Engineering, who recently published a paper on the results in the journal Stem Cells.

The identity of the cells was further confirmed by lineage tracing experiments, where the reprogrammed cells were implanted in chicken embryos and acted just as neural crest cells do.

Stem cells have been derived from adult cells before, but not without adding genes to alter the cells. The new process yields neural crest cells without addition of foreign genetic material. The reprogrammed neural crest cells can become smooth muscle cells, melanocytes, Schwann cells or neurons.

In medical applications this has tremendous potential because you can always get a skin biopsy, Andreadis says. We can grow the cells to large numbers and reprogram them, without genetic modification. So, autologous cells derived from the patient can be used to treat devastating neurogenic diseases that are currently hampered by the lack of easily accessible cell sources.

The process can also be used to model disease. Skin cells from a person with a genetic disease of the nervous system can be reprogrammed into neural crest cells. These cells will have the disease-causing mutation in their chromosomes, but the genes that cause the mutation are not expressed in the skin. The genes are likely to be expressed when cells differentiate into neural crest lineages, such as neurons or Schwann cells, thereby enabling researchers to study the disease in a dish. This is similar to induced pluripotent stem cells, but without genetic modification or reprograming to the pluripotent state.

The discovery was a gradual process, Andreadis says, as successive experiments kept leading to something new. It was one step at a time. It was a very challenging task that took almost five years and involved a wide range of expertise and collaborators to bring it to fruition, Andreadis says. Collaborators include Gabriella Popescu, PhD, professor in the Department of Biochemistry in the Jacobs School of Medicine and Biomedical Sciences at UB; Song Liu, PhD, vice chair of biostatistics and bioinformatics at Roswell Park Cancer Institute and a research associate professor in biostatistics UBs School of Public Health and Health Professions; and Marianne Bronner, PhD, professor of biology and biological engineering, California Institute of Technology.

Andreadis credits the persistence of his then-PhD student, Vivek K. Bajpai, for sticking with it.

He is an excellent and persistent student, Andreadis says. Most students would have given up. Andreadis also credits a seed grant from UBs office of the Vice President for Research and Economic Developments IMPACT program that enabled part of the work.

The work recently received a $1.7 million National Institutes of Health grant to delve into the mechanisms that occur as the cells reprogram, and to employ the cells for treating the Parkinsons-like symptoms in a mouse model of hypomyelinating disease.

This work has the potential to provide a novel source of abundant, easily accessible and autologous cells for treatment of devastating neurodegenerative diseases. We are excited about this discovery and its potential impact and are grateful to NIH for the opportunity to pursue it further, Andreadis said.

The research, described in the journal Stem Cells under the title Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates, was supported by grants from the National Institutes of Health.

Reference:

Bajpai, V. K., Kerosuo, L., Tseropoulos, G., Cummings, K. A., Wang, X., Lei, P., Liu, B., Liu, S., Popescu, G. K., Bronner, M. E. and Andreadis, S. T. (2017), Reprogramming Postnatal Human Epidermal Keratinocytes Toward Functional Neural Crest Fates. Stem Cells. doi:10.1002/stem.2583

This article has been republished frommaterialsprovided by University at Buffalo. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Stem Cells Reprogrammed without Genetic Modification - Technology Networks

March 13, 2017 02:00 ET | Source: TiGenix NV

multilang-release

PRESS RELEASE REGULATED INFORMATION INSIDE INFORMATION

TiGenix Announces Top-Line Phase I/II Results of AlloCSC-01 in Acute Myocardial Infarction

Leuven (BELGIUM) - March 13, 2017, 07:00h CET - TiGenix NV (Euronext Brussels and Nasdaq: TIG), an advanced biopharmaceutical company focused on developing novel therapeutics from its two proprietary platforms of donor-derived expanded adipose derived stem cells (eASC) and donor-derived expanded cardiac stem cells (AlloCSCs), today announced top-line one-year results from the CAREMI clinical trial, an exploratory Phase I/II study of AlloCSCs in acute myocardial infarction (AMI).

CAREMI is the first-in-human clinical trial with the primary objective being safety and evaluating the feasibility of an intracoronary infusion of 35 million of AlloCSCs in patients with AMI and left ventricular dysfunction treated within the first week post-AMI. Importantly, the trial is the first cardiac stem cell study to integrate a highly discriminatory magnetic resonance imaging (MRI) strategy to select patients at increased risk of heart failure and late adverse outcomes. CAREMI was not powered to establish efficacy therefore no conclusion can be drawn on the secondary efficacy end-points.

The main findings of this study are:

"This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree, and in the worst possible setting represented by patients with an acute heart attack with left ventricular dysfunction," commented Professor Fernndez-Avils, Head of the Department of Cardiology at the Hospital General Universitario Gregorio Maran in Madrid (Spain), principal investigator on the trial in Spain. "It is especially encouraging that no cardiac or immunological side effects were observed."

"This is the first study in which we have used a state of the art comprehensive MRI analysis to include patients with a large myocardial infarction in an innovative cell therapy protocol," said Professor Janssens, Head of the Department of Cardiovascular Diseases, University Hospital, Leuven (Belgium), and principal investigator on the trial in Belgium. "Serial MRI analysis and extensive immunological profiling will allow us to further explore the encouraging signals we observed in cell treated patients with the worst MRI signature. These findings offer an exciting prospect for targeted follow-up studies in these high-risk patients."

"Besides confirming the long term safety of the treatment these results suggest interesting opportunities in populations with high unmet medical need," said Dr. Marie Paule Richard, Chief Medical Officer at TiGenix. "We look forward to working with our advisors to analyze the data in depth and determine the best way forward with AlloCSC-01 during the second half of this year."

Full data results from the CAREMI study will be presented at an upcoming medical congress.

###

For more information

Claudia D'Augusta Chief Financial Officer

T: +34 91 804 92 64

claudia.daugusta@tigenix.com

About TiGenix

TiGenix NV (Euronext Brussels and Nasdaq: TIG) is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic, or donor-derived, expanded stem cells. Two products from the adipose-derived stem cell technology platform are currently in clinical development: Cx601 in Phase III for the treatment of complex perianal fistulas in Crohn's disease patients; Cx611 which has completed a Phase I sepsis challenge trial and a Phase I/II trial in rheumatoid arthritis. Effective July 31, 2015, TiGenix acquired Coretherapix, whose lead cellular product, AlloCSC-01, has concluded a Phase II clinical trial in Acute Myocardial Infarction (AMI). In addition, the second product candidate from the cardiac stem cell-based platform acquired from Coretherapix, AlloCSC-02, is being developed in a chronic indication. On July 4, 2016, TiGenix entered into a licensing agreement with Takeda, a large pharmaceutical company active in gastroenterology, under which Takeda acquired the exclusive right to commercialize Cx601 for complex perianal fistulas outside the United States. TiGenix is headquartered in Leuven (Belgium) and has operations in Madrid (Spain). For more information, please visit http://www.tigenix.com.

About AlloCSC-01

AlloCSC-01 is a cellular product consisting of adult expanded allogeneic cardiac stem cells isolated from the right atrial appendages of donors, and expanded in vitro. Pre-clinical data has shown evidence of the strong cardio-protective and immune-regulatory activity of AlloCSC-01. In vivo studies suggest that AlloCSC-01 has cardio-reparative potential by activating endogenous regenerative pathways and by promoting the formation of new cardiac tissue. In addition, AlloCSC-01 has displayed a strong tropism for the heart enabling a high retention of cells in the myocardium after intracoronary administration.

About CAREMI

The CAREMI trial comprised two consecutive phases: an open-label dose-escalation phase (n=6) and a 2:1 randomized, double-blind, placebo-controlled phase (n=49). The objective of this clinical trial is to evaluate the safety and the efficacy of the cardiac stem cells product AlloCSC-01 in the acute phase of ischemic heart disease. The primary safety endpoint are all-cause mortality within 30 days and percentage of patients with major adverse cardiac events (MACE) within 30 days after treatment. MACE is a broader safety endpoint that covers all-cause mortality as well as new AMI, hospitalization due to heart failure, sustained ventricular tachycardia, ventricular fibrillation and stroke. Secondary safety endpoints include percentage of patients with MACE at 6 and 12 months after treatment, all-cause mortality at 12 months after treatment and percentage of patients with AE during the study. Secondary efficacy include MRI parameters (evolution of infarct size and evolution of biomechanical parameters) and clinical parameters (including the 6 minute walking test and the New York Heart Association scale). The CAREMI study has been conducted at the Hospital General Universitario Gregorio Maraon, Madrid, UZ Leuven, Hospital de Navarra, Hospital Clnico Universitario de Valladolid, Hospital Universitario de Donostia, Hospital Universitario de Salamanca, Hospital Clnico Universitario de Valencia, and Hospital Virgen de la Victoria de Mlaga. The CAREMI trial has benefitted from the support of the CARE-MI consortium (Grant Number 242038, http://www.caremiproject.eu/) funded by the Seventh Framework Programme of the European Commission under the coordination of the Centro Nacional the Investigaciones Cardiovasculares (CNIC) and the participation of research institutions and companies from nine EU countries.

Forward-looking information

This press release may contain forward-looking statements and estimates with respect to the anticipated future performance of TiGenix and the market in which it operates. Certain of these statements, forecasts and estimates can be recognised by the use of words such as, without limitation, "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will" and "continue" and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond the Company's control. Therefore, actual results, the financial condition, performance or achievements of TiGenix, or industry results, may turn out to be materially different from any future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of the publication of this press release. TiGenix disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in the Company's expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by Belgian law.

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TiGenix Announces Top-Line Phase I/II Results of AlloCSC-01 in Acute Myocardial Infarction - GlobeNewswire (press release)

It's a long road ahead for Madalayna Ducharme after her stem cell transplant Friday.

The seven-month-old baby was diagnosed with malignant infantile osteopetrosis, which stunts growth, impairs vision and hearing and if untreated can be fatal.

The girl's family put out a plea in January to try to find a stem cell donor.

It took a few months, with 1,300 people attending swab events and a match was found.

The family posted on Facebook that The best agreed upon opportunity/match for us after many tests and consultations is our son Henrik.

The 2-year-old Henrik had a bone marrow harvest Friday. The family says he had surgery to remove bone marrow from both hips. Madalyna later received her brother's bone marrow in the form of a blood transfusion.

Doctors told the family the surgery went well.

Henrik has since been discharged and is recovering at the Ronald Mcdonald House.

For Madalayna, it's a matter of wait and see to find out if the marrow going into her bones will work as her own.

It will take between 14 and 27 days before doctors can tell if the transfusion was successful.

In the Facebook post, the family thanked everyone for their thoughts and prayers and asked people to continue to think about the family.

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Baby's stem cell transplant goes well, doctors say - CTV News

By FPJ Bureau|Mar 19, 2017 12:05 am

Mumbai : A Bone Marrow Transplant (BMT) and Birthing Centre was launched by actor Shah Rukh Khan on Friday at Nanavati Super Speciality Hospital (NSSH) at Vile Parle. On the occasion, Khan told the people present to take their medicines on timeand do regular check ups and get rid of ailments.

BMT is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Bone marrow is the soft, fatty tissue inside our bones which produces blood cells. Stem cells are immature cells in the bone marrow that give rise to different blood cells.

Dr Nimish Kulkarni, Associate Consultant of BMT, said, BMT are considered the last-mile treatment solutions for patients with blood and cancer disorders. We specialise in providing transplant services for benign hematological disorders like Thalassemia, Sickle cell disease, Aplastic anemia, bleeding disorders and coagulation disorders. Shah Rukh said, I have been associated with Nanavati Hospital before 25 years ago as a patient. Every year more than 10,000 people are dying of cancer in India, even my parents succumbed to cancer. I am glad now we have a world class treatment.

Dr. Ali has looked after me and my various injuries. He has looked after my sister and wife too. Few people know that when my third son, Abram, was born, he was in a very critical condition. He was rushed to the Paediatric centre here and looked after even before we could meet him. Its a strange cycle that my child was saved in the same hospital which has a ward named after my mother. I am thankful to them, Khan added. Kulkarni added, The specialisation also extends to providing advanced transplant treatments for malignant hematological disorders like acute and chronic leukemia, Hodgkins and Non-Hodgkins Lymphoma, Multiple Myeloma, Myelodysplasia,

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SRK launches BMT, Birthing Centre at Nanavati hospital - Free Press Journal

Studying brain disorders is complicated for many reasons, not the least being the ethics of obtaining living neurons. To overcome that obstacle, UC San Francisco postdoc Aditi Deshpande, PhD, is starting with skin cells.

Thanks to developments in stem cell technology, new information about the human brain is now being gleaned from a simple cheek swab or skin sample. This technology is key to the kind of progress Despande and researchers like her are making. It allows them to work with cells otherwise unobtainable living brain cells that have the same genetics as the patients.

Deshpande begins with skin cells obtained from the Simons Foundation from volunteers whose DNA contains a specific deletion or duplication of one chromosome. She cultures these cells and then turns them into induced pluripotent stem cells cells that have been coaxed back to their embryonic state and are able to become any other type of cell. From there, she reprograms them to become a specific type of neuron thats involved in attention and information processing.

The deletion or duplication Deshpande is looking for stems from a 2008 finding by Lauren Weiss, PhD, an associate professor of neurology in the UCSF Department of Psychiatry and the UCSF Institute for Human Genetics.

Weiss discovered a 29-gene region of DNA on chromosome 16 that is associated with autism, seizures and other brain disorders. Normally, a person has two copies of the region one on each copy of chromosome 16. In some of Deshpandes samples, the region is deleted from one chromosome, leaving one copy. In others, the region is duplicated, resulting in three copies. Subjects with only one copy of the region were more likely to have macrocephaly an enlarged brain than a typical subject, and those with three copies were more likely to have microcephaly a smaller brain.

Whats really interesting, said Deshpande, is that although these subjects seem to have opposite features in terms of brain size, we see a related effect, based on whether they have fewer or more copies of the region.

Some known models of autism show a connection between a neurons growth or appearance and macrocephaly, she explained. We wanted to know if the same thing is happening here.

To compare the effect of the mutation, Deshpande first stains the obtained skin cells so that she can visualize the neurons under a microscope. After staining, Deshpande used cell-counting software to assess several thousands of neurons from deletion and duplication samples and measure them against normal neurons. She found that the neurons missing the DNA region exhibited some differences compared to typical neurons.

Her next step in her research is to discern which of the regions 29 genes are involved in these differences.

The work is meticulous, but Deshpande doesnt mind. I simply love looking at neurons, she said. It really makes you appreciate the complexity of the brain.

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Science in Focus: Creating Neurons from Skin Cells to Understand ... - UCSF News Services

iPS cells may help halt failing vision

Getty

By Andy Coghlan

A woman in her 80s has become the first person to be successfully treated with induced pluripotent stem (iPS) cells. A slither of laboratory-made retinal cells has protected her eyesight, fighting her age-related macular degeneration a common form of progressive blindness.

Such stem cells can be coaxed to form many other types of cell. Unlike other types of stem cell, such as those found in an embryo, can be made from adult non-stem cells a discovery that in 2012.

Now, more than a decade after they were created, these stem cells have helped someone. at the RIKEN Laboratory for Retinal Regeneration in Kobe, Japan, and her team took skin cells from the woman and turned them into iPS cells. They then encouraged these to form retinal pigment epithelial cells, which are important for supporting and nourishing the retina cells that capture light for vision.

The researchers made a slither of cells measuring just 1 by 3 millimetres. Before in 2014, they first removed diseased tissue on her retina that was gradually destroying her sight. They then inserted the small patch of cells they had created, hoping they would become a part of her eye and stop her eyesight from degenerating.

Now the results are in. Published today, they show that the treatment hasnt made the womans vision any sharper, but it does seem to have prevented further deterioration with her vision now stable for more than two years. Since the graft, the woman says her vision is brighter.

Takahashi and her team have done incredible work, and deserve all the praise they get for this project, says , director of the Center for iPS Cell Research and Application at Kyoto University, who won the Nobel prize for and collaborated on this work. This is a landmark study and opens the door to similar treatments for many diseases, he says.

This first iPSC-derived retinal graft is an important landmark in the field of retinal regeneration, says at University College London, and head of a trial at Moorfields Eye Hospital in London of similar grafts made instead from human embryonic stem cells.

One worry about this approach is that turning the stem cells into new tissues could lead to cancer-causing genetic mutations though the team found no evidence of this in the treated woman. However, a trial of the technique in another person was cancelled in 2015, after tests revealed that the cells intended to be given to the man had developed genetic abnormalities.

But although it has taken many years to bring , many private centres around the world have been advertising unregulated treatments purporting to use stem cells for some time.

A second study published today shows just how badly some unregulated treatments described as stem cell therapies can go wrong. Three case reports of women given such treatments for age-related macular degeneration detail how one woman went blind and the vision of the other two became much worse.

All three ended up seeking emergency treatment in 2015, after each paid $5000 to a private clinic to receive injections of their own fatty tissue into their eyes.

Patients and physicians in the US should be made aware that not all stem cell clinics are safe, and that stem therapy as provided in private clinics in the US is unproven and potentially harmful, says at the University of Miamis Bascom Palmer Eye Institute, Florida, who subsequently treated two of the women.

Albini advises people to be suspicious of any procedure involving payment. Most legitimate research in the US does not require patients to pay for the experimental procedures, he says, adding that people should check whether a trial has been registered with the US Food and Drug Administration. Be aware that if it sounds too good to be true, it may indeed not be true.

Journal reference: New England Journal of Medicine, DOI: ;

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Vision saved by first induced pluripotent stem cell treatment ... - Concord Register