University researchers have developed a new testing system that can improve care for patients who need bone marrow and stem cell transplants.

Graft-versus-host disease is a life-threatening condition that can occur in response to transplants. GVHD causes immune cells from the transplant to attack the bodys healthy tissue. In patients with diseases such as leukemia, which compromises the bodys immune system, bone marrow or stem cell transplants are necessary.

John Levine, professor of pediatrics and the study's lead author, said in these types of cases, GVHD is a real danger.

Following transplantation surgeries, our major concern is the development of GVHD in our patients, Levine said. However, it is difficult to predict the severity of GVHD at the onset of the symptoms as it varies from patient to patient.

Prior to the research, there was no method for determining the severity of a GVHD case and whether or not it needed treatment. The treatment involves high doses of medication that reduce immune activity, so doctors must be extremely cautious when treating GVHD. Levine and his co-investigators assessed nearly 800 patients and created a scoring system that uses three proteins to assess the severity of each case of the disease.

We found out that it was not one protein but a combination of three recently validated biomarkers TNFR1, ST2, and Reg3, Levine said. We then formulated an equation which computes the concentration of the biomarkers into three Ann Arbor scores. The scores are positively correlated with the amount of risk the diagnosed patient is in, so a score 1 indicates a patient with minimal risk while a patient diagnosed with a score of 3 will subjected to intensive primary therapy.

The Ann Arbor scoring system will help ensure patients at lower risk are subjected to less aggressive treatments than patients at higher risk. Patients will then gain individualized treatments based on their needs.

More than half of the patients undergoing bone marrow transplantation develop GVHD. Though the degree of severity differs in patients, the disease is highly lethal if not treated immediately.

The research began in the late 1990s when investigators analyzed blood samples from 500 GVHD patients. The results were verified when another 300 patient blood samples from across the United States were analyzed.

The next step, according to Levine, is the launch of a clinical trial. The U.S. Food and Drug Administration has approved this step.

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The Villages, Florida (PRWEB) January 22, 2015

The Miami Stem Cell Treatment Center announces the opening of a new office in The Villages, Florida on January 28, 2015, with Dr. Thomas A. Gionis, Surgeon-in-Chief and Dr. Nia Smyrniotis, Medical Director and Surgeon.

Their new office is located at the Villages Endoscopy & Surgical Center, 10900 SE 174th PL. Rd., Summerfield, FL 34491. If you have any questions or would like further information please call us at (561) 331-2999.

The Miami Stem Cell Treatment Center (Miami; Boca Raton; Orlando; and now The Villages), along with sister affiliates, the Irvine Stem Cell Treatment Center (Irvine; Westlake Villages, Ca.) and the Manhattan Regenerative Medicine Medical Group (Manhattan, New York), abide by approved investigational protocols using adult adipose derived stem cells (ADSCs) which can be deployed to improve patients quality of life for a number of chronic, degenerative and inflammatory conditions and diseases. ADSCs are taken from the patients own adipose (fat) tissue (found within a cellular mixture called stromal vascular fraction (SVF)). ADSCs are exceptionally abundant in adipose tissue. The adipose tissue is obtained from the patient during a 15 minute mini-liposuction performed under local anesthesia in the doctors office. SVF is a protein-rich solution containing mononuclear cell lines (predominantly adult autologous mesenchymal stem cells), macrophage cells, endothelial cells, red blood cells, and important Growth Factors that facilitate the stem cell process and promote their activity.

ADSCs are the bodys natural healing cells - they are recruited by chemical signals emitted by damaged tissues to repair and regenerate the bodys injured cells. The Miami Stem Cell Treatment Center only uses Adult Autologous Stem Cells from a persons own fat no embryonic stem cells are used; and no bone marrow stem cells are used. Current areas of study include: Emphysema, COPD, Asthma, Heart Failure, Heart Attack, Parkinsons Disease, Stroke, Traumatic Brain Injury, Lou Gehrigs Disease, Multiple Sclerosis, Lupus, Rheumatoid Arthritis, Crohns Disease, Muscular Dystrophy, Inflammatory Myopathies, and degenerative orthopedic joint conditions (Knee, Shoulder, Hip, Spine). For more information, or if someone thinks they may be a candidate for one of the adult stem cell protocols offered by the Miami Stem Cell Treatment Center, they may contact Dr. Gionis or Dr. Smyrniotis directly at (561) 331-2999, or see a complete list of the Centers study areas at: http://www.MiamiStemCellsUSA.com.

About the Miami Stem Cell Treatment Center: The Miami Stem Cell Treatment Center, along with sister affiliates, the Irvine Stem Cell Treatment Center and the Manhattan Regenerative Medicine Medical Group, is an affiliate of the California Stem Cell Treatment Center / Cell Surgical Network (CSN); we are located in Boca Raton, Orlando, Miami and now The Villages, Florida. We provide care for people suffering from diseases that may be alleviated by access to adult stem cell based regenerative treatment. We utilize a fat transfer surgical technology to isolate and implant the patients own stem cells from a small quantity of fat harvested by a mini-liposuction on the same day. The investigational protocols utilized by the Miami Stem Cell Treatment Center have been reviewed and approved by an IRB (Institutional Review Board) which is registered with the U.S. Department of Health, Office of Human Research Protection (OHRP); and our studies are registered with Clinicaltrials.gov, a service of the U.S. National Institutes of Health (NIH). For more information, visit our websites: http://www.MiamiStemCellsUSA.com, http://www.IrvineStemCellsUSA.com, or http://www.NYStemCellsUSA.com.

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Cost of Stem Cells Transplant in Mexico l Placid Answer
In this Video you can get the best answers for your questions about Cost of Stem Cells Transplant in Mexico! http://www.placidway.com/answer-detail/1477/What-is-the-cost-of-stem-cells-transplant-...

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Godfrey Flies Cyberquad, Instrumental by Goodie Mob, Cell Therapy
Unmaned Aerial Vehicle, the Cyberquad flown by Godfrey Dimau. Suva Fiji Islands.

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Thursday 01/22: Celebrity Transformations; Stem Cell Therapy Debate; Samantha Harris Health Crisis
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The New Stem Cell Therapy 2015
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Regulatory News:

TxCell SA (Paris:TXCL) (FR0010127662 TXCL), a biotechnology company developing innovative, cost-effective, personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announces today that Dr. Miguel Forte, Sr. VP Clinical Development and Regulatory Affairs, TxCell, will present at the Cell & Gene Therapy Forum 2015, January 26-28, 2015 at Grand Hayatt in Washington DC, United States.

Dr Fortes presentation will be on critical success factors for a successful transition of cell therapy candidates to the clinic. The presentation is scheduled for Monday, January 26, 2015 at 14.30 p.m. EST. A roundtable debate discussing the most important topics raised will follow the presentation.

Dr. Forte will use as examples TxCells lead product candidate, Ovasave and second product candidate, Col-Treg from TxCells innovative proprietary ASTrIA technological platform. Ovasave is targeted at refractory Crohns disease and is currently evaluated in a placebo-controlled phase IIb study. Col-Treg is in development for the rare eye disease autoimmune uveitis. Col-Treg is planned to move into a first clinical study in the first half of 2015.

The key elements in the presentation include gathering strong pre-clinical and preliminary clinical data. Dr. Forte will also cover key process development activities necessary to provide the most robust and transferable manufacturing process and business model establishment and the development of a clear regulatory strategy. Dr. Forte will also present a roundup, from his perspective, on market access including economic viability and therapeutic adoption.

Patients with chronic inflammatory and autoimmune diseases that have no current therapeutic options continue to present significant unmet medical needs. As TxCell is one of the most advanced companies in the field of T-cell immunotherapy, we share our experiences regarding the development of innovative therapeutic approaches. This will benefit of the entire scientific community, said Miguel Forte, Sr. VP Clinical Development and Regulatory Affairs, TxCell. The Cell & Gene Therapy Forum 2015 is an ideal event to position biotechnology companies developing new value-adding treatments and strategies, like TxCells personalized antigen specific Treg cell-based therapies.

The Cell & Gene Therapy Forum is a meeting designed to help advance regulatory, manufacturing, research and commercial strategies and drive cell and gene therapy products forward. The Forum enables to gather executives from biotechnology companies, representatives of big pharma and big biotech, regulators, and public and private investors.

About TxCell

TxCell develops innovative, cost-effective, personalized T cell immunotherapies for the treatment of severe chronic inflammatory diseases with high medical need. TxCell has created ASTrIA, a unique and proprietary technology platform based on the properties of autologous antigen-specific regulatory T lymphocytes (Ag-Tregs). The company has initiated a phase IIb study of its lead product candidate, Ovasave in refractory Crohns disease patients. This follows a phase I/IIa study in the same patient population reporting positive clinical efficacy and good tolerability. TxCell has a strategic partnership for the development of Ovasave with the Swiss company Trizell Holding SA and Ferring International Center remains the intended final commercializing party. Both companies are affiliates of the Dr Frederik Paulsen Foundation. TxCells second product candidate, Col-Treg is for the treatment of autoimmune uveitis, a rare disease of the eye. A placebo-controlled, dose-ranging proof of principle clinical study is planned to start in the first half of 2015. Listed on Euronext-Paris, TxCell, a spin-off of Inserm (Frances National Institute for Health and Medical Research) is located in the Sophia Antipolis technology park, Nice, France. The company has 56 employees based at its headquarters and at its manufacturing site in Besanon.

For more information, please visit http://www.txcell.com

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A major research study has uncovered several new genetic mutations that could drive testicular cancer -- and also identified a gene which may contribute to tumours becoming resistant to current treatments.

The study is the first to use state-of-the-art sequencing technology to explore in detail testicular germ cell tumours -- which make up the vast majority of testicular cancers and are the most common cancers in young men.

It was led by scientists at The Institute of Cancer Research, London, and funded by the Movember Foundation.

The researchers, whose study was published in Nature Communications, used a genetic technique called whole-exome sequencing to examine tumour samples from 42 patients with testicular cancer treated at the Royal Marsden NHS Foundation Trust.

They uncovered a number of new chromosome duplications and other abnormalities that could contribute to the development of this cancer, as well as confirming a previous association with the gene KIT.

Their study also found defective copies of a DNA repair gene called XRCC2 in a patient who had become resistant to platinum-based chemotherapy. They were able to verify the link between XRCC2 and platinum resistance by sequencing an additional sample from another platinum-resistant tumour.

Although generally testicular cancer responds well to treatment, resistance to platinum-based chemotherapy is associated with a poor long-term survival rate. The research provides a clue to why around 3 per cent of patients develop resistance to platinum chemotherapy, as well as new insights into testicular germ cell tumours generally.

Dr Clare Turnbull, Team Leader in Predisposition and Translational Genetics at The Institute of Cancer Research, London, and Honorary Consultant in Clinical Genetics at The Royal Marsden NHS Foundation Trust, said:

"Our study is the largest comprehensive sequencing study of testicular tumours published to date, describing their mutational profile in greater detail than has been possible using previous technologies. We have identified new potential driver mutations for this type of cancer, and provided new evidence of a link between mutations in the gene XRCC2 and platinum treatment-resistant tumours.

"We now need additional studies with a larger number of patients, focusing in particular on platinum-resistant tumours, to help our discoveries lead to new options for those unlucky men whose cancer progresses in spite of the best available treatments."

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Mice with one rather than the normal two copies of the gene Myc (also found in humans) lived 15 percent longer and had considerably healthier lives than normal mice, according to a new Brown University-led study in Cell.

A team of scientists based at Brown University has found that reducing expression of a fundamentally important gene called Myc significantly increased the healthy lifespan of laboratory mice, the first such finding regarding this gene in a mammalian species.

Myc is found in the genomes of all animals, ranging from ancestral single-celled organisms to humans. It is a major topic of biomedical research and has been shown to be a central regulator of cell proliferation, growth, and death. It is of such widespread and fundamental importance that animals cannot live without it. But in humans and mice, too much expression of the protein that Myc encodes has been closely linked to cancer, making it a well-known but elusive target of drug developers.

In a new study in the journal Cell, the scientists report that when they bred laboratory mice to have only one copy of the gene, instead of the normal two, thus reducing the expression of the encoded protein, those mice lived 15 percent longer on average -- 20 percent longer for females and 10 percent longer among males -- than normal mice. Moreover, the experimental mice showed many signs of better health into old age.

The experimental -- "heterozygous" -- mice grew to be about 15 percent smaller than the normal mice (a probable disadvantage in the wild) but that was the only discernable downside found to date for lacking a second copy of the gene, said senior author John Sedivy, the Hermon C. Bumpus Professor of Biology and professor of medical science at Brown.

"The animals are definitely aging slower," he said. "They are maintaining the function of their organs and tissues for longer periods of time."

Physiological differences

That assessment is based on detailed studies of the physiology -- down to the molecular level -- of the heterozygous and normal mice. The researchers conducted these experiments to try to understand the longevity difference between the two groups.

Co-lead author Jeffrey Hoffman, a medical and doctoral student, led the studies of the health of the mice, including various bodily systems. In many cases they were just like their normal counterparts. They reproduced just as well, for example.

"These mice are incredibly normal, yet they are really long-lived," Sedivy said. "The reason why we were struck by that is because in many other longevity models like caloric restriction or treatment with rapamycin, the animals live longer but they also have some health issues."

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IMAGE:Using the zebrafish facility at Washington University School of Medicine in St. Louis, graduate student Sarah Ackerman (left) and senior author Kelly Monk, PhD, identified a gene that regulates how... view more

Credit: Robert Boston

Scientists have identified a gene that helps regulate how well nerves of the central nervous system are insulated, researchers at Washington University School of Medicine in St. Louis report.

Healthy insulation is vital for the speedy propagation of nerve cell signals. The finding, in zebrafish and mice, may have implications for human diseases like multiple sclerosis, in which this insulation is lost.

The study appears Jan. 21 in Nature Communications.

Nerve cells send electrical signals along lengthy projections called axons. These signals travel much faster when the axon is wrapped in myelin, an insulating layer of fats and proteins. In the central nervous system, the cells responsible for insulating axons are called oligodendrocytes.

The research focused on a gene called Gpr56, which manufactures a protein of the same name. Previous work indicated that this gene likely was involved in central nervous system development, but its specific roles were unclear.

In the new study, the researchers found that when the protein Gpr56 is disabled, there are too few oligodendrocytes to provide insulation for all of the axons. Still, the axons looked normal. And in the relatively few axons that were insulated, the myelin also looked normal. But the researchers observed many axons that were simply bare, not wrapped in any myelin at all.

Without Gpr56, the cells responsible for applying the insulation failed to reproduce themselves sufficiently, according to the study's senior author, Kelly R. Monk, PhD, assistant professor of developmental biology. These cells actually matured too early instead of continuing to replicate as they should have. Consequently, in adulthood, there were not enough mature cells, leaving many axons without insulation.

Monk and her team study zebrafish because they are excellent models of the vertebrate nervous system. Their embryos are transparent and mature outside the body, making them useful for observing developmental processes.

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Hidden topics amp; journalists Genetic Engineering

By: WORLD FOOD INSTITUTE

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The breakthrough was achieved by Harvard and Yaleuniversities They modified E. coli so it couldn't survive without an amino acid Scientists say they could stop supplying amino acid to kill bacteria It is possible to extend the technique to genetically modified crops This may ease concerns about GM strains mixing with organic food

By Ellie Zolfagharifard For Dailymail.com

Published: 12:18 EST, 21 January 2015 | Updated: 20:17 EST, 22 January 2015

Extreme gene manipulation has been used to modify bacteria that die if they get out of human control.

Instead of using traditional genetic engineering - which moves a gene from one organism to another - scientists have rewritten the language of genetics.

The breakthrough is a potential step toward better management of genetically engineered organisms, including crops.

Extreme gene manipulation tools have been used to modify bacteria that die if they get out of human control

Genetically altered microbes are used now in industry to produce fuels, medicines and other chemicals.

The new technique might also reduce the risk of using them outdoors, such as for cleaning up toxic spills.

Scientists from Harvard and Yale introduced the new approach in two papers released this week by the journal Nature.

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Self-destructing bacteria could lead to artificial life: Scientists build 'kill' system into GM organisms to ease ...

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NEW DELHI: A global team of about 300 scientists studied over 30,000 brain scans of people from 33 countries and found that eight common genetic mutations are behind the aging of brain. This was the largest collaborative study of the brain to date and its findings could lead to targeted therapies and interventions for Alzheimer's disease, autism and other neurological conditions.

The study known as the Enhancing Neuro Imaging Genetics through Meta Analysis (ENIGMA) Network was led by researchers from the Keck School of Medicine of the University of Southern California (USC) and involved 190 institutions. The findings are published on Jan 21 in Nature. This is the first high-profile study since the National Institutes of Health (NIH) of the US launched its Big Data to Knowledge (BD2K) centers of excellence in 2014.

"ENIGMA's scientists screen brain scans and genomes worldwide for factors that help or harm the brainthis crowd-sourcing and sheer wealth of data gives us the power to crack the brain's genetic code," said Paul Thompson, professor at Keck School of Medicine and principal investigator of ENIGMA. "Our global team discovered eight genes that may erode or boost brain tissue in people worldwide. Any change in those genes appears to alter your mental bank account or brain reserve by 2 or 3 percent. The discovery will guide research into more personalized medical treatments for Alzheimer's, autism, depression and other disorders."

The study could help identify people who would most benefit from new drugs designed to save brain cells, but more research is necessary to determine if the genetic mutations are implicated in disease.

The ENIGMA researchers screened millions of "spelling differences" in the genetic code to see which ones affected the size of key parts of the brain in magnetic resonance images (MRIs) from 30,717 individuals. The MRI analysis focused on genetic data from seven regions of the brain that coordinate movement, learning, memory and motivation. The group identified eight genetic variants associated with decreased brain volume, several found in over one-fifth of the world's population. People who carry one of those eight mutations had, on average, smaller brain regions than brains without a mutation but of comparable age; some of the genes are implicated in cancer and mental illness.

In October 2014, the NIH invested nearly $32 million in its Big Data Initiative, creating 12 research hubs across the United States to improve the utility of biomedical data. USC's two BD2K centers of excellence, including ENIGMA, were awarded a total of $23 million over four years.

"The ENIGMA Center's work uses vast datasets as engines of biomedical discovery; it shows how each individual's genetic blueprint shapes the human brain," said Philip Bourne, Ph.D., associate director for data science at the NIH. "This 'Big Data' alliance shows what the NIH Big Data to Knowledge (BD2K) Program envisions achieving with our 12 Centers of Excellence for Big Data Computing."

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The endeavor known as precision medicine, which Obama singled out in his State of the Union Address, may sound futuristic, but its been around long enough for people to have screwed it up, and badly. One of the worst medical scandals this century started with cancer researchers at Duke promising something that sounded a little too good to be true and ended with retracted papers, dashed patient hopes and lawsuits.

But precision medicine is obviously moving forward. To learn more about it, and what lessons the past has to offer, I caught up with Keith Baggerly, whose dogged investigations uncovered the problem with the Duke project. Baggerly is a professor in the Department of Bioinformatics and Computational Biology and Division of Quantitative Sciences at UT MD Anderson Cancer Center. (He is also a witness in a pending lawsuit filed by patients and their families.)

Though precision medicine has different meanings, medical researchers tend to use that term or personalized medicine to refer to the use of individual DNA differences in tailoring treatments to patients. The strategy is being driven by advances in the ability to quickly and cheaply read the sequences of code characters in DNA and by the growing use of big data to find patterns. As described in this Philadelphia Inquirer story, a number of big data cancer initiatives are gathering momentum.

The dream of precision medicine has been particularly tantalizing for cancer treatment, since cancer cells are just ordinary cells with broken DNA mutations that change the cells instructions and cause them to run amok.

And so, in 2006, cancer researchers around the word took notice when a team led by Dr. Anil Potti at Duke claimed in the prestigious journal Nature Medicine that theyd created a highly complex mathematical system that could assess a given patients tumor and determine from its genetic make-up exactly which drugs would give that patient the best odds of survival. While investigations have revealed fraud on the part of Anil Potti, many other people made mistakes in ignoring whistle blowers and allowing the technique to be used on cancer patients in a clinical trial.

While some avenues of precision medicine could lead to new, prohibitively expensive drugs used for rare subsets of patient, the Duke technique promised to chart the best course among existing treatments said Baggerly.

It would be based on the DNA in individual patients tumors. And it didnt just apply to one kind of cancer but to cancers across the board. Instead of telling a patient there was a 70% chance a drug would work to kill her tumor, he said, they could find out ahead of time if she was in the other 30% and prescribe an alternative course of treatment.

Doctors were excited and thought if the system worked, they owed it to their own patients to adopt a form of it, he said. Several groups asked Baggerly to look into it. One danger with the approach, he said, was that it was impossible to know how the technique worked. The data were so big they were measuring thousands of things per patient and there was this perception that the analysis of such data sets would be complex, he said. In most medical tests, theres some understanding of how they work. Thats true in some of the early advances in precision medicine. In some cases of melanoma, for example, theres a break in a particular gene called BRAF, and drugs that target cells with that broken gene. Theres a mechanistic understanding of how it all works.

But with the Duke project, he said, nobody has a good intuition of what 50 or 60 things are doing at once. And so there was no way for intuition to tell anyone whether it worked at all. When Baggerly started to re-analyze how the Duke researchers created the system in the first place, it didnt work. Was he using the system wrong or was there something wrong with the system?

As he investigated further, he found egregious errors that should have prevented it from working. The team had relied on cancer cell samples that had various degrees of resistance to an array of drugs. Those had been mislabeled. Some were reversed, so that the cells that were most resistant were labelled as the least.

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Investigator Offers Lessons From Precision Medicine's Cancer Debacle

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Dr. Mack Crayton - Genetics and Race (EURO)
Dr. Mack Crayton, Assistant Professor of Biology at Xavier University discusses genetics and race in light of the European-American Unity and Rights Organiza...

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Bodybuilding Motivation 2015 ep 1 - Genetics
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Does Genetics Matter When It Comes to Building Muscle and Strength?
"Jason Yamamoto - Never Forget (Breakbeat Album of the Year)" Download here: http://ulozto.net/xrp972Ph/jason-yamamoto-never-forget-rar "For The Homies Mixta...

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The genetics underpinning resistance to a frontline malaria drug, artemisinin, have been revealed, scientists say.

In South East Asia, malaria parasites have developed tolerance to the treatment, and there are fears that this will spread.

Now, in the largest genetic study to date, scientists have identified mutations in the parasite genome that are linked to resistance.

The study is published in Nature Genetics.

The researchers say the findings will help them to identify areas where artemisinin resistance could spread.

Lead author Dr Olivo Miotto from the Mahidol-Oxford Tropical Research Unit (MORU), in Thailand, said: "Artemisinin is the best drug we have had for a very long time, and we want to continue this success story.

"And for that its effectiveness has to be protected and sustained."

When the first malaria drug, chloroquine, was developed, researchers thought that the disease would be eradicated within years.

But the malaria parasite has proved far tougher than they ever imagined. Drug after drug has been rendered useless as the parasite has evolved to evade treatment.

Mysteriously, each time resistance has emerged, it has started in the same place - on the Cambodia-Thai border - before spreading across Asia and into Africa.

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Genetics Of Malaria Drug Resistance Revealed

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Washington, D.C.-basedgene therapy outfit Regenxbio just raised $30 million to bringits platforminto the clinic.

The dollars will help Regenxbio generate clinical proof of concept data, as well as work toward in-licensing new programs. The companys also using the money to beef up its clinical and manufacturing processes, it said in a statement.

Regenxbio has developed what it calls NAV Technology a form of adeno-associated viral gene therapy that treats lysosomal storage disorders and ocular disease. Its got drugs in the pipeline that treat Hurler syndrome, Hunter syndrome, wet age-related macular degeneration and X-linked retinitis pigmentosa.

Heres how it works: In Hurler syndrome, for instance, children dont carry a gene that develops an enzyme called IDUA that breaks down complex sugars. These build up, and ultimately impairmental development, organ function, physical abilities and appearance. The NAV platform delivers a normal copy of the IDUA-producing gene which ultimately embeds itself into a patients DNA in a one-time doze so that patients can produce the enzyme. The research comes out of the University of Pennsylvania, and has been successful in vivo so its a matter of testing its efficacy in a real patient pool.

The companys also out-licensing this technology for other indications, with companies like Baxter, Dimension Therapeutics and Lysogene developing viral vector-based gene therapy for a number of other indications.

The Series C funding was led by Venrock and Brookside Capital, with other investors like Deerfield Management, FoxKiser and Fidelity Biosciences.

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PHILADELPHIA, Penn., January 20, 2015- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today it has initiated a Phase 1/2 clinical trial for the potential treatment of patients with choroideremia (CHM) utilizing its gene therapy product SPK-CHM. The Phase 1/2 trial is an open-label, dose-escalating trial designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The Phase 1/2 trial will be conducted at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania, and has plans to enroll up to 10 patients afflicted with the CHM genetic mutation.

"Spark's groundbreaking announcement today brings real hope of a treatment for blindness caused by choroideremia, and promises to pave the way for treatments of other retinal diseases impacting millions of people around the World," said Dr. Chris Moen, President of the Choroideremia Research Foundation (curechm.org), the leading advocacy and fundraising organization focused on finding a cure for CHM. "The Choroideremia Research Foundation is proud to have provided key pre-clinical funding to Jean Bennett, MD, PhD and her team atthe Perelman School of Medicine at the University of Pennsylvania,that has helped bring us to the gene therapy human clinical trials being announced today."

Dr. Jean Bennett's pre-clinical work, which was funded in part by consistent financial support from the Choroideremia Research Foundation (curechm.org), demonstrated the ability of the SPK-CHM gene therapy to restore REP-1 protein production, membrane trafficking and retinal structure.

"Throughout my career's work developing genetic therapies for inherited retinal dystrophies I have had my target set on a number of different conditions, in particular, choroideremia," said Dr. Bennett, who is also one of Spark's scientific co-founders and a scientific advisor on the SPK-RPE65 clinical trials being conducted at CHOP. "The SPK-CHM program, for the first time, creates the potential for patients to use their vision for longer and see more things."

In addition to evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify appropriate endpoints for subsequent clinical trials. With SPK-CHM, Spark is leveraging the experience and technology utilized in the development of its gene therapy for Leber's Congenital Amaurosis (LCA), SPK-RPE65, including the same vector, target cells and route of administration, as well as the same manufacturing process. SPK-RPE65 is currently in a fully-enrolled pivotal Phase 3 clinical trial.

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U.S. Gene Therapy Clinical Trial to treat Choroideremia initiated

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MIAMI (PRWEB) January 22, 2015

Regenestem, a division of the Global Stem Cells Group, Inc., has announced the launch of a new stem cell treatment center in Veracruz, Mexico. The new facility offers the most advanced protocols and techniques in cellular medicine to patients from around the world.

The opening of Regenestem Veracruz is in partnership with Eleuterio Arrieta, M.D., Director of Santa Teresita Hospital in Veracruz. Dr. Arrieta has extensive experience in management of chronic degenerative diseases with autologous stem cell therapies, expertise he will use to deliver cutting edge therapies and follow-up treatment under the Regenestem brand in Veracruz.

Under the direction of Global Stem Cells Group, Regenestem is expanding its clinical presence worldwide by partnering with qualified physicians experienced in stem cell therapies to open new clinics, licensed and developed under the Regenestem banner.

In 2014, Global Stem Cells Group expanded the Regenestem Networks presence to 20 countries, adding new state-of-the-art regenerative medicine facilities to the company's growing global presence.

Regenestem offers stem cell treatments to help with a variety of diseases and conditions including arthritis, autism, chronic obstructive pulmonary disease (COPD), diabetes, and pain due to injuries at various facilities worldwide. Regenestem Veracruz will have an international staff experienced in administering the leading cellular therapies available.

Regenestem is certified for the medical tourism market, and staff physicians are board-certified or board-eligible. Regenestem clinics provide services in more than 10 specialties, attracting patients from the United States and around the world.

The Global Stem Cells Group and Regenestem are committed to the highest of standards in service and technology, expert and compassionate care, and a philosophy of exceeding the expectations of their international patients.

For more information, visit the Regenestem Network website, email info(at)regenstem(dot)com, or call 305-224-1858.

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Regenestem Network, a division of Global Stem Cells Group, Announces Launch of New Stem Cells and Regenerative ...

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The International Society for Stem Cell Research's McEwen Award recipient Hans Clevers extends breakthrough work

CHICAGO -- The International Society for Stem Cell Research (ISSCR) has awarded Dr. Hans Clevers, senior author on two important papers published recently in the scientific journal Cell, the society's McEwen Award for Innovation. The papers describe the development of a culturing system for human liver stem cells, as well as stem cells from pancreatic cancer, discoveries with the potential to revolutionize liver transplantation and aid in the fight against pancreatic cancer, respectively.

Clevers is a professor at the Hubrecht Institute and president of the Royal Netherlands Academy of Arts and Sciences. He shares the McEwen Award for Innovation with Dr. Irving Weissman, Stanford School of Medicine, for the identification, prospective purification and characterization of somatic (adult) tissue-associated stem cells and advancement of this research toward clinical applications.

"These new discoveries by Hans Clevers extend the work for which he was awarded the McEwen Award, the ISSCR's most prestigious award," Dr. Rudolf Jaenisch, ISSCR president, said. "The innovative approach Dr. Clevers took in the gut has borne fruit and proven the basis of these significant advances in the liver and pancreas, which hold great promise for the study of and treatments for diseases impacting these organs."

Organoids

Until recently, it appeared impossible to keep healthy or diseased tissue from patients alive under laboratory conditions, let alone multiply it. However, in 2009, the research group headed by Clevers described a revolutionary culturing method that allowed the culturing of mini-guts from single mouse intestine stem cells. These organoids are functional miniature organs that can grow in tissue culture. The same research group now adds a culturing system for liver stem cells and stem cells from pancreatic cancer to their record. In the future, cultured stem cells could conceivably replace donor organs for transplantation. They also offer prospects for personalized medicine, the development of treatments specifically geared to individual patients.

Cultured Liver Stem Cells

The technology described in Cell can be used for the long-term replication in the laboratory of minute amounts of tissue harvested from a healthy or diseased liver. Over a period of four months, the equivalent of a full-grown liver can be cultured from a single liver stem cell. All analyses show that this cultured tissue is genetically the same as healthy liver tissue and is very stable.

The cultured human mini-livers have already been successfully transplanted in mice with liver damage. This is the first step toward using this cultured liver tissue to replace donor livers for transplantation. As such, this technology could solve the worldwide shortage of donor livers. Moreover, this technology offers future potential for personalized medicine. Organoids could, for instance, be grown from the tissue of patients suffering from genetic liver diseases, so that drugs could be tested on this patient material first, before being administered to the patients themselves. Examples of such diseases are alpha-1 antitrypsin deficiency and Alagille Syndrome.

Pancreatic Stem Cells

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Scientists announce revolutionary culturing technique for liver and pancreas

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By Amy Norton HealthDay Reporter

TUESDAY, Jan. 20, 2015 (HealthDay News) -- A therapy that uses patients' own primitive blood cells may be able to reverse some of the effects of multiple sclerosis, a preliminary study suggests.

The findings, published Tuesday in the Journal of the American Medical Association, had experts cautiously optimistic.

But they also stressed that the study was small -- with around 150 patients -- and the benefits were limited to people who were in the earlier courses of multiple sclerosis (MS).

"This is certainly a positive development," said Bruce Bebo, the executive vice president of research for the National Multiple Sclerosis Society.

There are numerous so-called "disease-modifying" drugs available to treat MS -- a disease in which the immune system mistakenly attacks the protective sheath (called myelin) around fibers in the brain and spine, according to the society. Depending on where the damage is, symptoms include muscle weakness, numbness, vision problems and difficulty with balance and coordination.

But while those drugs can slow the progression of MS, they can't reverse disability, said Dr. Richard Burt, the lead researcher on the new study and chief of immunotherapy and autoimmune diseases at Northwestern University's Feinberg School of Medicine in Chicago.

His team tested a new approach: essentially, "rebooting" the immune system with patients' own blood-forming stem cells -- primitive cells that mature into immune-system fighters.

The researchers removed and stored stem cells from MS patients' blood, then used relatively low-dose chemotherapy drugs to -- as Burt described it -- "turn down" the patients' immune-system activity.

From there, the stem cells were infused back into patients' blood.

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Early Study Says Stem Cells May Reverse Multiple Sclerosis Disability

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Food safety watchdog in Europe is countering findings that Bisphenol A, a chemical used to make plastic, is harmful to your health Researchers say stem cells could provide new treatment options for reversing the disability caused by multiple sclerosis http://pmd.680news.com/podcasts/health/jan21-health-news_Health-Report_2015-01-22.mp3

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Jan. 21: Bisphenol A research, multiple sclerosis treatment

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Scientists are hesitant to call it a cure, but they have succeeded in significantly reducing -- and in some cases, reversing -- disability caused by the crippling disorder multiple sclerosis. The therapy involves using the patients own stem cells in a single treatment.

The stem cells are collected from the patients blood and they are used to reset the immune system, so the body's fighter cells no longer mistake the patients own nervous system tissue for an invader. Those attacks cause disability and autoimmune diseases like multiple sclerosis.

MS is a degenerative disease. Ten years after being diagnosed, half of all MS patients are unable to work, and after 25 years with the disease, half lose the ability to walk.

Richard Burt, chief of the Division of Immunotherapy at Northwestern University in Evanston, Illinois, led a study of 150 MS patients, most with the mildest form of the disease called relapsing-remitting MS, in which they received the stem-cell therapy.

He said their disability seemed to reverse itself or disappear.

This is a one-time treatment and then you are done. And so we hope patients never need to be treated again, said Burt.

About half of the patients tested two years after the study showed significant improvement in their movement and cognitive function. Of the three dozen participants tested after four years, 23 had continued improvement of their symptoms, and 80 percent were free of relapses or flare-ups.

Ten percent of patients required another treatment after five years. Some of the participants, who had a more serious form of MS, did not improve.

MS disease progression and severity are measured by the number of lesions in the brain seen by high-tech imaging. Most of the patients who received the stem cells had fewer brain lesions.

Treatment for multiple sclerosis is expensive, typically close to $50,000 or more per year.

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Researchers Reduce MS Disability With Stem Cells

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