Editor's Choice Main Category: Muscular Dystrophy / ALS Also Included In: Transplants / Organ Donations Article Date: 29 Jun 2012 - 11:00 PDT

Current ratings for: Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice

A new study published in Science Translational Medicine reveals that researchers have, for the first time, managed to turn fibroblast cells, i.e. common cells within connective tissue, from muscular dystrophy patients into stem cells and subsequently changed these cells into muscle precursor cells. After modifying the muscle precursor cells genetically, the researchers transplanted them into mice.

In future, this new technique could be used in order to treat patients with the rare condition of limb-girdle muscular dystrophy, which primarily affects the shoulders and hips, and maybe other types of muscular dystrophies. The method was initially developed in Milan at the San Raffaele Scientific Institute and was completed at UCL.

Muscular dystrophy is a genetic disorder, which typically affects skeletal muscles. The condition leads to severely impaired mobility and can, in severe cases result in respiratory and cardiac dysfunction. At present, there is no effective treatment for the condition. A number of new potential therapies, including cell therapy, are entering clinical trials.

The scientists of this study concentrated their research on genetically modifying mesoangioblasts, i.e. a self-renewing cell that originates from the dorsal aorta and differentiates into most mesodermal tissues, which demonstrated its potential for treating muscular dystrophy in earlier studies.

Given that the muscles of patients with muscular dystrophy are depleted of mesonangioblasts, the researchers were unable to obtain sufficient numbers of these cells from patients with limb-girdle muscular dystrophy, and therefore "reprogrammed" adult cells from these patients into stem cells, which enabled them to prompt them to differentiate into mesoangioblast-like cells.The team then genetically corrected these 'progenitor' cells by using a viral vector, and injected them into mice with muscular dystrophy so that the cells targeted damaged muscle fibers.

In a mice study, the same process demonstrated that dystrophic mice were able to run on a treadmill for longer a longer time than dystrophic mice that did not receive the cells.

Research leader, Dr Francesco Saverio Tedesco, from UCL Cell & Developmental Biology, who led the study, explained:

Professor Giulio Cossu, also an author at UCL, concluded:

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Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice

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Editor's Choice Main Category: Huntingtons Disease Also Included In: Stem Cell Research;Neurology / Neuroscience Article Date: 29 Jun 2012 - 14:00 PDT

Current ratings for: Brain Cells Derived From Skin Cells For Huntington's Research

3 (1 votes)

At present, there is no cure for the disease and no treatments are available. These findings open up the possibility of testing treatments for the deadly disorder in a petri dish.

The study is the work of a Huntington's Disease iPSC Consortium, including researchers from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, and six other groups.

Huntington's disease is an inherited, deadly neurodegenerative disorder. The onset of HD generally occurs during midlife, although it can also strike in childhood - as in the patient who donated the material for the cells generated in this study. The disease causes jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and ultimately death.

Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the lead researchers of the study, explained:

The team are currently testing small molecules for the ability to block HP iPSC degeneration. According to the researchers, these molecules could potentially be developed into new drugs for Huntington's disease.

Furthermore, the teams ability to create "HD in a dish" may also have implications for similar research in other diseases such as Parkinson's and Alzheimer's.

In the study, the team took a skin biopsy from a 7-year-old patient with very early onset of severe HD. In the laboratory of Hongjun Song, Ph.D., a professor at Johns Hopkins' Institute for Cell Engineering, the skin cells were grown in culture and then created into pluripotent stem cells. In addition, a second cell line was created in the same way in Dr. Ross's lab from an individuals without HD.Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

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Brain Cells Derived From Skin Cells For Huntington's Research

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June 29, 2012

Connie K. Ho for redOrbit.com Your Universe Online

In 1993, the autosomal dominant gene mutation responsible for Huntingtons Disease (HD) was discovered. However, no treatments are known to slow its progression. New research may pave the way to better understanding of the disease. Researchers at Johns Hopkins recently announced that they were able to produce stem cells from skin cells from a person who had severe, early-onset form of HD; the cells were then changed into neurons that degenerated like the cells affected by HD.

The research was recently published in the journal Cell Stem Cell. The investigators worked with an international consortium in creating HD in a dish. The group was made up of scientists from Johns Hopkins University School of Medicine, Cedars-Sinai Medical Center, the University of California at Irvine, as well as six other groups. The team looked at many other HD cell lines and control cell lines to verify that the results were consistent and reproducible in other labs. The investigators believe that the findings allow them to better understand and eliminate cells in people in with HD. They hope to study the effects of possible drug treatments on cells that would be otherwise found deep in the brain.

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

The team of researchers is studying small molecules for the ability to block HD iPSC degeneration to see if they can be developed into new drugs for HD. As well, the ability to produce from stem cells the same neurons found in HD may have effects for similar research in other neurodegenerative diseases like Alzheimers and Parkinsons. In the experiment, Ross took a skin biopsy from a patient with very early onset HD. The patient was seven years old at the time, with a severe form of disease and a mutation that caused it. By using cells from a patient who had quickly progressing HD, Ross team were able to mimic HD in a way that could be used by patients who had different forms of HD.

The skin cells were grown in culture and reprogrammed to induce stem cells that were pluripotent. Then, another cell line was created in the same way from someone who didnt have HD. The other HD and control iPS cells were produced as part of the NINDS funded HD iPS cell consortium. Investigators from Johns Hopkins and the other consortium labs changed the cells into typical neurons and then into medium spiny neurons. The process took a total of three months and the scientists found the medium spiny neurons from the HD cells acted how the medium spiny neurons form an HD patient would. The cells demonstrated quick degeneration when cultured in the lab with a basic culture medium that didnt include extensive supporting nutrients. On the other hand, control cell lines didnt demonstrate neuronal degeneration.

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

Source: Connie K. Ho for redOrbit.com Your Universe Online

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Skin Cells Create Stem Cells In Huntington Disease Study

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28-06-2012 13:54 About 5 months ago, she came home from the beach with my husband limping on her right back leg. Now 8 weeks later after stem cell therapy... we were happy (well, maybe not so much...) to see her back to her old, wild, hyper self again.

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Ipo 8 weeks after stem cell therapy at Surf Paws Animal Hospital - Video

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29-06-2012 14:27 Of all the methods of gene transfer, lentiviral transduction and retroviral transduction are ideal because they allow development of stably transduced mammalian cell lines. But traditional methods require adding cell-killing additives. Learn how cell-friendly RetroNectin Reagent can dramatically boost transduction efficiency - while keeping your cells happy!

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Efficient Gene Transduction - Make Your Cells Happy! - Video

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29-06-2012 19:25 Clip 1. GENETIC CHILE What is a GMO? Stephen Hanson, PhD from New Mexico State University defines GMO as a genetically modified organism, really refers to organisms that are created through genetic engineering. *** GENETIC CHILE, An eye-opening look at the world of genetically modified foods through the lens of New Mexico's iconic chile pepper. The chile pepper defines New Mexican cuisine and is considered a sacred plant by many cultures. Despite overwhelming evidence of gene flow, persistent safety questions, predatory multinational agribusiness corporations and potential economic damage, the State of New Mexico funded research to produce a GMO chile, which is the first time a US state has done so. Because the funding is public, filmmaker Chris Dudley, was able to force a rare interview with a genetic researcher at NMSU. This film is packed with information about the harmful use of GMO technology and the ignorance shown by the proponents of GMO crops. Documentary, by Chris Dudley. In English | 60 minutes. |

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Genetic Chile Clip 1 - Video

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29-06-2012 19:33 Clip 2. GENETIC CHILE Monsanto, the largest seed company in the world and NMSU working together to create and sell genetically manufactured seeds. *** GENETIC CHILE, An eye-opening look at the world of genetically modified foods through the lens of New Mexico's iconic chile pepper. The chile pepper defines New Mexican cuisine and is considered a sacred plant by many cultures. Despite overwhelming evidence of gene flow, persistent safety questions, predatory multinational agribusiness corporations and potential economic damage, the State of New Mexico funded research to produce a GMO chile, which is the first time a US state has done so. Because the funding is public, filmmaker Chris Dudley, was able to force a rare interview with a genetic researcher at NMSU. This film is packed with information about the harmful use of GMO technology and the ignorance shown by the proponents of GMO crops. Documentary, by Chris Dudley. In English | 60 minutes. |

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Genetic Chile Clip 2 - Video

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29-06-2012 19:42 Clip 3. GENETIC CHILE Are GMOs good for us? No one knows what the long term effects of GMOs will be, but during the first Bush Administration it was decided that GMOs would be "generally regarded as safe". *** GENETIC CHILE, An eye-opening look at the world of genetically modified foods through the lens of New Mexico's iconic chile pepper. The chile pepper defines New Mexican cuisine and is considered a sacred plant by many cultures. Despite overwhelming evidence of gene flow, persistent safety questions, predatory multinational agribusiness corporations and potential economic damage, the State of New Mexico funded research to produce a GMO chile, which is the first time a US state has done so. Because the funding is public, filmmaker Chris Dudley, was able to force a rare interview with a genetic researcher at NMSU. This film is packed with information about the harmful use of GMO technology and the ignorance shown by the proponents of GMO crops. Documentary, by Chris Dudley. In English | 60 minutes. |

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Genetic Chile Clip 3 - Video

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29-06-2012 19:52 Clip 5. GENETIC CHILE The International Assessment of Agricultural Knowledge, Science and Technology for Development (IAAKSTD) compiled a 600 page comprehensive study titled "Agriculture at a Crossroads" which directly addresses and recommends solutions to world hunger. *** GENETIC CHILE, An eye-opening look at the world of genetically modified foods through the lens of New Mexico's iconic chile pepper. The chile pepper defines New Mexican cuisine and is considered a sacred plant by many cultures. Despite overwhelming evidence of gene flow, persistent safety questions, predatory multinational agribusiness corporations and potential economic damage, the State of New Mexico funded research to produce a GMO chile, which is the first time a US state has done so. Because the funding is public, filmmaker Chris Dudley, was able to force a rare interview with a genetic researcher at NMSU. This film is packed with information about the harmful use of GMO technology and the ignorance shown by the proponents of GMO crops. Documentary, by Chris Dudley. In English | 60 minutes. |

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Genetic Chile Clip 5 - Video

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June 29, 2012

Connie K. Ho for redOrbit.com Your Universe Online

The common cold, otherwise known as the flu, can creep up on unsuspecting people. It can leave people with fevers, sore throat, cough, runny nose, chills, fatigue, nausea, among other symptoms. Researchers have been looking into the flu to better understand the bodys responses to viruses. Scientists recently found how a new gene in the influenza virus could control the virus to manage the bodys actions against an infection.

The research was completed by a collaborative team of researchers from the University of Cambridge, University College Cork, the University of Edinburgh, the University of Utah, the Institute of Systems Biology, and the United States National Institutes of Health. The findings are published online in the journal Science.

Even though the virus manages the bodys response, it decreases the influence of the infection. In particular, when mice were infected with the active virus gene PA-X, they often recovered from having the flu. Researchers believe that the findings will help in terms of understanding how the flu can initiate severe infections. They believe that the new research will assist in the development of new treatments.

Just finding this gene in the first place is important, but the find is even more significant because of the role it seems to play in the bodys response to flu, noted Paul Digard, a member of The Roslin Institute at the University of Edinburgh, in a prepared statement.

The study focused on the how the gene affected the response of the Spanish flu, which is a virulent strain of influenza that initiated a pandemic that occurred in 1918. The researchers discovered the gene by studying the genetic information for patterns of changes. They analyzed thousands of different flu strains.

The flu virus has a very, very small genome just 12 genes. Finding a new gene makes a pretty significant change to our understanding of this virus, commented Dr. Andrew Firth, a researcher at the University of Cambridge, in the statement.

In particular, each of the influenza viruses has a shell that contains eight strains of RNA. RNA is a genetic molecule that is connected to DNA. While some of the strands can encode many different genes, each of the strains creates a different protein. Before the research, scientists thought that there were only eight strains that had 12 different genes. However, the new study shows that there may possibly be 13 different genes. As such, the influenza genome is thought to have overlapping instructions for protein production.

According to Discover Magazine, the new gene discovered by the researchers is also found in the virus third RNA strand that was thought to only have the PA gene. The PA gene assists the virus to make a copy of its genome. When the gene PA allowed the virus to make a copy of the genome, it gave PA-X a different task in cutting up bits of RNA from the viruss host and stopped the host from activating genes. As a result, the host-cell turned off and the host wasnt able to create a defense against the virus. Furthermore, the host ended up producing proteins based off the genetic instructions from the virus rather than eliminating the RNA.

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Gene Study Shows Flu Connection With Severe Infections

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Public release date: 29-Jun-2012 [ | E-mail | Share ]

Contact: Mary Guiden mary.guiden@seattlechildrens.org 206-987-7334 Seattle Children's

SEATTLE June 28, 2012 A research team led by Seattle Children's Research Institute has discovered new gene mutations associated with markedly enlarged brain size, or megalencephaly. Mutations in three genes, AKT3, PIK3R2 and PIK3CA, were also found to be associated with a constellation of disorders including cancer, hydrocephalus, epilepsy, autism, vascular anomalies and skin growth disorders. The study, "De novo germline and postzygotic mutations in AKT3, PIK3R2 and PIK3CA cause a spectrum of related megalencephaly syndromes," was published online June 24 in Nature Genetics.

The discovery offers several important lessons and hope for the future in medicine. First, the research team discovered additional proof that the genetic make-up of a person is not completely determined at the moment of conception. Researchers previously recognized that genetic changes may occur after conception, but this was believed to be quite rare. Second, discovery of the genetic causes of these human diseases, including developmental disorders, may also lead directly to new possibilities for treatment.

AKT3, PIK3R2 and PIK3CA are present in all humans, but mutations in the genes are what lead to conditions including megalencephaly, cancer and other disorders. PIK3CA is a known cancer-related gene, and appears able to make cancer more aggressive. Scientists at Boston Children's Hospital recently published similar findings related to PIK3CA and a rare condition known as CLOVES syndrome in the American Journal of Human Genetics.

Physician researcher James Olson, MD, PhD, a pediatric cancer expert at Seattle Children's and Fred Hutchinson Cancer Research Center who was not affiliated with the study, acknowledged the two decades-worth of work that led to the findings. "This study represents ideal integration of clinical medicine and cutting-edge genomics," he said. "I hope and believe that the research will establish a foundation for successfully using drugs that were originally developed to treat cancer in a way that helps normalize intellectual and physical development of affected children. The team 'knocked it out of the park' by deep sequencing exceptionally rare familial cases and unrelated cases to identify the culprit pathway." The genes AKT3, PIK3R2 and PIK3CAall encode core components of the phosphatidylinositol-3-kinase (P13K)/AKT pathway, the "culprit pathway" referenced by Olson.

The research provides a first, critical step in solving the mystery behind chronic childhood conditions and diseases. At the bedside, children with these conditions could see new treatments in the next decade. "This is a huge finding that provides not only new insight for certain brain malformations, but also, and more importantly, provides clues for what to look for in less severe cases and in conditions that affect many children," said William Dobyns, MD, a geneticist at Seattle Children's Research Institute. "Kids with cancer, for example, do not have a brain malformation, but they may have subtle growth features that haven't yet been identified. Physicians and researchers can now take an additional look at these genes in the search for underlying causes and answers."

Researchers at Seattle Children's Research Institute will now delve more deeply into the findings, with an aim to uncover even more about the potential medical implications for children. "Based on what we've found, we believe that we can eventually reduce the burden of and need for surgery for kids with hydrocephalus and change the way we treat other conditions, including cancer, autism and epilepsy," said Jean-Baptiste Rivire, PhD, at Seattle Children's Research Institute. "This research truly helps advance the concept of personalized medicine."

Drs. Dobyns, Rivire and team made this discovery through exome sequencing, a strategy used to selectively sequence the coding regions of the genome as an inexpensive but effective alternative to whole genome sequencing. An exome is the most functionally relevant part of a genome, and is most likely to contribute to the phenotype, or observed traits and characteristics, of an organism.

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Study finds new gene mutations that lead to enlarged brain size, cancer, autism, epilepsy

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28-06-2012 04:23 I also posted a text version of my article on sacns! The Daily Telegraph is reporting on an amazing achievement... a vaccine, which causes nicotine to have no effect whatsoever... on mice. Nicotine calms a person, and causes a slowing in heartbeat. These positive effects of smoking, are something the scientists, think... need to end. So... How does it work? Genetic Engineering... yes, I said it... Genetically Modified (GM) antibody... as a cure. The anti-body filters nicotine out of the blood, and after it appears once, the bodies... of the mice, mimic it. Such means: no more pleasure from smoking. It is suggested that, soon they may graduate from Mice to Men. But if you are a mouse or a man... the best laid schemes and plans of either... to quit, might determine if one is a mouse or a man... There is still the emotional addiction to deal with, if the process works, and any side affects, as yet unspoken. For more on what professor of Genetic Medicine, Dr Ronald Crystal, Weill Cornell Medical College, New York, has to say to the Daily Telegraph: A jab that 'vaccinates' people against smoking for life being developed - Telegraph » Scientists have invented a jab that takes the pleasure out of smoking, it has emerged. 'A jab that 'vaccinates' people against smoking for life being developed; Scientists have invented a jab that takes the pleasure out of smoking, it has emerged.' by Richard Alleyne at 7:51AM BST 28 Jun 2012 www ...

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Scientists invent cure to smoking... - Video

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Public release date: 29-Jun-2012 [ | E-mail | Share ]

Contact: Dana Mortensen mortensen@email.chop.edu 267-426-6067 Children's Hospital of Philadelphia

Douglas C. Wallace, Ph.D., director of the Center for Mitochondrial and Epigenomic Medicine at The Children's Hospital of Philadelphia, will receive the 2012 Genetics Prize of The Gruber Foundation. This prestigious international awarda $500,000 prizerecognizes Wallace's pioneering scientific investigations of the wide-ranging role of mitochondria in the development of disease and as markers of human evolution. Mitochondria are the tiny power plants within the cytoplasm of animal and plant cells.

Wallace will receive the award on November 9 at the annual meeting of the American Society of Human Genetics in San Francisco. The Gruber Foundation, now based at Yale University, announced the Genetics Prize on June 28. The Foundation's Genetics Prize annually honors leading scientists for groundbreaking contributions to genetics research.

Philip R. Johnson, MD, chief scientific officer at The Children's Hospital of Philadelphia, acknowledged Wallace's achievements, saying, "The Children's Hospital of Philadelphia Research Institute is privileged to number Douglas Wallace among our research leaders. His commitment to the field of mitochondrial genetics and his pioneering nature embody the mission of research at CHOP, and his research and leadership are shaping the way we approach therapies for genetic disorders previously considered beyond treatment."

"Douglas Wallace's contributions to our understanding of mitochondrial genetics have changed the way human and medical geneticists think about the role of mitochondria in human health and disease," said Dr. Elizabeth Blackburn, chair of the Selection Advisory Board to the Prize. Blackburn, who shared the 2009 Nobel Prize in Physiology or Medicine, also received the Gruber Genetics Prize in 2006.

Wallace, who came to The Children's Hospital of Philadelphia in 2010 to launch the Center for Mitochondrial and Epigenomic Medicine, first achieved prominence in the 1970s as the leader of a research team at Stanford University that defined the genetics of mitochondrial DNA. This DNA resides within each mitochondrion, as distinct from the more familiar nuclear DNA inside chromosomes. His group showed that human mitochondrial DNA is inherited exclusively from the mother.

This discovery, coupled with other findings, allowed the researchers to reconstruct ancient human migration patterns over hundreds of millennia, a major contribution that bridges genetics and anthropology. Wallace and colleagues also have linked mutations in mitochondrial DNA to a broad range of human diseases, including types of blindness, deafness, metabolic disorders such as diabetes, neuropsychiatric conditions, and age-related diseases such as heart disease and cancer.

The Center for Mitochondrial and Epigenomic Medicine at Children's Hospital researches mitochondrial dysfunction in many clinical problems, and also focuses on preclinical studies relevant to developing therapies for mitochondrial diseases, for which few effective clinical treatments currently exist.

Wallace holds the Michael and Charles Barnett Endowed Chair in Pediatric Mitochondrial Medicine at Children's Hospital and also is a professor of Pathology and Laboratory Medicine in the Perelman School of Medicine at the University of Pennsylvania. He is a member of the National Academy of Sciences, the nation's premier organization of leading researchers, as well as the Academy's Institute of Medicine, and is also a member of the American Academy of Arts and Sciences.

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Dr. Douglas Wallace to receive Gruber Foundation 2012 Genetics Prize

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Cant kick cigarettes? A vaccine may one day help by preventing nicotine from reaching its target in the brain, according to research published this week.

Most smoking therapies do a poor job of stopping the habit 70% to 80% of smokers who use an approved drug therapy to quit relapse. Scientists say this is because the targets of existing therapies are imperfect, only slightly weakening nicotines ability to find its target in the brain.

So some scientists have been trying a different approach creation of a vaccine. It would work like this: People would inject the vaccine like a shot, and the vaccine would create nicotine antibodies, molecules that can snatch up nicotine from the bloodstream before it reaches the brain. The vaccine could be used by smokers who want to quit or people who are worried about getting addicted to cigarettes in the future.

Researchers have tried to create vaccines in the past, but the ones theyve come up with have not been particularly effective. The authors of the new study say this may be because previous vaccines just didnt create enough antibodies to get rid of all the nicotine.

The new report, published in the journal Science Translational Medicine, attempts to solve this problem via gene therapy, in which a new gene is inserted into the body to do a particular job.

First the scientists at Weill Cornell Medical College in New York City put a gene that produces a nicotine antibody into mice. The gene was taken into the mices livers, and the liver started producing the antibody. Once produced, the antibody connected with nicotine, trapping it and preventing it from making its way to the brain, where it would otherwise have caused the pleasurable, addictive effects it is so known for.

Because of this trick, the researchers say that the new vaccine should only have to be injected once, and it will work for life, continuing to produce new antibodies in the liver.

The vaccine was effective: When mice were given nicotine intravenously, ones with the vaccine had a 47-fold drop in levels of nicotine in the blood compared with ones that hadnt received the vaccine. The antibody had successfully captured the nicotine in the bloodstream before it could reach the brain.

The work is still preliminary, and the authors admit the technology is far from ready for human use; it has only been used in rodents so far. But given the results, and the continued public health effect of smoking, it may not be too long before all those boxes of Nicorette are replaced with a single trip to the doctors office.

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New gene therapy for smoking kills the pleasure of nicotine

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MOBILE, Alabama -- The Baldwin County doctor that treated former Alabama football players with adult stem cells also has treated at least two people diagnosed with amyotrophic lateral sclerosis, also known as Lou Gehrigs disease.

One of the ALS patients, former NFL football player and college coach Frank Orgel, recently underwent a new stem cell reprogramming technique performed by Dr. Jason R. Williams at Precision StemCell in Gulf Shores.

Before the injections, Orgels health had declined. He could not move his left arm or leg. He couldnt walk or stand on his own, he said.

Within a few days of having the stem cell treatment, Orgels constant muscle twitching diminished, said Bob Hubbard, director of stem cell therapy at the practice. Within weeks, he was able to walk in a pool of water and stand unassisted.

I think its helped me, said Orgel, who was a defensive coordinator at Auburn under former head coach Pat Dye. Im walking in the pool and I used to drag my feet. Now my left leg is picking up.

ALS is a progressive neuro-degenerative disease that affects nerve cells in the brain and the spinal cord. The progressive degeneration of the motor neurons in ALS eventually leads to death, according to the ALS Association.

Stem cells, sometimes called the bodys master cells, are precursor cells that develop into blood, bones and organs, according to the U.S. Food and Drug Administration, which regulates their use. Their promise in medicine, according to many scientists and doctors, is that the cells have the potential to help and regenerate other cells.

While Williams treatments are considered investigational, he has said, they meet FDA guidelines because the stem cells are collected from a patients fat tissue and administered back to that patient during the same procedure.

Orgel, 74, said Williams told him it would take between eight months to a year for his nerves to regrow. He is traveling to Gulf Shores from his home in Albany, Ga., this weekend for another stem cell treatment, Orgel said: I need to get to where I can walk.

In recent years, Orgel has gone to Mexico at least three times for different types of treatments, not sanctioned in the U.S. At least once, he said, he had placenta cells injected into his body. That didnt work, Orgel said. I didnt feel any better.

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Former Auburn coach getting stem cell treatments for Lou Gehrig's disease

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Public release date: 29-Jun-2012 [ | E-mail | Share ]

Contact: Elisabeth (Lisa) Lyons elyons@cell.com 617-386-2121 Cell Press

Latest annual citation reports confirm Cell Press delivers highly valued, highly cited research and reviews to the scientific community it serves

We are delighted to report that the new impact factors align with community perception and confirm that Cell Press continues to publish the highest impact research and reviews in the biomedical sciences, according to the latest Journal Citation Reports published by Thomas Reuters.

Cell Press's flagship journal Cell received an impressive impact factor of 32.403. Showing strong and steady growth, Cell's impact factor has increased by 9% since 2005, maintaining its status as the premier research journal in its field. Cell is currently ranked the number one research journal in the 'Cell Biology' and 'Biochemistry & Molecular Biology' categories.

Over 70% of journals within the Trends review journal series increased in impact factor this year, with significant growth across several life science disciplines. Top performers include Trends in Cognitive Science, which increased by 30% to 12.586, Trends in Immunology, which grew 9% to 10.403, and Trends in Ecology and Evolution, which rose 9% to 15.748. Published by Cell Press since 2007, Trends journals offer the unparalleled level of in-house editorial expertise that exists within all of the Cell Press journals, with the support of committed and enthusiastic editorial boards and an extensive range of fair and knowledgeable reviewers.

The substantial increase for Trends in Cognitive Sciences is also reflected in the other Cell Press neuroscience journals. Neuron, which has been publishing leading neuroscience research and reviews since 1988, increased by 5% to 14.736, and Trends in Neurosciences is up from 13.320 to 14.235.

"We are very pleased to see the scientific community's response to the work published in Cell Press journals. We are grateful to the authors who entrust their best work to us and to the reviewers who provide invaluable advice and guidance," said Emilie Marcus, Editor-in-Chief and CEO of Cell Press. "Cell Press editors work hard to maintain the high editorial standards expected of them by our authors and readers, and understand the importance of engaging with, and being accessible to, the life science research community which we are all proud to be a part of."

Cell Press's more recent journal launches, aimed at expanding our scope into translational biomedical areas, continue to maintain their influence within the scientific community. Launched in 2007, Cell Stem Cell has an impact factor of 25.421 and has been named a "Rising Star" in the field of Clinical Medicine by Thomson Reuters. This means that, in 2011, Cell Stem Cell had the highest percentage growth in citations in its field. Celebrating a decade of high impact publication in 2012, Cancer Cell has a well established impact factor of 26.566.

The 2011 Journal Citation Reports ranks the Cell Press journals' impact factors as follows:

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Cell Press journals continue to deliver high impact

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28-06-2012 14:46

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Stem Cell Therapy | Producers Direct | Kansas City, Missouri - Video

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COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics, Inc. (OSIR), announced today the expansion of its intellectual property protection around Prochymal (remestemcel-L). The United States Patent and Trademark Office recently granted Osiris two patents that cover multiple mechanisms of action related to cardiac tissue repair. Additionally, Osiris has enhanced its mesenchymal stem cell (MSC) patent estate with the issuance of patents across Europe and Australia covering stem cells expressing all therapeutically useful levels of cell surface receptors for TNF-alpha, a receptor essential to the cell's ability to counteract inflammation. These patents further support Osiris' considerable intellectual property position, which includes 48 issued U.S. patents around the production, composition, testing and use of the mesenchymal stem cell from both allogeneic and autologous sources.

"These recent additions to Osiris patent estate, combined with the existing broad coverage of our pioneering MSC platform technology, reinforce our industry leading IP portfolio and bolster our dominant position regarding the manufacture and use of mesenchymal stem cells for the treatment of a broad range of diseases, said Chris Alder, Chief Intellectual Property Counsel of Osiris. We have invested significant time and resources building our intellectual property estate, and with the commercialization of Prochymal, we are preparing to take the necessary action to enforce our considerable rights.

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program. With Prochymal (remestemcel-L) entering commerce, Osiris has initiated the process of identifying entities that may be infringing upon its intellectual property rights and will take appropriate action as necessary.

About Prochymal (remestemcel-L)

Prochymal is the worlds first approved drug with a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of MSCs, which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is approved in Canada and New Zealand for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Program. Prochymal is currently in a Phase 3 trial for refractory Crohns disease and is also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.

About Osiris Therapeutics

Osiris Therapeutics, Inc. is the leading stem cell company, having developed the worlds first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets. In Biosurgery, Osiris currently markets Grafix for burns and chronic wounds, and Ovation for orthopedic applications. Osiris is a fully integrated company with capabilities in research, development, manufacturing and distribution of stem cell products. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign patents.

Osiris, Prochymal, Grafix and Ovation are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, http://www.Osiris.com. (OSIRG)

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Osiris Bolsters its Stem Cell Intellectual Property Estate

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By Daily Mail Reporter

PUBLISHED: 07:58 EST, 28 June 2012 | UPDATED: 08:44 EST, 28 June 2012

Robin Roberts' older sister has spoken out about being her sole bone marrow donor after learning she was a match.

Sally-Ann, who anchors a morning show in New Orleans, will be essential to her GMA host sister's treatments for myelodysplastic syndrome (MDS), a blood and bone marrow disease.

The mother-of-three, 55, told the New York Post yesterday how she had been so desperate to be a match for her sister, she and her friends made a prayer circle around the test kit.

'We prayed, "please let this be a match,"' she admitted.

Perfect match: Sally-Ann Roberts, pictured with Robin earlier this month, has spoken about how she learned she would be her sister's sole bone marrow donor

She admitted: 'Im the big sister. Im the one whos supposed to be suffering because of age. But thats not the way it is.'

To donate her bone marrow, Sally-Ann explained that she will have five days of injections to boost her blood cell count, before her blood is passed through a machine that will extract the stem cells her sister, 51, so desperately needs.

'The way it is explained to me is that they will first have to knock out her immune system in order for my stem cells to be accepted by her body,' she said.

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'We prayed that I'd be a match': Robin Roberts' sister Sally-Ann on learning she was the sole bone marrow donor

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Main Category: Huntingtons Disease Also Included In: Stem Cell Research Article Date: 28 Jun 2012 - 9:00 PDT

Current ratings for: Huntington's Research Tool Developed Using Stem Cells

Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntington's disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journal's Aug. 3 issue, scientists at Cedars-Sinai's Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntington's disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntington's.

"This Huntington's 'disease in a dish' will enable us for the first time to test therapies on human Huntington's disease neurons," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. "In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. It's a new way of doing trailblazing science."

The Huntington's Disease iPSC Consortium united some of the world's top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntington's patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntington's researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntington's, known to the public, for example, as the cause of folksinger Woody Guthrie's death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntington's results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntington's, nor therapies to slow its progression.

The consortium showed Huntington's cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or "stressing" them, and found that Huntington's neurons died even faster.

"It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques," said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. "It was very reassuring and significantly strengthens the value of this study."

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Huntington's Research Tool Developed Using Stem Cells

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Newswise Researchers at the Buck Institute have corrected the genetic mutation responsible for Huntingtons Disease (HD) using a human induced pluripotent stem cell (iPSC) that came from a patient suffering from the incurable, inherited neurodegenerative disorder. Scientists took the diseased iPSCs, made the genetic correction, generated neural stem cells and then transplanted the mutation-free cells into a mouse model of HD where they are generating normal neurons in the area of the brain affected by HD. Results of the research are published in the June 28, 2012 online edition of the journal Cell Stem Cell.

iPSCs are reverse-engineered from human cells such as skin, back to a state where they can be coaxed into becoming any type of cell. They can be used to model numerous human diseases and may also serve as sources of transplantable cells that can be used in novel cell therapies. In the latter case, the patient provides a sample of his or her own skin to the laboratory. We believe the ability to make patient-specific, genetically corrected iPSCs from HD patients is a critical step for the eventual use of these cells in cell replacement therapy, said Buck faculty Lisa Ellerby, PhD, lead author of the study. The genetic correction reversed the signs of disease in these cells the neural stem cells were no longer susceptible to cell death and the function of their mitochondria was normal. Ellerby said the corrected cells could populate the area of the mouse brain affected in HD, therefore, the next stage of research involves transplantation of corrected cells to see if the HD-afflicted mice show improved function. Ellerby said these studies are important as now we can deliver patient-specific cells for cell therapy, that no longer have the disease causing mutation.

Huntington's disease (HD) is a devastating, neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. It typically becomes noticeable in mid-adult life, with symptoms beginning between 35 and 44 years of age. Life expectancy following onset of visual symptoms is about 20 years. The worldwide prevalence of HD is 5-10 cases per 100,000 persons. More than a quarter of a million Americans have HD or are "at risk" of inheriting the disease from an affected parent. Key to the disease process is the formation of specific protein aggregates (essentially abnormal clumps) inside some neurons.

All humans have two copies of the Huntingtin gene (HTT), which codes for the protein Huntingtin (Htt). Part of this gene is a repeated section called a trinucleotide repeat, which varies in length between individuals and may change between generations. When the length of this repeated section reaches a certain threshold, it produces an altered form of the protein, called mutant Huntingtin protein (mHtt). Scientists in the Ellerby lab corrected the mutation by replacing the expanded trinucleotide repeat with a normal repeat using homologous recombination. Homologous recombination is a type of genetic recombination where two molecules of DNA are exchanged. In this case the diseased DNA sequence is exchanged for the normal DNA sequence.

Contributors to the work: Mahru An and Ningzhe Zhang are shared first authors of this study. Other Buck Institute researchers involved in the study include Gary Scott, Daniel Montoro, Tobias Wittkop, and faculty members Sean Mooney and Simon Melov. The work was funded by the Buck Institute and the National Institutes of Health.

About the Buck Institute for Research on Aging The Buck Institute is the U.S.s first and foremost independent research organization devoted to Geroscience focused on the connection between normal aging and chronic disease. Based in Novato, CA, The Buck is dedicated to extending Healthspan, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimers and Parkinsons, cancer, cardiovascular disease, macular degeneration, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics and bioinformatics. For more information: http://www.thebuck.org.

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Scientists Correct Huntington's Mutation in Induced Pluripotent Stem Cells

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ScienceDaily (June 28, 2012) An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients.

The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

UCI scientists were part of a consortium that in 1993 identified the autosomal dominant gene mutation responsible for HD, but there is still no cure, and no treatments are available to even slow its onset or progression. The research, published online June 28 in the journal Cell Stem Cell, is the work of the Huntington's Disease iPSC Consortium. Participants examined several other cell lines and control cell lines to ensure that their results were consistent and reproducible in different labs.

"Our discovery will enable us for the first time to test therapies on human Huntington's disease neurons," said Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, one of the world's leading HD experts and a senior author of the study. "This has been a remarkable time in HD research, with the advent of stem cell technologies that have allowed these scientific advancements. Also, having a team of scientists working together as a consortium has benefited the research tremendously and accelerated its pace."

Leslie Lock, a UCI assistant professor of developmental & cell biology and biological chemistry whose lab helped develop the induced pluripotent stem cells (iPSC), added: "It's exciting to be carrying out work that provides hope for HD patients and their families."

Thompson said that UCI scientists will use the new model to study the specific gene expression changes in human brain cells that trigger the onset of HD, helping them understand how these changes happen and how to correct them.

Huntington's disease afflicts about 30,000 people in the U.S. -- typically striking in midlife -- and another 75,000 carry the gene that will eventually lead to it. Caused by a mutation in the gene for a protein called huntingtin, the disease damages brain cells so that individuals with HD progressively lose their ability to walk, talk and reason. It invariably culminates in death. While rare, HD is the most common inherited neurodegenerative disease.

Alvin King, Malcolm Casale, Sara Winokur, Gayani Batugedara, Marquis Vawter and Peter Donovan of UCI contributed to the study.

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Human model of Huntington's disease created from skin's stem cells

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Editor's Choice Main Category: Huntingtons Disease Also Included In: Stem Cell Research;Neurology / Neuroscience Article Date: 29 Jun 2012 - 14:00 PDT

Current ratings for: Brain Cells Derived From Skin Cells For Huntington's Research

At present, there is no cure for the disease and no treatments are available. These findings open up the possibility of testing treatments for the deadly disorder in a petri dish.

The study is the work of a Huntington's Disease iPSC Consortium, including researchers from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, and six other groups.

Huntington's disease is an inherited, deadly neurodegenerative disorder. The onset of HD generally occurs during midlife, although it can also strike in childhood - as in the patient who donated the material for the cells generated in this study. The disease causes jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and ultimately death.

Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the lead researchers of the study, explained:

The team are currently testing small molecules for the ability to block HP iPSC degeneration. According to the researchers, these molecules could potentially be developed into new drugs for Huntington's disease.

Furthermore, the teams ability to create "HD in a dish" may also have implications for similar research in other diseases such as Parkinson's and Alzheimer's.

In the study, the team took a skin biopsy from a 7-year-old patient with very early onset of severe HD. In the laboratory of Hongjun Song, Ph.D., a professor at Johns Hopkins' Institute for Cell Engineering, the skin cells were grown in culture and then created into pluripotent stem cells. In addition, a second cell line was created in the same way in Dr. Ross's lab from an individuals without HD.Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

Over three months, the researchers converted the cells into generic neurons and then into medium spiny neurons. The team discovered that the medium spiny neurons they created showed rapid degeneration without extensive supporting nutrients. Control cells lines on the other hand, showed no degeneration.

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Brain Cells Derived From Skin Cells For Huntington's Research

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Last April, when doctors first suspected that Good Morning America host Robin Roberts may have contracted a rare blood disease from the chemotherapy treatments she took for breast cancer, she showed up at her mothers house with a bag full of cheek swabs.

The swabs were to test her family to see if one of them could if worse came to worst be a bone-marrow donor.

Robins oldest sister, Sally-Ann, who anchors a morning show on the CBS station in New Orleans, says she and a friend made a prayer circle around the test kit.

We prayed: Please let this be a match, Sally-Ann told The Post yesterday.

Roger Wong/INFphoto.com

MATCH: Robin Roberts sister Sally-Ann (right), a morning-show host in New Orleans, beat 1-in-4 odds to be donor.

AP

TOUGH: Robin shows off an elastic band Tuesday that covers the intravenous port where she gets medication.

And it worked.

Sally-Ann is going to be Robins sole bone-marrow donor for a series of treatments later this year.

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Sis to serve as Robin's bone-marrow donor

Recommendation and review posted by Bethany Smith

By Daily Mail Reporter

PUBLISHED: 07:58 EST, 28 June 2012 | UPDATED: 08:44 EST, 28 June 2012

Robin Roberts' older sister has spoken out about being her sole bone marrow donor after learning she was a match.

Sally-Ann, who anchors a morning show in New Orleans, will be essential to her GMA host sister's treatments for myelodysplastic syndrome (MDS), a blood and bone marrow disease.

The mother-of-three, 55, told the New York Post yesterday how she had been so desperate to be a match for her sister, she and her friends made a prayer circle around the test kit.

'We prayed, "please let this be a match,"' she admitted.

Perfect match: Sally-Ann Roberts, pictured with Robin earlier this month, has spoken about how she learned she would be her sister's sole bone marrow donor

She admitted: 'Im the big sister. Im the one whos supposed to be suffering because of age. But thats not the way it is.'

To donate her bone marrow, Sally-Ann explained that she will have five days of injections to boost her blood cell count, before her blood is passed through a machine that will extract the stem cells her sister, 51, so desperately needs.

'The way it is explained to me is that they will first have to knock out her immune system in order for my stem cells to be accepted by her body,' she said.

Originally posted here:
'We prayed that I'd be a match': Robin Roberts' sister Sally-Ann on learning she was the sole bone marrow donor

Recommendation and review posted by Bethany Smith