British scientists have launched a pioneering trial to see whether gene therapy can potentially replace heart transplants.

Lee Adams, a 37-year-old carpenter from Hertfordshire, is the first of 24 patients with advanced heart failure to be recruited.

Like all the other participants, he has been fitted with a mechanical pump to keep his blood flowing while waiting for a suitable donor heart.

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The study is the first in the world to investigate the use of gene therapy in heart failure patients kept alive by a Left Ventricular Assist Device (LVAD).

Sixteen randomly chosen patients will be treated with a corrective gene to help their hearts beat more strongly. Eight others are to receive a "dummy" placebo therapy.

Mr Adams, who has had an LVAD for more than two and a half years, does not know which group he is in because the trial is "blinded".

He said: "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients.

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Could This Gene Therapy Spell The End Of Heart Transplants?

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Newswise Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety of neural stem cell transplantation in patients with chronic spinal cord injuries. This Phase I clinical trial is recruiting eight patients for the 5-year study.

The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries, said Joseph Ciacci, MD, principal investigator and neurosurgeon at UC San Diego Health System. The studys immediate goal, however, is to determine whether injecting these neural stem cells into the spine of patients with spinal cord injury is safe.

Related goals of the clinical trial include evaluating the stem cell grafts survival and the effectiveness of immunosuppression drugs to prevent rejection. The researchers will also look for possible therapeutic benefits such as changes in motor and sensory function, bowel and bladder function, and pain levels.

Patients who are accepted for the study will have spinal cord injury to the T7-T12 level of the spines vertebrae and will have incurred their injury between one and two years ago.

All participants will receive the stem cell injection. The scientists will use a line of human stem cells approved by the U.S. FDA for human trials in patients with chronic traumatic spinal injuries. These cells were previously tested for safety in patients with amyotrophic lateral sclerosis (ALS).

Since stem cell transplantation for spinal cord injury is just beginning clinical tests, unforeseen risks, complications or unpredictable outcomes are possible. Careful clinical testing is essential to ensure that this type of therapy is developed responsibly with appropriate management of the risks that all medical therapies may present.

Pre-clinical studies of these cells by Ciacci and Martin Marsala, MD, at the UC San Diego School of Medicine, showed that these grafted neural stem cells improved motor function in spinal cord injured rats with minimal side effects indicating that human clinical trials are now warranted.

This clinical trial at UC San Diego Health System is funded by Neuralstem, Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The Center was recently created to advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy.

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Clinical Trial Evaluates Safety of Stem Cell Transplantation in Spine

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MDI Science Caf Regenerative Medicine and the End of Aging
What lies behind the remarkable potential of the human body to rebuild itself and why aren #39;t we better at it? Our imaginations have been captivated by the prospect of stem cells in our adult...

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14 hours ago The red swamp crayfish (Procambarus clarkii) is native to the southeastern United States. This species is a popular model organism for studies of the nervous system, and has been used to study fundamental mechanisms involved in the production of new neurons in the adult brain. Credit: Jeanne Benton

Researchers have strived for years to determine how neurons are produced and integrated into the brain throughout adult life. In an intriguing twist, scientists reporting in the August 11 issue of the Cell Press journal Developmental Cell provide evidence that adult-born neurons are derived from a special type of circulating blood cell produced by the immune system. The findingswhich were made in crayfishsuggest that the immune system may contribute to the development of the unknown role of certain brain diseases in the development of brain and other tissues.

In many adult organisms, including humans, neurons in some parts of the brain are continually replenished. While this process is critical for ongoing health, dysfunctions in the production of new neurons may also contribute to several neurological diseases, including clinical depression and some neurodegenerative disorders. Dr. Barbara Beltz of Wellesley College and her colleagues studied crayfish to understand how new neurons are made in adult organisms. When they marked the cells of one crayfish and used this animal as a blood donor for transfusions into another crayfish, the researchers found that the donor blood cells could generate neurons in the recipient.

"These blood cellscalled hemocyteshave functions similar to certain white blood cells in mammals and are produced by the immune system in a blood-forming organ that is functionally analogous to bone marrow," explains Dr. Beltz. "When these cells are released into the circulation, they are attracted to a specialized region in the brain where stem cells divide, and their descendants develop into functional neurons."

The current work demonstrates that the immune system can produce cells with stem cell properties that can give rise to different types of cells, including both hemocytes and nerve cells. "Our findings in crayfish indicate that the immune system is intimately tied to mechanisms of adult neurogenesis, suggesting a much closer relationship between the immune system and nervous system than has been previously appreciated," says co-author Dr. Irene Sderhll, of Uppsala University in Sweden. The flexibility of these immune cells in producing neurons in adult animals raises the intriguing possibility of the presence of similar types of flexibility in other animals. If further studies demonstrated a similar relationship between the immune system and brain in mammals, the findings would stimulate a new area of research into immune therapies to target neurological diseases.

Explore further: New discovery on early immune system development

More information: Developmental Cell, Benton et al.: "Cells from the immune system generate adult-born neurons in crayfish." http://www.cell.com/developmental-cel 1534-5807(14)00405-5

Journal reference: Developmental Cell

Provided by Cell Press

Researchers at Lund University have shed light on how and when the immune system is formed, raising hope of better understanding various diseases in children, such as leukaemia.

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(PRWEB UK) 11 August 2014

BioEden the specialist tooth stem cell bank calls for a more intelligent approach, transparency and public education regarding stem cell banking.

"The public needs to be made aware that the success of stem cell medicine is largely dependant on the right material being available at the right time," says Tony Veverka Group CEO of the rapidly expanding specialist bank.

"With 1 in 3 people predicted to use stem cell therapy within their lifetime people need to know what their choices are at a time when they are able to do something about it, for example obtaining stem cells from their childrens naturally shed baby teeth."

BioEden pioneered the banking of stem cells from childrens baby teeth in 2006 in Austin Texas, and now operates in 21 countries.

BioEden says its unique process has many advantages over other forms and sources of stem cells, and eliminates the costly and painful process of getting stem cells from bone marrow for example.

The BioEden process is patent protected and offers the most natural form of stem cell banking that exists today.

"It is nonsense to say that a dental surgeon needs to extract a childs baby tooth in order to get the best result. The tooth falls out naturally and providing the stem cell bank offers quality transportation and processing, not even dental intervention is required," says Mr Veverka.

There are significant advantages in banking stem cells from teeth over cord blood for example, including the potential for a much wider therapeutic application, its non-invasive, not limited to the number of cells such as with cord blood during the birthing process, and is the least expensive form of private banking there is.

Banking your child's cells is the only way of ensuring a perfect stem cell match, eliminating the emotional distress caused when no match can be found.

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Newswise PHILADELPHIA Twenty years after the first identification of the BRCA1 gene, the University of Pennsylvanias Basser Research Center for BRCA will honor the geneticist credited with its founding with the second annual Basser Global Prize. The award will go to human genetics researchers and expert Mary-Claire King, PhD, American Cancer Society Research Professor of Genetics and Medicine at the University of Washington. King has been a pioneer in the development of experimental and bioinformatics genomics tools to study common, complex human diseases and health conditions.

The Basser Global Prize provides $200,000 in unrestricted support of the winner's innovative BRCA1/2 related research efforts. As part of the award, King will give the keynote address at the annual Basser Research Center for BRCA Symposium scheduled for May 11-12, 2015, at which time she will receive the Basser Trophy and a personal $10,000 cash prize.

Were very excited to honor Dr. Kings accomplishments in BRCA-related research, particularly as this year marks twenty years since the initial cloning of the BRCA1 gene, said Susan Domchek, MD, executive director of the Basser Research Center and the Basser Professor of Medicine in Penns Abramson Cancer Center. The identification of BRCA1 was the first critical step in work to improve outcomes for individuals with inherited susceptibility to breast cancer. Supporting research projects that are similarly devoted to the prevention and treatment of BRCA-related cancers is a primary mission of the Basser Center.

In 1990, Mary-Claire King demonstrated that a single gene on chromosome 17q21 (which she named BRCA1) was responsible for breast and ovarian cancer in many families. Her discovery of BRCA1 revolutionized the study of numerous other common inherited diseases. Dr. Kings current research focuses on identifying and characterizing critical genes and their interaction with environmental influences that play a role in the development of conditions such as breast and ovarian cancer, schizophrenia, and hearing loss.

In 2012, the Basser Center was established through a $25 million gift from Penn alumni Mindy and Jon Gray in memory of Mindy Grays sister Faith Basser, who died of ovarian cancer at age 44. The Basser Global Prize, a marquee component of the center, was established by Shari Basser Potter and Leonard Potter to honor a visionary scientist who has conceptually advanced BRCA1/2 related research that has led to improvements in clinical care. Professor Alan Ashworth, FRS, Chief Executive Officer of the Institute for Cancer Research in London and leader of the Gene Function team in the ICRs Breakthrough Breast Cancer Research Centre, was named the inaugural recipient of the Basser Global Prize in 2013.

Earlier this year, Mindy and Jon Gray made an additional $5 million gift to launch the Basser External Research Grant Program, a unique funding program for high impact translational cancer research projects aimed at advancing the care of people living with BRCA1 and BRCA2 mutations. Penn Medicines Abramson Cancer Center will serve as steward of the $5 million grant to four research teams that demonstrate the potential for translation into clinical practice. The Basser Center also provides funding to Penn investigators whose efforts are making strides in BRCA related research. Recipients of the first Basser External Research Grant Program funding, and the third year of internal grant funding will be announced in the coming weeks.

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Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise. The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 17 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $392 million awarded in the 2013 fiscal year.

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Penn's Basser Research Center for BRCA Names BRCA1 Founder Mary-Claire King Winner of the 2014 Basser Global Prize

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A new research has helped discover the gene, which plays a crucial role in formation of blood vessel.

Professor David Beech at the University of Leeds, who led the research, said that the blood vessel networks were not already pre-constructed but emerged rather like a river system. Vessels do not develop until the blood is already flowing and they are created in response to the amount of flow. The gene, Piezo1, provides the instructions for sensors that tell the body that blood is flowing correctly and gives the signal to form new vessel structures.

The gene gives instructions to a protein which forms channels that open in response to mechanical strain from blood flow, allowing tiny electrical charges to enter cells and trigger the changes needed for new vessels to be built, he added.

The research team further plans to study the effects of manipulating the gene on cancers, which require a blood supply to grow, as well as in heart diseases such as atherosclerosis, where plaques form in parts of blood vessels with disturbed blood flow.

(Posted on 11-08-2014)

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Contact: Randy Sachs randel.sachs@childrens.com 214-456-1523 UT Southwestern Medical Center

DALLAS Aug. 11, 2014 Researchers at the Childrens Medical Center Research Institute at UT Southwestern (CRI) have identified a gene that contributes to the development of several childhood cancers, in a study conducted with mice designed to model the cancers. If the findings prove to be applicable to humans, the research could lead to new strategies for targeting certain childhood cancers at a molecular level. The study was published today in the journal Cancer Cell.

We and others have found that Lin28b a gene that is normally turned on in fetal but not adult tissues is expressed in several childhood cancers, including neuroblastoma, Wilms tumor and hepatoblastoma, a type of cancer that accounts for nearly 80 percent of all liver tumors in children, said Dr. Hao Zhu, a principal investigator at CRI, and Assistant Professor of Pediatrics and Internal Medicine at UT Southwestern Medical Center. In our study, we found that overproduction of Lin28b specifically causes hepatoblastoma, while blocking Lin28b impairs the cancers growth. This opens up the possibility that pediatric liver cancer patients could one day be treated without resorting to chemotherapy.

Lin28b is an attractive therapeutic target in cancer because it is ordinarily only expressed in embryos, so blocking it in children should specifically hinder cancer growth without introducing many side effects.

Each year in the United States, 700 children are newly diagnosed with neuroblastoma, 500 with Wilms tumor and 100 with hepatoblastoma. At Childrens Medical Center in Dallas, more than 100 children have been treated for those three types of cancers over the last two years.

Previous studies found that Lin28b is a critical factor in stem cell and fetal tissue development, leading Dr. Zhu and his team to hypothesize that the same gene would play a significant role in the development of certain cancers.

We looked at Lin28b in a multitude of ways in mice to study its effects on cancer, from increasing it significantly to deleting it, said Dr. Zhu, co-senior author of the paper. From this and earlier studies, it appears that Lin28b activates the metabolic pathways that provide the building blocks of growth for certain cancers.

The next step for the Zhu lab is to establish whether genes related to Lin28b have similar effects on the development of cancer, and to determine if those genes might be more effective targets for potential therapies.

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Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 11, 2014 -- Nearly 6% of all children and teens in the U.S. are severely obese, and the prevalence of severe obesity is increasing faster than that of moderate obesity or overweight. This is an alarming trend as about 90% of these youths will grow up to be obese adults. The serious health problems associated with severe obesity and the poor long-term prognosis and quality of life projected for these children and teens demand more serious consideration of safe and effective treatment options that go beyond diet and lifestyle modifications, as proposed in an Editorial published in Childhood Obesity, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Childhood Obesity website at http://online.liebertpub.com/doi/full/10.1089/chi.2014.1041 until September 12, 2014.

In "Pediatric Severe Obesity: Time to Establish Serious Treatments for a Serious Disease," Stephen R. Daniels, MD, PhD, University of Colorado School of Medicine and Children's Hospital Colorado (Aurora), and Aaron S. Kelly, PhD, University of Minnesota Medical School and Children's Hospital (Minneapolis), note that while healthy lifestyle changes made during childhood may be quite helpful in weight reduction, these less intensive types of approaches tend to be less effective in treating severely obese teenagers. According to the authors, better access to specialty medical weight management programs, pharmacotherapy, and weight loss surgery are all important components of a more comprehensive strategy to combat severe obesity among teens.

"Drs. Daniels and Kelly are performing a vital service by directing our attention to this serious and increasingly prevalent problem," says David L. Katz, MD, MPH, Editor-in-Chief of Childhood Obesity and Director, Yale University Prevention Research Center. "We need commensurately serious solutions which I believe can include lifestyle interventions, but only of adequate scope and intensity. Just as lifestyle has been proven a worthy alternative to coronary bypass surgery, our sons and daughters deserve alternatives to bariatric surgery in combating this problem that our culture has handed them."

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About the Journal

Childhood Obesity is a bimonthly journal, published in print and online, and the journal of record for all aspects of communication on the broad spectrum of issues and strategies related to weight management and obesity prevention in children and adolescents. The Journal includes peer-reviewed articles documenting cutting-edge research and clinical studies, opinion pieces and roundtable discussions, profiles of successful programs and interventions, and updates on task force recommendations, global initiatives, and policy platforms. It reports on news and developments in science and medicine, features programs and initiatives developed in the public and private sector, and includes a Literature Watch. Tables of content and a sample issue may be viewed on the Childhood Obesity website at http://www.liebertpub.com/chi.

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More intensive interventions needed to combat severe obesity in teens

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11-Aug-2014

Contact: Dana Weidig weidigd@email.chop.edu 267-426-6092 Children's Hospital of Philadelphia

How genes affect intelligence is complicated. Multiple genes, many yet unknown, are thought to interact among themselves and with environmental factors to influence the diverse abilities involved in intelligence.

A large new genetic study in thousands of children and adolescents offers early glimpses of the overall patterns and connections among cognitive abilities such as language reasoning, reading skill and types of memory. The findings may lead to new tools in understanding human cognitive development and neuropsychiatric disorders.

"This research is one of the first to use a molecular genetic approach to evaluate complex cognitive traits in a pediatric sample," said one of the study's two co-senior leaders, Hakon Hakonarson, M.D., Ph.D., director of the Center for Applied Genomics at The Children's Hospital of Philadelphia. "Uncovering the genetic architecture of these diverse cognitive abilities may offer new insights into cognitive development and may ultimately allow investigators to identify useful biomarkers for diagnosing and predicting risks of neuropsychiatric conditions."

The study appeared online July 15 in Molecular Psychiatry. Hakonarson's co-senior author is psychiatrist Raquel Gur, M.D., Ph.D., director of Neuropsychiatry in the Perelman School of Medicine at the University of Pennsylvania. "We are beginning to reap the benefits of the major investment we made in recruiting this highly informative youth cohort," said Gur. She added, "They were genotyped and underwent 'deep phenotyping' that included medical, neuropsychiatric, and neurocognitive evaluations and multi-modal neuroimaging in a large subsample. This cohort will continue to yield novel information on how genes and environment interact to produce a mature, healthy brain and will elucidate how aberrations in brain function lead to cognitive deficits and psychopathology."

The study team represents a collaboration among the CHOP and Penn investigators with colleagues at the Broad Institute, Harvard Medical School and Massachusetts General Hospital (MGH) who developed a powerful gene software tool called genomewide complex trait analysis (GCTA). The study's first author is Elise B. Robinson, Ph.D., of the MGH Analytic and Translational Genetics Unit, led by Mark Daly, Ph.D.

The researchers used GCTA to analyze a subset of 3,689 individuals aged 8 to 21, all of European ancestry, drawn from the Philadelphia Neurodevelopmental Cohort, a general-population sample of close to 10,000 individuals who received care within CHOP's pediatric network for a broad range of health needs. The researchers performed genotypes of all participants, administered a battery of neurocognitive tests and assessed participants in structured psychiatric interviews.

The GCTA analyzes common SNPs (single-nucleotide polymorphisms, changes of a single base in DNA) to estimate how much these common gene variants contribute to differences in cognitive abilities within the total sample. The study team grouped cognitive traits within five broad domains: executive function, memory, complex cognition, social cognition and reading ability.

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11-Aug-2014

Contact: Lisa Anderson lisama2@gwu.edu 202-994-3121 George Washington University

WASHINGTON (Aug. 11, 2014) According to a recent World Health Organization report of the leading causes of death worldwide, one-third of all deaths are due to infectious and parasitic diseases. There are currently no vaccines for parasitic nematode (PN), or worm infections in humans, and development of new drugs and vaccines will stall until researchers have a better understanding of PN biology.

John Hawdon, Ph.D., associate professor of microbiology, immunology, and tropical medicine at the George Washington University (GW) School of Medicine and Health Sciences, was recently awarded $430,722 from the National Institutes of Health to develop a model system to study PN infection, which will lead to greater understanding of the infective process and the host's immune response to infection.

Due to the requirement of an obligate host and lack of good animal models, investigations have been previously limited. However, thanks to an interdisciplinary team, which includes Hawdon, Damien O'Halloran, Ph.D., and Ioannis Eleftherianos, Ph.D., both assistant professors of biology at the GW Columbian College of Arts and Sciences, a viable model will be developed using the insect PN Heterorhabditis bacteriophora.

"The techniques we are developing will allow us to practice RNA interference and transgenesis, which will allow us to insert copies of DNA into the nematode, look at gene function, and knock out certain genes," said Hawdon. "This research fills a need for further development of these techniques in this worm."

The research team will focus on targeting the infective juvenile and early parasitic stages in order to investigate infection mechanisms. Their approaches will provide a foundation for future investigation into the mechanism of PN infection.

The development of these methods for gene knock down, or reverse genetics, and transfection in this novel model nematode would represent a significant advance for PN research, and will facilitate investigation of PN gene function during infection.

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Newswise INDIANAPOLIS -- Researchers have discovered a previously unknown cardiac molecule that could provide a key to treating, and preventing, heart failure.

The newly discovered molecule provides the heart with a tool to block a protein that orchestrates genetic disruptions when the heart is subjected to stress, such as high blood pressure.

When the research team, led by Ching-Pin Chang, M.D., Ph.D., associate professor of medicine at the Indiana University School of Medicine, restored levels of the newly discovered molecule in mice experiencing heart failure, the progression to heart failure was stopped. The research was published in the online edition of the journal Nature.

The newly discovered molecule is known as a long non-coding RNA. RNA's usual role is to carry instructions -- the code -- from the DNA in a cell's nucleus to the machinery in the cell that produces proteins necessary for cell activities. In recent years, scientists have discovered several types of RNA that are not involved in protein coding but act on their own. The role in the heart of long non-coding RNA has been unknown.

But the researchers determined that the newly discovered non-coding RNA, which they named Myheart -- for myosin heavy-chain-associated RNA transcript -- is responsible for controlling a protein called BRG1 (pronounced "berg-1"). In earlier research published in Nature in 2010, Dr. Chang and his colleagues discovered that BRG1 plays a crucial role in the development of the heart in the fetus.

But as the heart grows and needs to mature into its adult form, BRG1 is no longer needed, so very little of it is produced. That is, until the adult heart is subjected to significant stress such as high blood pressure or damage from a heart attack. Dr. Chang's previous research showed that in those conditions, BRG1 re-emerges and begins altering the heart's genetic activity, leading to heart failure. At the same time, production of Myheart is suppressed, so BRG1 can latch onto the DNA and alter the genetic material unchecked.

In the current Nature paper, the researchers reported that in mice with stress-induced high levels of BRG1, they were able to restore Myheart to normal levels using gene transfer technology. Restoring Myheart levels blocked BRG1 actions and prevented heart failure, they said.

"I think of Myheart as a molecular crowbar that pries BRG1 off the genomic DNA and prevents it from manipulating genetic activity," said Dr. Chang, director of molecular and translational medicine at the Krannert Institute of Cardiology.

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Lets Play The Sims 3 Perfect Genetics-100 Baby Challenge Live Stream Part 14
Perfect Genetics and 100 Baby Challenge all in 1 -- Watch live at http://www.twitch.tv/gbabychallenger.

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Minecraft: Block Party #19 - ADVANCED GENETICS (Yogscast Complete Mod Pack)
Join brent on the Block Party as he starts working with ADVANCED GENETICS a mod where you can mutate and gain powers. What will be his first goal?! FOLLOW Br...

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Attack of the B-Team with JtP Ep: 16 Advanced Genetics
Subscribe: http://goo.gl/z30fiY Twitter: http://goo.gl/de2h7n Donate: http://goo.gl/bjFNzv Twitch: http://goo.gl/42LwF3 ------------------------------- The HyperTeam Server is owned...

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If successful the heart muscle would be able to pump blood around the body without any help.

Mr Adams, of Hertfordshire, is the first person in the world to take part in the trial.

"It took some getting used to, living with a (heart pump), he said.

You can't just jump in the bath or the shower and it's difficult sleeping whilst being attached to it. Everywhere I go I have to carry the power supply and spare batteries in a backpack.

"Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients.

If it does prove to be successful it would be exciting for patients who need a transplant but end up on the waiting list for a long time because of the shortage of donors."

Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK.

Some patients with advanced heart failure are fitted with a Left Ventricular Assist Device (LVAD), a mechanical pump which supports the failing heart and aims to restore normal blood flow.

The pump moves the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body.

Currently there are around 100 to 150 people in the UK living with a pump.

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Groundbreaking gene therapy trial offers hope to heart patients

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11-Aug-2014

Contact: Franca Davenport f.davenport@imperial.ac.uk 44-207-594-3415 Imperial College London

For the first time in the world, a patient with a mechanical heart pump has taken part in a new gene therapy trial for heart failure at Harefield Hospital, London.

This is the start of a new clinical trial that will assess gene therapy for patients with heart pumps and provide detailed insight on its impact on the heart muscle.

Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK. Some individuals with an advanced heart failure can be fitted with a Left Ventricular Assist Device (LVAD), which supports the failing heart and aims to restore normal blood flow.

The LVAD is an electrically driven pump, moving the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body. Individuals with advanced heart failure who require a transplant may be fitted with an LVAD to keep them alive until a suitable donor heart becomes available. Currently there are around 100 to 150 people in the UK living with an LVAD.

The new trial, led by Imperial College London and funded by the British Heart Foundation (BHF) and Celladon Corporation, will explore whether this gene therapy could help these patients' hearts recover and potentially provide an alternative treatment. It is the first study of gene therapy in this patient group. The work was initiated with support from the Leducq Foundation.

This particular gene therapy is designed to increase levels of SERCA2a protein in heart muscle cells. SERCA2a plays an important role in heart muscle contraction. Genes are inserted into the heart muscle cells to increase the level of SERCA2a using a harmless engineered virus that is based on a naturally occurring virus. In this study the research team will take small biopsy samples of the heart muscle six months after treatment to measure if the gene is present, detectable and functional in the patients' hearts.

Professor Sian Harding, Professor of Cardiac Pharmacology and Head of the BHF Centre of Regenerative Medicine in Imperial College London, who helped develop the treatment, said: "We will be using state-of-the art methods to gain detailed information on how and where the gene therapy takes effect, which will potentially help us develop and improve the therapy. It's important to remember that the therapy is not correcting a gene defect. We are working much more downstream, which means that no matter what the cause of the heart failure, the therapy should be equally beneficial for patients whether their heart problems stem from genes, lifestyle or the environment or a mixture of all of these."

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First gene therapy trial launched for heart patients with mechanical pumps

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The therapy involves injecting a harmless altered virus into the blood stream [GETTY/PIC POSED BY MODEL]

Lee Adams, a 37-year-old carpenter, is the first patient in the world to take part in a gene therapy trial while wired up to a mechanical heart pump.

The study is to investigate the use of the therapy on 24 patients with advanced heart failure, recruited from Harefield Hospital, London, and Papworth Hospital, Cambridgeshire.

All the participants are kept alive by a Left Ventricular Assist Device while they wait for heart transplants.

Many such patients endure agonising delays in finding a suitable donor.

Sixteen randomly chosen patients will be treated with a corrective gene to help their hearts beat more strongly.

Eight others will receive a dummy placebo therapy.

Lee, from Rickmansworth, Herts does not know which group he is in.

He said: Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a miracle cure.

No matter what the cause of the heart failure, the therapy should be equally beneficial for patients

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Gene therapy could replace heart transplant operations

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Stamford, CT (PRWEB) August 11, 2014

Alliance for Cancer Gene Therapy, Inc. (ACGT) the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer has announced that David Darst and Alexandra Landes have joined the foundations Board of Directors. Both Darst and Landes lost their mothers to cancer and share a commitment to uncovering innovative methods for treating cancer with reduced side-effects.

ACGT is based in Stamford, CT and funds top physicians and researchers at medical institutions in the US and Canada. The foundation supports a number of gene therapy treatments including immunotherapy, which activates patients own immune systems to battle cancerous cells. Recently, the FDA granted fast-track status to an immunotherapy treatment for leukemia for which ACGT provided early funding. ACGT has given almost $25 million in grants to researchers since its founding in 2001 by Barbara Netter and her late husband, Edward.

David Darst, a 32-year-old New York City resident, is a graduate of Harvard College and Harvard Business School. Darst has held positions with Pfizer, Orbimed Advisors, and a slate of other companies that are working to develop and finance new drugs in the biotech industry. Darst is currently the CEO at Rgenix, Inc., where he leads the companys drug development initiatives and fundraising activities. He is also the co-founder and former Executive Vice President and Director of Potentia Pharmaceuticals, a biotech that achieved breakthroughs in treating age-related macular degeneration.

I was impressed by the quality of the science funded by ACGT, and its positive impact on cancer patients' lives, Darst said. It's an organization I know well, as my mother, Diane W. Darst, was a founding Director. I look forward to serving on its Board and continuing to help fund promising gene therapy research by leading young academic investigators.

Alexandra Landes, a 28-year-old Pacific Palisades, CA resident, is a graduate of Tufts University. Landes is Executive Director of Wendy Walk, a national nonprofit named for her late mother that has raised $2 million since 2010 to combat Liposarcoma. Landes is an accomplished development professional who has held leadership and board positions at the Metropolitan Council, Seeds of Peace, and the NYC Public Advocates Office.

Alliance for Cancer Gene Therapys goal of making cancer a treatable, manageable disease is so compelling, Landes said. Our current options for battling cancer take a distressing toll on patients its encouraging to know less harrowing methods are becoming available.

David has that rare expertise of identifying how new, creative treatments dovetail with financing and fundraising, said Barbara Netter, Co-Founder and President of ACGT. Alexandra has a deep commitment to the cause, and a profound understanding of how immunotherapy can revolutionize the way we treat cancer. Together, David and Alexandra will help to further introduce ACGTs mission to younger supporters and advocates.

About Alliance for Cancer Gene Therapy (ACGT) Established in 2001, ACGT (http://www.acgtfoundation.org) is the nations only not-for-profit exclusively dedicated to cell and gene therapy treatments for all types of cancer. One-hundred percent of contributions go directly to research. ACGT has funded 44 grants in the U.S. and Canada since its founding in 2001 by Barbara Netter and her late husband, Edward, to conduct and accelerate critically needed innovative research. ACGTs Scientific Advisory Council, comprised of 16 of the nations most distinguished physicians and researchers in cell and gene therapy, thoroughly review all grants. Since its inception, ACGT has awarded 29 grants to Young Investigators and 15 grants to Clinical Investigators, totaling $24.7 million in funding. Barbara Netter, as President, together with other members of the Board of Directors, are fully committed to ACGTs continued support of this research. ACGT is located at 96 Cummings Point Road, Stamford, CT 06902.

ACGT on Facebook: http://www.facebook.com/ACGTfoundation ACGT on Twitter: http://www.twitter.com/ACGTfoundation ACGT on YouTube: http://www.youtube.com/user/ACGTfoundation

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Alliance for Cancer Gene Therapy (ACGT) Introduces New Board Members David Darst and Alexandra Landes

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LONDON (Reuters) - A 37-year-old British man who needs a mechanical pump to keep his heart working has kicked off tests to see if gene therapy could help him recover and potentially avoid the need for a heart transplant.

U.S. biotech firm Celladon hopes that its Mydicar treatment can help patients like Lee Adams who have advanced heart failure and rely on so called Left Ventricular Assist Devices (LVAD) to keep them alive until a donor heart becomes available.

Celladon's treatment works by inserting a gene called SERCA2a - the lack of which makes hearts pump weakly - directly into heart cells via a catheter to repair them. The trial will evaluate how much of the gene is getting to the heart muscle and how well it is working.

Adams, from Hertfordshire, north of London, is the first of 24 patients with heart pumps who will be given either the gene therapy or a placebo as part of a clinical study partially funded by the British Heart Foundation and sponsored by Imperial College London.

"Advanced heart failure is a progressive condition that results in a poor quality of life and shortened life expectancy," said Dr Nick Banner, the consultant cardiologist at Harefield Hospital who carried out the first infusion.

"The best treatment currently available is a heart transplant but the shortage of donor organs in the UK means that many patients will die on the transplant waiting list."

Adams is on the waiting list and has been living for more than two and a half years with an LVAD, which must always be connected to an external power supply via a lead through his abdomen.

"You can't just jump in the shower...and it's difficult sleeping whilst being attached to it. Everywhere I go I have to carry the power supply and spare batteries in a backpack," he said.

"Of course the best thing that could happen would be...for the gene therapy to prove to be a 'miracle cure' for myself and other patients."

Plans for the study were first announced last year and complement an ongoing 250-patient trial, also funded by Celladon, which is investigating the benefits of gene therapy in 250 people with less advanced heart failure from Europe and the United States.

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Gene therapy clinical trial gives hope to heart failure patients wait-listed for a heart transplant

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InVivo makes strides against spinal cord injury

By: Venture Portfolio Mag

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InVivo makes strides against spinal cord injury - Video

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11 hours ago Cells grown on hydrogels of the same stiffness all display fat cell markers and deform the underlying matrix material the same way. Credit: Adam Engler, UC San Diego Jacobs School of Engineering

Bioengineers at the University of California, San Diego have proven that when it comes to guiding stem cells into a specific cell type, the stiffness of the extracellular matrix used to culture them really does matter. When placed in a dish of a very stiff material, or hydrogel, most stem cells become bone-like cells. By comparison, soft materials tend to steer stem cells into soft tissues such as neurons and fat cells. The research team, led by bioengineering professor Adam Engler, also found that a protein binding the stem cell to the hydrogel is not a factor in the differentiation of the stem cell as previously suggested. The protein layer is merely an adhesive, the team reported Aug. 10 in the advance online edition of the journal Nature Materials.

Their findings affirm Engler's prior work on the relationship between matrix stiffness and stem cell differentiations.

"What's remarkable is that you can see that the cells have made the first decisions to become bone cells, with just this one cue. That's why this is important for tissue engineering," said Engler, a professor at the UC San Diego Jacobs School of Engineering.

Engler's team, which includes bioengineering graduate student researchers Ludovic Vincent and Jessica Wen, found that the stem cell differentiation is a response to the mechanical deformation of the hydrogel from the force exerted by the cell. In a series of experiments, the team found that this happens whether the protein tethering the cell to the matrix is tight, loose or nonexistent. To illustrate the concept, Vincent described the pores in the matrix as holes in a sponge covered with ropes of protein fibers. Imagine that a rope is draped over a number of these holes, tethered loosely with only a few anchors or tightly with many anchors. Across multiple samples using a stiff matrix, while varying the degree of tethering, the researchers found no difference in the rate at which stem cells showed signs of turning into bone-like cells. The team also found that the size of the pores in the matrix also had no effect on the differentiation of the stem cells as long as the stiffness of the hydrogel remained the same.

"We made the stiffness the same and changed how the protein is presented to the cells (by varying the size of the pores and tethering) and ask whether or not the cells change their behavior," Vincent said. "Do they respond only to the stiffness? Neither the tethering nor the pore size changed the cells."

"We're only giving them one cue out of dozens that are important in stem cell differentiation," said Engler. "That doesn't mean the other cues are irrelevant; they may still push the cells into a specific cell type. We have just ruled out porosity and tethering, and further emphasized stiffness in this process."

Explore further: Researchers find stem cells remember prior substrates

More information: Interplay of matrix stiffness and protein tethering in stem cell differentiation, Nature Materials, DOI: 10.1038/nmat4051

(Phys.org) A team of researchers working at the University of Colorado has found that human stem cells appear to remember the physical nature of the structure they were grown on, after being moved to a ...

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Matrix stiffness is an essential tool in stem cell differentiation, bioengineers report

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Infusing stem cells into the brain may help boost recovery after a stroke, according to a pilot study by Imperial College London.

Scientists believe the cells encourage new blood vessels to grow in damaged areas of the brain.

They found most patients were able to walk and look after themselves independently by the end of the trial, despite having suffered severe strokes.

Larger studies are needed to evaluate whether this could be used more widely.

In this early trial - designed primarily to look at the safety of this approach - researchers harvested stem cells from the bone marrow of five people who had recently had a stroke.

'Independent living'

They isolated particular types of stem cells - known as CD34+. These have the ability to stimulate the growth of new blood vessels.

They were infused directly into damaged sections of the brain, via the major artery that supplies this area.

Scientists monitored the patients for six months, charting their ability to carry out everyday activities independently.

Four of the five patients had suffered particularly severe strokes - resulting in the loss of speech and marked paralysis down one side of the body.

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'Stem Cells Show Promise In Stroke Recovery'

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Beverly Hills, California (PRWEB) August 11, 2014

Nationally recognized Beverly Hills orthopedic surgeon, Dr. Raj, is now offering stem cell procedures to help patients avoid the need for surgery. This may include joint replacement along with tendon or ligament surgery. For more information on stem cell therapy and scheduling at the Beverly Hills Orthopedic Institute, call (310) 247-0466.

As the benefit of stem cells for repair and regeneration of human tissue has evolved, the opportunity to avoid surgery has too. This includes those with cartilage degeneration, tendonitis and ligament injury. Dr. Raj has been a pioneer in bringing stem cell therapies to the forefront, and is now offering the procedures to all patients.

According to Celebrity Fitness Expert Dr. Raj, a nationally recognized Double Board Certified Orthopedic Surgeon at the Beverly Hills Orthopedic Institute, stem cell injections are being used to heal conditions that used to require surgery. Dr. Raj has been featured on The Doctors, SPIKE TV, NBC, CBS, Martha Stewart Living Radio, Beverly Hills Times and has been named Best of LA by KCAL 9 as well we making Americas Top Orthopedics List in 2007, 2009, 2010, and 2011.

Dr. Raj explains that stem cells have started a medical revolution and have altered the way doctors approach treatment. Stem cells help to regenerate the damaged cartilage within a joint and allow patients to take a more conservative route, adds Dr. Raj. Surgery should be a last case scenario after all other options have been exhausted.

For those individuals suffering from joint arthritis of the hip, knee, shoulder or ankle, surgery is an elective decision with nonsurgical options that traditionally did not alter the course of arthritis. They have merely acted as a "band aid" for pain relief, but not effective at long term relief due to healing arthritis.

Stem cell therapy offers the opportunity for relief and increased activity, while staying out of the operating room. The procedures are outpatient and safe, with minimal risks involved.

For more information on the stem cell procedures provided with the top orthopedic doctor in Los Angeles and Beverly Hills, call (310) 247-0466.

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Nationally Recognized Beverly Hills Orthopedic Surgeon, Dr. Raj, Now Offering Stem Cell Procedures to Help Patients ...

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PUBLIC RELEASE DATE:

10-Aug-2014

Contact: Ben Jones B.P.Jones@leeds.ac.uk 44-011-334-38059 University of Leeds

Scientists from the University of Leeds have discovered a gene that plays a vital role in blood vessel formation, research which adds to our knowledge of how early life develops.

The discovery could also lead to greater understanding of how to treat cardiovascular diseases and cancer.

Professor David Beech, of the School of Medicine at the University of Leeds, who led the research, said: "Blood vessel networks are not already pre-constructed but emerge rather like a river system. Vessels do not develop until the blood is already flowing and they are created in response to the amount of flow. This gene, Piezo1, provides the instructions for sensors that tell the body that blood is flowing correctly and gives the signal to form new vessel structures.

"The gene gives instructions to a protein which forms channels that open in response to mechanical strain from blood flow, allowing tiny electrical charges to enter cells and trigger the changes needed for new vessels to be built."

The research team is planning to study the effects of manipulating the gene on cancers, which require a blood supply to grow, as well as in heart diseases such as atherosclerosis, where plaques form in parts of blood vessels with disturbed blood flow.

Professor Beech added: "This work provides fundamental understanding of how complex life begins and opens new possibilities for treatment of health problems such as cardiovascular disease and cancer, where changes in blood flow are common and often unwanted.

"We need to do further research into how this gene can be manipulated to treat these diseases. We are in the early stages of this research, but these findings are promising."

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Scientists unlock key to blood vessel formation

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