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New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia

Posted: Published on July 25th, 2012

ScienceDaily (July 25, 2012) A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published in Human Gene Therapy. The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zo Coulombe, and Joel Rousseau, Laval University, Quebec,and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia. The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors. "This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene … Continue reading

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US stem cell coup for Horizon

Posted: Published on July 25th, 2012

Technology from Cambridge UK personalised medicines pioneer, Horizon Discovery, is being used for a major stem cell project in the US. The agreement will enable the NIH CRM to apply Horizons GENESIS genome editing technology to engineer stem cells in order to create isogenic disease models with induced mutations and/or lineage markers. These disease models will be used to help researchers understand the effects of specific genes and mutations as stem cells differentiate, as well as create lineage reporters for stem cell differentiation. The NIH CRM was recently established through support from the NIH Common Fund to provide the infrastructure to support and accelerate the clinical translation of stem cell-based technologies, and to develop widely available resources to be used as standards in stem cell research. The GENESIS technology harnesses rAAV vectors to perform accurate and efficient gene-editing functions in human cells, by switching on a natural high-fidelity DNA-repair mechanism called homologous recombination (HR). When harnessed using rAAV gene-editing vectors, HR allows the precise alteration of any DNA sequence, permitting the accurate modelling of genetic diseases in human cells, including stem cells, in vitro. The use of these models in oncology is well established, and has predicted patient responses to … Continue reading

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New genetic test predicts prostate cancer risk

Posted: Published on July 25th, 2012

By Lynne Friedmann A genetic test to predict the risk for prostate cancer could reduce the need for repeat biopsies in men who have previously had negative biopsies. In a clinical trial, 1,654 men who had prostate biopsies also had genetic studies conducted that looked for the presence of genetic variations that may have an association with prostate cancer risk. The genetic test outperformed the widely used PSA (Prostate-Specific Antigen) test in assessing cancer risk. Because this genetic score is available at any time in a mans lifetime it could be used as a pre-screening test thus leaving aggressive PSA screening only to men at higher genetic risk. The goal is to avoid, particularly in older men, unnecessary repeat biopsy procedures which carry with them the risk of infection and potential hospitalizations. Findings appear in the journal of European Urology. News release at http://bit.ly/M7iaHV Inhibiting malaria parasite development Malaria is responsible worldwide for more than 1.2 million human deaths annually. Severe forms of the disease are caused by the parasite Plasmodium falciparum transmitted to humans by the bite of female Anopheles mosquitoes. Lack of vaccines, together with the parasites ability to develop drug resistance, has thwarted eradication efforts. An international … Continue reading

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Citizen researchers help unlock European genetic heritage

Posted: Published on July 25th, 2012

ScienceDaily (July 25, 2012) A University of Sheffield academic is helping a team of citizen scientists to carry out crucial research into European genetic heritage. Citizen Scientists are not required to have a scientific background or training, but instead they possess a passion for the subject and are increasingly being empowered by the scientific community to get involved in research. Dr Andy Grierson, from the University of Sheffield's Institute for Translational Neuroscience (SITraN), has helped a team of citizen scientists from Europe and North America to identify vital new clues to tell the story of Europe's genetic history. Dr Grierson explained: "Understanding European history since man first arrived on the continent is a huge challenge for archaeologists and historians. "One way that scientists can help is by studying the genetics of European men. All men carry a Y chromosome that they inherit from their father, which has been passed down the generations from father to son for thousands of years. So most men in Europe will share common ancestry at some point in the past, and we are able to investigate this shared ancestry using genetic studies of the Y chromosome. "However, up until recently, there have not been many … Continue reading

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Citizen science helps unlock European genetic heritage

Posted: Published on July 25th, 2012

Public release date: 25-Jul-2012 [ | E-mail | Share ] Contact: Amy Stone a.f.stone@sheffield.ac.uk 01-142-221-046 University of Sheffield A University of Sheffield academic is helping a team of citizen scientists to carry out crucial research into European genetic heritage. Citizen Scientists are not required to have a scientific background or training, but instead they possess a passion for the subject and are increasingly being empowered by the scientific community to get involved in research. Dr Andy Grierson, from the University of Sheffield's Institute for Translational Neuroscience (SITraN), has helped a team of citizen scientists from Europe and North America to identify vital new clues to tell the story of Europe's genetic history. Dr Grierson explained: "Understanding European history since man first arrived on the continent is a huge challenge for archaeologists and historians. "One way that scientists can help is by studying the genetics of European men. All men carry a Y chromosome that they inherit from their father, which has been passed down the generations from father to son for thousands of years. So most men in Europe will share common ancestry at some point in the past, and we are able to investigate this shared ancestry using genetic … Continue reading

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Research and Markets: Genetic Testing Market Report: 2012 Edition

Posted: Published on July 25th, 2012

DUBLIN--(BUSINESS WIRE)-- Research and Markets (http://www.researchandmarkets.com/research/j69n56/genetic_testing_ma) has announced the addition of the "Genetic Testing Market Report: 2012 Edition" report to their offering. The genetic testing market segment represents the highest growth opportunity compared to other segments within the molecular diagnostics market. The growth of the market is backed by increasing prevalence of genetic diseases, aging population, and rising awareness. Vast application areas including newborn screening, prenatal and preimplantation testing is also expected to add to the future growth of the market. The technological advancements of genetic testing are expected to break new grounds in the field of personalized medicine as genetic testing helps not only in the better diagnosis of genetic diseases but also aids in selecting the best medication depending on the DNA structure of the patient to ensure superior drug response. Direct to customer (DTC) genetic testing is one of the major trends in the market, which is expected to bring positive changes in the popularity of genetic testing. However, the quality, accuracy, and proper interpretation of test results of DTC genetic testing continue to challenge the future growth of the market. Furthermore, the risk of genetic discrimination and psychological consequences of genetic testing are also expected to … Continue reading

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Stem Cell Treatment Alleviated Lorna Tolentino's Back Pain

Posted: Published on July 25th, 2012

By ROWENA JOY A. SANCHEZ LT is noticeably more blooming these days (Publicity photo) MANILA, Philippines As if by stroke of magic, Lorna Tolentinos back pain was eliminated after getting stem cell treatment at a hospital in Germany on July 3. The whole process only took four days, which included a day for an interview about her health history. The second day was for the treatment itself, with Lorna receiving 10 injections. She rested on the third day and was discharged on the fourth. In a recent interview with Bulletin Entertainment and some members of the media, Lorna enthused, Ang instant [na nawala] sa akin yung back pain talaga Kasi iba yung pakiramdam ng wala kang iniinda na sakit sa likod. Dati pag lumalakad ako, mabagal Pag lumalakad ako pinupukpok ko na tong paa ko dahil nagna-numb na yung dito (part of her legs) ko. The back pain, she noted, was caused by scoliosis, a disorder that causes an abnormal curve of the spine, or backbone, according to medicinenet.com. Although her aura is noticeably more bloomingwhich can perhaps be attributed to the treatmentthe Kapatid actress emphasized that it will take three months before the changes completely take effect. Such immediate … Continue reading

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Seventeenth Patient Dosed in Neuralstem ALS Stem Cell Trial

Posted: Published on July 25th, 2012

ROCKVILLE, Md., July 25, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the seventeenth patient was treated in the ongoing Phase I trial of its spinal cord neural stem cells for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). This patient is also the second to return to the trial for additional injections. In this treatment, the patient received five injections in the cervical (upper back) region of the spinal cord, in addition to the ten he had previously received in the lumbar (lower back) region, for a total of 15 injections. The final previously treated patient of this cervical cohort is expected to return to the trial in August, provided the inclusion requirements continue to be met. This ground-breaking stem cell trial is taking place at Emory University Hospital in Atlanta, Georgia. (Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO ) "We are pleased that this phase of the trial, in which we have been permitted by the FDA to take the unprecedented step of dosing patients for the second time, is progressing as planned," said Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "These are the first patients in the world to receive our cells in both the lumbar … Continue reading

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Tracing the nano-landscape

Posted: Published on July 25th, 2012

Mumbai, July 25: How big is small? Nano technologies are igniting innovations across the world. And tracing this nanoscape the complex pathways from nanoventing to the commercialisation of nanovations is the book Nanotechnology Intellectual Property Rights: Research, Design, and Commercialization. The inclusive nature of nanotechnology gives it a very special status as it mothers innovations to deliver inventions in nanobiotechnology, nanostructures, nanocomposites, nanomedicine, nanotaggants for security systems, nanoelectronics, nanodevices etc, according to excerpts from the book written by intellectual property experts Dr Prabuddha Ganguli and Dr Siddharth Jabade. The book is slated for launch in Hyderabad this week. Nanotechnology allows scientists to dabble at the small, building-block atomic or molecular level. And while the technology is not without its sceptics, a section of the scientific community believes that nanotechnology would help develop break-through newer applications in several fields. Illustrating this nano-inclusiveness, the authors say, the protection of intellectual property is important to the nanotechnology industry because of its complex knowledge matrix. Driving the nano to success will require cross-disciplinary expertise to contemplate, foresee and address as many social, legal including intellectual property rights, cultural, ethical, religious, philosophical and political implications of the nanoworld, tomorrow and the days after, the book … Continue reading

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NIH funds development of tissue chips to help predict drug safety

Posted: Published on July 25th, 2012

Embargoed for Release Tuesday, July 24, 2012 11 a.m. EDT Seventeen National Institutes of Health grants are aimed at creating 3-D chips with living cells and tissues that accurately model the structure and function of human organs such as the lung, liver and heart. Once developed, these tissue chips will be tested with compounds known to be safe or toxic in humans to help identify the most reliable drug safety signals ultimately advancing research to help predict the safety of potential drugs in a faster, more cost-effective way. The initiative marks the first interagency collaboration launched by the NIH's recently created National Center for Advancing Translational Sciences (NCATS). Tissue chips merge techniques from the computer industry with modern tissue engineering by combining miniature models of living organ tissues on a transparent microchip. Ranging in size from a quarter to a house key, the chips are lined with living cells and contain features designed to replicate the complex biological functions of specific organs. NIH's newly funded Tissue Chip for Drug Screening initiative is the result of collaborations that focus the resources and ingenuity of the NIH, Defense Advanced Research Projects Agency (DARPA) and U.S. Food and Drug Administration. NIH's Common Fund … Continue reading

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