Growing up in Devon in the 1980s, brothers James and Matt (names have been changed) had to be careful with their antics – a cut or bruise could land them in hospital.
Jan 15, 2019
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Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania ...
Oct 18, 2018
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This week, a 40-year-old patient was treated at UNC's Clinical and Translational Research Center (CTRC) with SB-913, an investigational genome editing therapy for individuals with mucopolysaccharidosis type II (MPS II), a ...
Mar 22, 2018
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Researchers at Lund University in Sweden have developed a method that enables them to use donated brain tissue from people with epilepsy for 48 hours. Previously, the researchers only had 12 hours to test new treatments before ...
Mar 9, 2018
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A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment ...
Dec 6, 2017
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Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, ...
Jul 25, 2017
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A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
Jul 12, 2017
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Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that ...
Jun 8, 2017
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Common knowledge says that genetic mutations are bad. This is true for most mutations of lipoprotein lipase (LPL), the enzyme in the blood responsible for the breakdown of lipoproteins, which allows tissue to utilize energy ...
Mar 1, 2017
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For the last several decades, scientists worldwide have been seeking to harness the power of stem cells to develop therapies for human diseases and conditions. At UCLA's Broad Stem Cell Research Center, the potential to bring ...
Jun 14, 2016
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