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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A girl saw her mother's face for the first time. A boy tore through the aisles of Target, marveling at toys he never knew existed. A teen walked onto a stage and watched the stunned expressions of celebrity judges as he wowed ...
Oct 9, 2017
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The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie "Lorenzo's Oil."
Oct 4, 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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On July 12, a Food and Drug Administration panel unanimously recommended approval for the first-ever gene therapy treatment for cancer. The treatment, known as CTL019, is a T-cell therapy developed by the pharmaceutical company ...
Jul 19, 2017
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Researchers at University of Massachusetts Medical School and Auburn University College of Veterinary Medicine are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.
Jul 18, 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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