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Gene therapy for muscular dystrophy shows promise beyond safety.

Researchers have cleared a safety hurdle in efforts to develop a gene therapy for treatment of a form of muscular dystrophy that disables patients by gradually weakening muscles near the hips and shoulders. Described as the first gene therapy trial in muscular dystrophy demonstrating promising findings, researchers from the University of Florida (UF), Nationwide Children's Hospital in Columbus, Ohio, and The Ohio State University report how they safely transferred a gene to produce a protein necessary for healthy muscle fiber growth into three teenagers with limb-girdle muscular dystrophy. The findings, which have relevance to genetic disorders beyond muscular dystrophy as well as conditions in which muscles atrophy, were published online recently in the Annals of Neurology. Research subjects received a dose of the gene on one side of the body and saline on the opposite side. Neither the researchers nor the patients knew which of the foot muscles received the actual treatment until the end of the experiment. The volunteers were evaluated at set intervals through 180 days, and therapy effectiveness was measured by assessing alpha-SG protein expression in the muscle, which was four to five times higher than in the muscles that received only the saline. The volunteers encountered no adverse health events, and the transferred genes continued to produce the needed protein for at least six months after treatment. The research was supported by the Muscular Dystrophy Association and the National Institutes of Health. For more information, go to 1299&page=204&type=new&id=626&clk=199750.

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Title Annotation:WHAT'S HAPPENING
Publication:The Exceptional Parent
Date:Jun 1, 2009
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