eric olson | sciencenewsnet.in

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New Gene Editing Strategies Developed For Duchenne Muscular Dystrophy

April 30, 2021 sarah JonasResearch Results

DALLAS – April 30, 2021 – UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.