A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal models, according to a first-of-its-kind study from Penn Medicine researchers. The findings, published online today in Nature Medicine, puts the field within closer reach of a safe and effective gene therapy that uses a "substitute" protein without triggering immune responses known to hinder other therapeutic approaches.
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