Penn Medicine developed gene therapy to treat Duchenne muscular dystrophy (DMD

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On Oct. 7, 2019, Penn Medicine announced it had developed gene therapy to treat Duchenne muscular dystrophy (DMD) successfully, and safely stopped its associated severe muscle deterioration in animal models.

The findings, published online 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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Source: Penn Medicine
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