dystrophin reaction in gene therapy for Duchenne muscular dystrophy DMD

An unexpected immune response to dystrophin has researchers proceeding cautiously; the finding changes how future gene therapy trials will be conducted

posted on April 21, 2010 - 10:59am
Unwanted responses by the immune system to dystrophin have been seen in a small, MDA-supported clinical trial of gene therapy for Duchenne muscular dystrophy (DMD) — an unexpected finding, investigators say. Rather than a setback, the finding is “the beginning of a new way of thinking” about gene therapy, said Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children's and a...

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