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Study Looks to Genome Editing to Treat Deadly Degenerative Disorder

Harvard University stem-cell researchers led by Amy Wagers, the Forst Family Professor of Stem Cell and Regenerative Biology, are embarking on a major study of Duchenne muscular dystrophy (DMD). Supported by research funding from Sarepta Therapeutics, under a multiyear collaboration agreement coordinated by Harvard’s Office of Technology Development (OTD), the project aims to use in vivo genome editing in mouse models of DMD to fully and precisely restore the function of a protein crucial for proper muscular growth and development. Approaches validated by this work may point the way to an eventual therapeutic strategy to reverse DMD in humans

Duchenne muscular dystrophy is a genetic disease caused by the lack of a protein called dystrophin that normally helps support the structural integrity of muscle fibers, including those in the heart. Without dystrophin, cells are weaker and degenerate more quickly. Over time, affected individuals — boys, typically, as DMD is a recessive X-linked disorder — lose their capacity to move independently.


Study Looks To Genome Editing To Treat Deadly Degenerative Disorder