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CRISPR editing could offer new DMD hope
April 2015
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CHICAGO—A promising advance in gene modification for patients with Duchenne muscular dystrophy was recently reported by Charles Gersbach, assistant professor of biomedical engineering at Duke University, in a Nature Communications paper. Gersbach is a Muscular Dystrophy Association (MDA) research grantee. The approach being investigated by Gersbach and his team has the potential to treat more than half of Duchenne patients as a permanent, one-time treatment. Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene that results in insufficient production of the dystrophin protein; this new approach uses CRISPR-Cas9 genome editing to target a large area of the dystrophin gene to catalyze production of dystrophin protein that is shorter than normal but still functional.
 
Dr. Grace Paylath, senior vice president and scientific program director at MDA, said that “MDA is proud to have supported Dr. Gersbach for the development of this game-changing advance.”
 
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