New gene editing strategies developed for Duchenne muscular dystrophy

DALLAS - April 30, 2021 - UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.

"Thousands of different mutations causing Duchenne have been identified, but they tend to cluster into certain parts of the dystrophin gene," says study leader END