| abstract |
The present disclosure provides methods for treating or preventing Duchenne Muscular Dystrophy (DMD) in a subject in need thereof, the methods comprising administering to the subject a Cas9 nuclease or a sequence encoding a Cas9 nuclease and a gRNA or a sequence encoding a gRNA, wherein the gRNA targets a splice donor or splice acceptor site of a dystrophin gene. Administration restores dystrophin expression in at least a portion of the cardiomyocytes in the subject, and can restore myocardial contractility at least partially or completely. |