Sarepta Therapeutics, Inc.
SYSTEMIC DELIVERY OF ADENO-ASSOCIATED VIRUS VECTOR EXPRESSING GAMMA-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY

Abstract:

Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCG vector, methods of expressing gamma-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.