Systemic delivery of adeno-associated virus vector expressing gamma-sarcoglycan and the treatment of muscular dystrophy

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United States of America

PATENT NO 11925675
SERIAL NO

17468086

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Abstract

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Described herein are methods of treating muscular dystrophy comprising administering to a subject a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCG vector, methods of expressing gamma-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV scAAVrh74.MHCK7.hSGCG for usage in such methods, the doses and the ranges thereof for such methods, and methods of generating and producing the rAAV scAAVrh74.MHCK7.hSGCG.

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Patent Owner(s)

  • SAREPTA THERAPEUTICS, INC.

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Pozsgai, Eric Cambridge, US 2 1
Rodino-Klapac, Louise Cambridge, US 29 32

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