Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy

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United States of America

PATENT NO 12258573
APP PUB NO 20220290180A1
SERIAL NO

17635978

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Abstract

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Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSGCA using a systemic route of administration and at a dose of about 1.0×1012 vg/kg to about 5.0×1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.

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Patent Owner(s)

Patent OwnerAddress
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL700 CHILDREN'S DRIVE COLUMBUS OH 43205

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Griffin, Danielle Canal Winchester, US 4 3
Mendell, Jerry R Columbus, US 27 124
Rodino-Klapac, Louise Grove City, US 33 56

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