ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY

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United States of America Patent

APP PUB NO 20240182533A1
SERIAL NO

18482526

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Abstract

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Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vectors(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.

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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
Mendell, Jerry R Columbus, US 27 124
Rodino-Klapac, Louise Grove City, US 33 56

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