Recombinant virus products and methods for inhibition of expression of myotilin

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United States of America Patent

PATENT NO 9133482
APP PUB NO 20140045925A1
SERIAL NO

14058909

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Abstract

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The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

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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
Harper, Scott Quenton Powell, US 23 115
Liu, Jian Columbus, US 667 5710

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