RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN

Abstract: 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.
Patent Number: US2016310548(A1)
Public Date:
Inventor: HARPER SCOTT QUENTON [US]; LIU JIAN [US]
Applicant: NATIONWIDE CHILDREN'S HOSPITAL INC [US]
Application Number: US201514853517 20150914Global Dossier
Application Date:
Priority Number: US201514853517 20150914;US201314058909 20131021;WO2012US34408 20120420;US201161478012P 20110421
International Classification: A61K35/76; C12N15/113; C12N7/00
Cooperative Classification: C07K14/4707; C12N15/113; C12N15/1138; C12N15/86; C12N2310/14; C12N2310/141; C12N2320/32; C12N2330/51
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