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US 2022/0064641 A1Published application
Recombinant virus products and methods for inhibition of expression of myotilin
AssigneeNationwide Children's Hospital, Inc.
InventorsScott Quenton Harper, Jian Liu
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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.
Claims13
1-10. (canceled)
A recombinant adeno-associated virus encoding a myotilin (MYOT) microRNA (miRNA) comprising an antisense guide strand as set out in any one of SEQ ID NOs:
- 7-5483, 5483-7353, 7355-7367, 7369-7422, 7424-7666, 7668-7797, 7799-7943, 7946-8012, 8014-8055, or 8057-11266, wherein the recombinant adeno-associated virus lacks rep and cap genes.
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Dependencies are inferred from the claim text.