| CPC C12N 15/113 (2013.01) [C07K 14/4707 (2013.01); C12N 15/1138 (2013.01); C12N 15/86 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2320/32 (2013.01); C12N 2330/51 (2013.01); C12N 2750/14143 (2013.01)] | 10 Claims |
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1. 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: 7701, 7798, 7945, 7967, 7990, 8057, 8078, and 8148, wherein the recombinant adeno-associated virus lacks rep and cap genes.
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