Recombinant virus products and methods for inhibition of expression of myotilin

摘要

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.

主权项

1. A recombinant adeno-associated virus comprising the MYOT miRNA-encoding DNA set out in SEQ ID NO: 1, 2, 3 or 4, wherein the recombinant adeno-associated virus lacks rep and cap genes.