OLIGONUCLEOTIDES AND METHODS FOR TREATMENT OF CARDIOMYOPATHY USING RNA INTERFERENCE

摘要

Compositions and methods for treating cardiomyopathy using RNA interference are disclosed. In particular, embodiments of the invention relate to the use of oligonucleotides for treatment of cardiomyopathy, including small interfering RNAs (siRNAs) and short hairpin RNAs (shRNAs) that silence expression of disease-causing mutant alleles, such as the myosin MYL2 allele encoding human regulatory light chain (hRLC)-N47K and the MYH7 allele encoding human myosin heavy chain (hMHC)-R403Q while retaining expression of the corresponding wild-type allele.