Article
Allele-specific silencing of mutant Myh6 transcripts in mice suppresses hypertrophic cardiomyopathy.
Science (New York, N.Y.) - 4 Oct 2013
Jiang Jianming, Wakimoto Hiroko, Seidman J G, Seidman Christine E
Abstract excerpt
Dominant mutations in sarcomere proteins such as the myosin heavy chains (MHC) are the leading genetic causes of human hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy. We found that expression of the HCM-causing cardiac MHC gene (Myh6) R403Q mutation in mice can be selectively silenced by an RNA interference (RNAi) cassette delivered by an adeno-associated virus vector. RNAi-transduced MHC(403/+)...
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