Specific RNA inhibition of causal alleles: A potential therapy for familial hypertrophic cardiomyopathy

Abstract

For a recent article in Science, Jiang et al1 used RNA inhibition to suppress specifically the expression of a mutant allele known to cause human familial hypertrophic cardiomyopathy. The RNA inhibition treatment prevented the development of familial hypertrophic cardiomyopathy in the mouse. This is an exciting, innovative finding which could pave the way for specific treatment of this disease in humans. If shown to be safe and effective, RNA inhibition could be applicable for many inherited autosomal dominant diseases. © 2014 American Heart Association, Inc.