Rewriting the sarcomere: Gene therapy approaches for hypertrophic cardiomyopathy from bench to bedside.
Hypertrophic cardiomyopathy (HCM) is a prototypical inherited cardiomyopathy with well-defined sarcomeric genetic underpinnings that make it an attractive target for molecular therapy. We review recent advances in gene-based approaches for HCM, including adeno-associated virus-mediate gene replacement, allele-specific...