Duchenne Muscular Dystrophy Mice and Men: Can Understanding a Genetic Cardiomyopathy Inform Treatment of Other Myocardial Diseases?

Abstract

Duchenne muscular dystrophy (DMD) mouse models have a predictable and reproducible time-course of cardiomyopathy progression with discrete pathogenic steps, which closely parallel what we know occurs in the cardiomyopathy of patients with DMD. The slow progression of early pathogenic steps common to many cardiomyopathies may make DMD models useful for identifying novel treatment targets and testing the therapeutic value of new treatment paradigms for an at-risk patient population far beyond those with muscular dystrophies.