Humanizing the mdx mouse model of DMD: the long and the short of it.

Yucel, Nora; Chang, Alex C; Day, John W; Rosenthal, Nadia; Blau, Helen M · NPJ Regen Med · 2018

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Abstract

Duchenne muscular dystrophy (DMD) is a common fatal heritable myopathy, with cardiorespiratory failure occurring by the third decade of life. There is no specific treatment for DMD cardiomyopathy, in large part due to a lack of understanding of the mechanisms underlying the cardiac failure. <i>Mdx</i> mice, which have the same dystrophin mutation as human patients, are of limited use, as they do not develop early dilated cardiomyopathy as seen in patients. Here we summarize the usefulness of the various commonly used DMD mouse models, highlight a model with shortened telomeres like humans, and identify directions that warrant further investigation.