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Article

Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin

30 Apr 2021

Abstract excerpt

ΔR4-R23/ΔCT micro-dystrophin (μDys) is a miniaturized version of dystrophin currently evaluated in a Duchenne muscular dystrophy (DMD) gene therapy trial to treat skeletal and cardiac muscle disease. In pre-clinical studies, μDys efficiently rescues cardiac histopathology, but only partially normalizes cardiac function. To gain insights into factors that may impact the cardiac therapeutic efficacy of μDys, we...

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PMID: 33949649
DOI: 10.1093/hmg/ddab133

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