P.316Adeno-associated virus mediated SUMO1 overexpression improves cardiac disease phenotype in mouse models of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a genetic, muscle degenerative disorder whose major cause of death is heart failure (HF). We have developed a novel therapy for HF based on the overexpression of small ubiquitin-like modifier type 1 (SUMO1) protein that improves cardiac function by activating sarcoplasmic reticulum Ca2+-ATPase (SERCA2a). This concept has been demonstrated by therapeutic efficacy in both small and large animal models of HF. Calcium overload in cardiac muscle cells has been a major determinant of the DMD cardiopathogenesis, which we hypothesized would be mitigated by targeting SUMO1.

Source: Neuromuscular Disorders - September 30, 2019 Category: Neurology Authors: P. Lee, J. Oh, A. Lee, D. Jeong, V. Chepurko, R. Hajjar, C. Kho Source Type: research