Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate

Summary: Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. Administration of recombinant adeno-associated viral vectors delivering microdystrophin or ribo...

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Bibliographic Details
Main Authors: Stephen C. Kolwicz, Jr., PhD, John K. Hall, PhD, Farid Moussavi-Harami, MD, Xiolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD, Guy L. Odom, PhD
Format: Article
Language:English
Published: Elsevier 2019-11-01
Series:JACC: Basic to Translational Science
Online Access:http://www.sciencedirect.com/science/article/pii/S2452302X19301913