Article

Gene Therapy for Cardiomyopathy associated with Duchenne Muscular Dystrophy in a Pig Model

2023-10-03

Abstract excerpt

<h4>Background</h4> Genetic cardiomyopathies caused by mutations in the dystrophin gene (DMD) are only partially responsive to current pharmacological heart failure treatments, although dilated and arrhythomogenic phenotypes of cardiomyopathy are frequent. <h4>Objective</h4> In this study, we tested whether a normalization of Ca 2+ -handling by forced expression of SERCA2a in cardiomyocytes mitigates heart fa...

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Literature Corpus work: f6985e51-1721-545d-bc90-eb1a4620024e
DOI: 10.1101/2023.10.02.560452

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