Article

Allele-specific silencing ameliorates restrictive cardiomyopathy due to a human myosin regulatory light chain mutation

2019-02-24

Abstract excerpt

<h4>Background</h4> Restrictive cardiomyopathy (RCM) is a rare heart disease associated with mutations in sarcomeric genes and with phenotypic overlap with hypertrophic cardiomyopathy. There is no approved therapy. Here, we explore the potential of an interfering RNA (RNAi) therapeutic for a human sarcomeric mutation in MYL2 causative of restrictive cardiomyopathy in a mouse model. <h4>Methods</h4> AAV9-M7.8L sh...

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Literature Corpus work: 88aac944-25c8-50e6-873f-52d69571984e
DOI: 10.1101/559468

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