Article
Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Attributable to a Human Myosin Regulatory Light Chain Mutation.
Circulation - 27 Aug 2019
Zaleta-Rivera Kathia, Dainis Alexandra, Ribeiro Alexandre J S, Cordero Pablo, Rubio Gabriel, Shang Ching, Liu Jing, Finsterbach Thomas, Parikh Victoria N, Sutton Shirley, Seo Kinya, Sinha Nikita, Jain Nikhil, Huang Yong, Hajjar Roger J, Kay Mark A, Szczesna-Cordary Danuta, Pruitt Beth L, Wheeler Matthew T, Ashley Euan A
Abstract excerpt
BACKGROUND: Restrictive cardiomyopathy is a rare heart disease associated with mutations in sarcomeric genes and with phenotypic overlap with hypertrophic cardiomyopathy. There is no approved therapy directed at the underlying cause. 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. METHODS: A...
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