Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Due to a Human Myosin Regulatory Light Chain Mutation.

CONCLUSIONS: Our results show the feasibility, efficacy, and safety of RNAi therapeutics directed at human restrictive cardiomyopathy. This is a promising step towards targeted therapy for a prevalent human disease.
PMID: 31315475 [PubMed - as supplied by publisher]

Source: Circulation - July 17, 2019 Category: Cardiology Authors: Zaleta-Rivera K, Dainis A, Ribeiro AJS, Sanchez Cordero P, Rubio G, Shang C, Liu J, Finsterbach T, Parikh VN, Sutton S, Seo K, Sinha N, Jain N, Huang Y, Hajjar RJ, Kay MA, Szczesna-Cordary D, Pruitt BL, Wheeler MT, Ashley EA Tags: Circulation Source Type: research