Article
Myosin inhibitor reverses hypertrophic cardiomyopathy in genotypically diverse pediatric iPSC-cardiomyocytes to mirror variant correction.
Cell reports. Medicine - 21 May 2024
Kinnear Caroline, Said Abdelrahman, Meng Guoliang, Zhao Yimu, Wang Erika Y, Rafatian Naimeh, Parmar Neha, Wei Wei, Billia Filio, Simmons Craig A, Radisic Milica, Ellis James, Mital Seema
Abstract excerpt
Pathogenic variants in MYH7 and MYBPC3 account for the majority of hypertrophic cardiomyopathy (HCM). Targeted drugs like myosin ATPase inhibitors have not been evaluated in children. We generate patient and variant-corrected iPSC-cardiomyocytes (CMs) from pediatric HCM patients harboring single variants in MYH7 (V606M; R453C), MYBPC3 (G148R) or digenic variants (MYBPC3 P955fs, TNNI3 A157V). We also generate CMs...
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