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Article

Myosin inhibitor reverses hypertrophic cardiomyopathy in pediatric iPSC-cardiomyocytes to mirror variant correction

2023-04-14

Abstract excerpt

<h4>SUMMARY</h4> Hypertrophic cardiomyopathy (HCM) is mainly caused by sarcomere gene variants in MYH7 and MYBPC3. Targeted drugs like myosin ATPase inhibitors have shown efficacy in adult HCM but have not been evaluated in children. We generated iPSC-cardiomyocytes (CMs) from four children with HCM harboring variants in MYH7 ( V606M; R453C) or MYBPC3 (G148R; P955fs and TNNI3_A157P ), variant-corrected control...

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Literature Corpus work
df27773f-a2a8-5f8d-a537-8f0a05ac6932
DOI
10.1101/2023.04.14.536782
Open publication

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Myosin inhibitor reverses hypertrophic cardiomyopathy in pediatric iPSC-cardiomyocytes to mirror variant correctionDOI 10.1101/2023.04.14.536782
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