Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy

Variants in myosin-binding protein C3 ( MYBPC3 ) gene are a main cause of hypertrophic cardiomyopathy (HCM), accounting for 30% to 40% of the total number of HCM mutations. Gene editing represents a potential permanent cure for HCM. The aim of this study was to investigate whether genome editing of...

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