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Article

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

Gene therapy - 1 Jun 2023

Nie Jiali, Han Yu, Jin Zhiyuan, Hang Weijian, Shu Hongyang, Wen Zheng, Ni Li, Wang Dao Wen

Abstract excerpt

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 MYBPC3 using the CRISPR/Cas9 system in vivo could rescue the phenotype of rats with HCM. We generated a rat model of...

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PMID: 36765144
DOI: 10.1038/s41434-023-00384-3

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Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy.PMID 36765144Read ↗

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