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Article

Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics.

Nature cardiovascular research - 1 Dec 2023

Bradford William H, Zhang Jing, Gutierrez-Lara Erika J, Liang Yan, Do Aryanne, Wang Tsui-Min, Nguyen Lena, Mataraarachchi Nirosh, Wang Jie, Gu Yusu, McCulloch Andrew, Peterson Kirk L, Sheikh Farah

Abstract excerpt

Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a fatal genetic heart disease characterized by cardiac arrhythmias, in which fibrofatty deposition leads to heart failure, with no effective treatments. Plakophilin 2 (PKP2) is the most frequently mutated gene in ARVC, and although altered RNA splicing has been implicated, there are no models to study its effect and therapeutics. Here, we generate a mouse...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39196150
DOI: 10.1038/s44161-023-00370-3
PMCID: PMC11357983

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