Gene editing reverses arrhythmia susceptibility in humanized PLN-R14del mice: modelling a European cardiomyopathy with global impact.

Cardiovascular research(2022)

引用 16|浏览28
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摘要
This study is the first to show that disruption of hPLN-R14del allele by AAV9-CRISPR/Cas9 improves cardiac function and reduces VT susceptibility in humanized PLN-R14del mice, offering preclinical evidence for translatable approaches to therapeutically suppress the arrhythmogenic phenotype in human patients with PLN-R14del disease.
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关键词
CRISPR/Cas9,Gene therapy,Humanized mouse,Phospholamban R14del mutation,Ventricular tachycardia
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