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AAV-mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans

2023-07-12

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Pathogenic variants in plakophilin-2 (PKP2) cause arrhythmogenic right ventricular cardiomyopathy (ARVC), a disease characterized by life-threatening arrhythmias and progressive cardiomyopathy leading to heart failure. No effective medical therapy is available to prevent and/or arrest the disease. We tested the hypothesis that AAV-mediated delivery of the human PKP2 gene to...

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Literature Corpus work
2f5042fb-40a9-5b9f-8271-2443ec85c1f8
DOI
10.1101/2023.07.12.548590
Open publication

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AAV-mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in HumansDOI 10.1101/2023.07.12.548590
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