Article
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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Identifiers and source
- Literature Corpus work
- 2f5042fb-40a9-5b9f-8271-2443ec85c1f8
- DOI
- 10.1101/2023.07.12.548590
