Preclinical efficacy and safety of AAVrh10-based plakophilin-2 gene therapy (LX2020) as a treatment for arrhythmogenic cardiomyopathy

Abstract Plakophilin-2 (PKP2) mutations cause fatal genetic heart disease and arrhythmogenic cardiomyopathy (ACM) with primary effects on the right ventricle (RV). Adeno-associated virus (AAV)-PKP2 gene therapy shows promise as a therapeutic strategy but lacks long-term data and guidelines on minima...

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Bibliographic Details

Published in:	npj Regenerative Medicine
Main Authors:	Jing Zhang, Erika Joana Gutierrez-Lara, Aryanne Do, Lena Nguyen, Anju Nair, Nithya Selvan, Tim Fenn, Eric Adler, Richie Khanna, Farah Sheikh
Format:	Article
Language:	English
Published:	Nature Portfolio 2025-04-01
Online Access:	https://doi.org/10.1038/s41536-025-00401-6