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

Chantal J M van Opbergen Bitha NarayananChester B SacramentoKatie M StilesVartika MishraEsther Frenk David M RicksGrace ChenMingliang ZhangPaul YarabeJonathan D Schwartz Mario DelmarChris D HerzogMarina Cerrone

Published in: Circulation. Genomic and precision medicine (2024)

These preclinical data demonstrate the potential for AAVrh.74-PKP2a (RP-A601) as a therapeutic for PKP2-related arrhythmogenic right ventricular cardiomyopathy in both early and more advanced stages of the disease.

Keyphrases

gene therapy
heart failure
cell therapy
electronic health record
big data
cardiopulmonary resuscitation
stem cells
human health
mesenchymal stem cells
machine learning
risk assessment
bone marrow
atrial fibrillation