AAV-mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans
C. J. van Opbergen, B. Narayanan, C. B. Sacramento, K. M. Stiles, V. Mishra, E. Frenk, D. Ricks, G. Chen, M. Zhang, P. Yarabe, J. Schwartz, M. Delmar, C. D. Herzog, M. Cerrone
This paper demonstrates that AAV-mediated delivery of PKP2a gene therapy arrests ARVC progression and prolongs survival in PKP2-deficient mice.
bioRxiv · Jul 12, 2023Physiology