PhysiologybioRxiv
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…·M. Cerrone
This paper demonstrates that AAV-mediated delivery of PKP2a gene therapy arrests ARVC progression and prolongs survival in PKP2-deficient mice.
DOI 10.1161/circgen.123.004305