PhysiologyCirculation Genomic and Precision MedicinePeer-reviewed
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 Narayanan, Chester Bittencourt Sacramento…·Marina Cerrone
This paper demonstrates that AAV-mediated delivery of PKP2a gene therapy arrests ARVC progression and prolongs survival in PKP2-deficient mice.
66 citationstop 1%