Electrophysiological abnormalities associated with a CACNA1D variant are rescued by AAV6-Cav1.3-C-terminus gene therapy in patient-iPSC-CMs
Jean‐Baptiste Reisqs, Yvonne Sleiman, Ivan Gando, Michael Cupelli, Vamsi Krishna Murthy Ginjupalli, Hélène Delanoë‐Ayari, William A. Coetzee, Yongxia Sarah Qu, Mario Delmar, Frank Cecchin, Reina Bianca Tan, Mohamed Chahine, Mohamed Boutjdir
This study shows that a CACNA1D variant causes electrophysiological abnormalities in hiPSC-CMs, which are reversed by AAV6-Cav1.3-C-terminus gene therapy.
bioRxiv · Dec 17, 2025 · PreprintPhysiology