Electrophysiological abnormalities associated with a CACNA1D variant are rescued by AAV6-Cav1.3-C-terminus gene therapy in patient-iPSC-CMs
在患者诱导多能干细胞来源的心肌细胞中,AAV6-Cav1.3-C端基因疗法可以挽救与CACNA1D变异相关的电生理异常。
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doi:10.64898/2025.12.15.694396
Reisqs, Jean-Baptiste; Sleiman, Yvonne; Gando, Ivan; Cupelli, Michael; Ginjupalli, Vamsi Krishna Murthy; Delanoe-Ayari, Helene; Coetzee, William; Qu, Yongxia S; Delmar, Mario; Cecchin, Frank; Tan, Reina B; Chahine, Mohamed; Boutjdir, Mohamed