AAV-mediated interneuron-specific gene replacement for Dravet syndrome
John K. Mich, Jiyun Ryu, Aguan Daniel Wei, Bryan B. Gore, Rong Guo, Angela M. Bard, Refugio A. Martinez, Yemeserach M. Bishaw, Emily M. Luber, Luiz Marcelo Oliveira, Nicole Miranda, Jan‐Marino Ramirez, Jonathan T. Ting, Ed S. Lein, Boaz P. Levi, Franck K. Kalume
This study demonstrates that interneuron-specific AAV-mediated SCN1A gene replacement significantly rescues Dravet syndrome symptoms in mouse models.
bioRxiv · Dec 15, 2023 · PreprintNeuroscience