Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates.
Hum Gene Ther. 2022 Jun;33(11-12):579-597. doi: 10.1089/hum.2022.037. Hum Gene Ther. 2022. PMID: 35435735 Free PMC article.