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
细胞选择性腺相关病毒介导的 SCN1A 基因调控疗法挽救了 Dravet 综合征小鼠模型中的死亡率和癫痫发作表型，并且在非人类灵长类动物中耐受性良好
期刊:Human Gene Therapy
影响因子:3.9
doi:10.1089/hum.2022.037
Annie Tanenhaus, Timothy Stowe, Andrew Young, John McLaughlin, Rangoli Aeran, I Winnie Lin, Jianmin Li, Raghavendra Hosur, Ming Chen, Jennifer Leedy, Tiffany Chou, Sirika Pillay, Maria Candida Vila, Jennifer A Kearney, Martin Moorhead, Archana Belle, Stephanie Tagliatela
