Open AccessCell-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
Author(s) -
Annie Tanenhaus,
T. D. Stowe,
Andrew L. Young,
John McLaughlin,
Rangoli Aeran,
Weitao Lin,
Jianmin Li,
Raghavendra Hosur,
Ming Chen,
Jennifer Leedy,
Tiffany Chou,
Sureshnee Pillay,
Maria Candida Vila,
Jennifer A. Kearney,
Martin Moorhead,
Archana Belle,
Stephanie Tagliatela
Publication year - 2022
Publication title -
human gene therapy
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.633
H-Index - 149
eISSN - 1557-7422
pISSN - 1043-0342
DOI - 10.1089/hum.2022.037
Subject(s) - dravet syndrome , gabaergic , biology , adeno associated virus , hippocampus , neuroscience , inhibitory postsynaptic potential , interneuron , epilepsy , pharmacology , vector (molecular biology) , genetics , gene , recombinant dna