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 | Zendy

Annie Tanenhaus | Zendy; T. D. Stowe | Zendy; Andrew L. Young | Zendy; John McLaughlin | Zendy; Rangoli Aeran | Zendy; IChun Lin | Zendy; Jianmin Li | Zendy; Raghavendra Hosur | Zendy; Ming Chen | Zendy; Jennifer Leedy | Zendy; Tiffany Chou | Zendy; Sirika Pillay | Zendy; Maria Candida Vila | Zendy; Jennifer A. Kearney | Zendy; Martin Moorhead | Zendy; Archana Belle | Zendy; Stephanie Tagliatela | Zendy

Open Access

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

Author(s) -

Annie Tanenhaus,

T. D. Stowe,

Andrew L. Young,

John McLaughlin,

Rangoli Aeran,

IChun 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

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

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