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

Dravet syndrome (DS) is a developmental and epileptic encephalopathy caused by monoallelic loss-of-function variants in the SCN1A gene. SCN1A encodes for the alpha subunit of the voltage-gated type I sodium channel (Na(V)1.1), the primary voltage-gated sodium channel responsible for generation of ac...

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Detalles Bibliográficos
Autores principales: Tanenhaus, Annie, Stowe, Timothy, Young, Andrew, McLaughlin, John, Aeran, Rangoli, Lin, I. Winnie, Li, Jianmin, Hosur, Raghavendra, Chen, Ming, Leedy, Jennifer, Chou, Tiffany, Pillay, Sirika, Vila, Maria Candida, Kearney, Jennifer A., Moorhead, Martin, Belle, Archana, Tagliatela, Stephanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9242722/
https://www.ncbi.nlm.nih.gov/pubmed/35435735
http://dx.doi.org/10.1089/hum.2022.037

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