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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.
Tanenhaus A, Stowe T, Young A, McLaughlin J, Aeran R, Lin IW, Li J, Hosur R, Chen M, Leedy J, Chou T, Pillay S, Vila MC, Kearney JA, Moorhead M, Belle A, Tagliatela S. Tanenhaus A, et al. Among authors: aeran r. 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.
Structure comparison of the chimeric AAV2.7m8 vector with parental AAV2.
Bennett A, Keravala A, Makal V, Kurian J, Belbellaa B, Aeran R, Tseng YS, Sousa D, Spear J, Gasmi M, Agbandje-McKenna M. Bennett A, et al. Among authors: aeran r. J Struct Biol. 2020 Feb 1;209(2):107433. doi: 10.1016/j.jsb.2019.107433. Epub 2019 Dec 16. J Struct Biol. 2020. PMID: 31859208 Free PMC article.