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Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates.
Ellsworth JL, Gingras J, Smith LJ, Rubin H, Seabrook TA, Patel K, Zapata N, Olivieri K, O'Callaghan M, Chlipala E, Morales P, Seymour A. Ellsworth JL, et al. Among authors: seymour a. PLoS One. 2019 Nov 26;14(11):e0225582. doi: 10.1371/journal.pone.0225582. eCollection 2019. PLoS One. 2019. PMID: 31770409 Free PMC article.
Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH.
Ahmed SS, Rubin H, Wang M, Faulkner D, Sengooba A, Dollive SN, Avila N, Ellsworth JL, Lamppu D, Lobikin M, Lotterhand J, Adamson-Small L, Wright T, Seymour A, Francone OL. Ahmed SS, et al. Among authors: seymour a. Mol Ther Methods Clin Dev. 2020 Mar 13;17:568-580. doi: 10.1016/j.omtm.2020.03.009. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32258219 Free PMC article.
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS.
Smith LJ, Schulman LA, Smith S, Van Lieshout L, Barnes CM, Behmoiras L, Scarpitti M, Kivaa M, Duong KL, Benard LO, Ellsworth JL, Avila N, Faulkner D, Hayes A, Lotterhand J, Rivas JI, Sengooba AV, Tzianabos A, Seymour AB, Francone OL. Smith LJ, et al. Among authors: seymour ab. Mol Ther Methods Clin Dev. 2022 Jun 30;26:224-238. doi: 10.1016/j.omtm.2022.06.013. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35859693 Free PMC article.
375 results