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.
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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.