Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy.
Kashiwakura Y, Endo K, Ugajin A, Kikuchi T, Hishikawa S, Nakamura H, Katakai Y, Baatartsogt N, Hiramoto T, Hayakawa M, Kamoshita N, Yamazaki S, Kume A, Mori H, Sata N, Sakata Y, Muramatsu SI, Ohmori T.
Kashiwakura Y, et al. Among authors: nakamura h.
Mol Ther Methods Clin Dev. 2023 Aug 22;30:502-514. doi: 10.1016/j.omtm.2023.08.016. eCollection 2023 Sep 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 37693948
Free PMC article.