Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice.
Kida S, Koshimura Y, Yoden E, Yoshioka A, Morimoto H, Imakiire A, Tanaka N, Tanaka S, Mori A, Ito J, Inoue A, Yamamoto R, Minami K, Hirato T, Takahashi K, Sonoda H.
Kida S, et al. Among authors: ito j.
Mol Ther Methods Clin Dev. 2023 May 12;29:439-449. doi: 10.1016/j.omtm.2023.05.010. eCollection 2023 Jun 8.
Mol Ther Methods Clin Dev. 2023.
PMID: 37251981
Free PMC article.