Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice
用转铁蛋白受体靶向 α-L-艾杜糖醛酸酶进行酶替代可挽救粘多糖贮积症 I 小鼠的脑病理
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2023.05.010
Sachiho Kida, Yuri Koshimura, Eiji Yoden, Aya Yoshioka, Hideto Morimoto, Atsushi Imakiire, Noboru Tanaka, Satowa Tanaka, Ayaka Mori, Jun Ito, Asuka Inoue, Ryuji Yamamoto, Kohtaro Minami, Tohru Hirato, Kenichi Takahashi, Hiroyuki Sonoda