Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase.
通过 AAV 介导的肝脏递送脑穿透性艾杜糖醛酸-2-硫酸酯酶,挽救 II 型粘多糖贮积症小鼠的神经系统疾病。
期刊:Neurotherapeutics
影响因子:6.9
doi:10.1016/j.neurot.2025.e00741
Jin Xiu, Ye Qin, Wu Xiaoyi, Su Jing, Song Li, Fu Jiamei, Xu Qiuxia, Luo Min, Liu Fanfei, Ren Chengda, Hu Ming, Liu Man, An Yifang, Li Qiqi, Li Manjun, Yang Yang