Adeno-associated virus expressing a blood-brain barrier-penetrating enzyme improves GM1 gangliosidosis in a preclinical model.
表达血脑屏障穿透酶的腺相关病毒在临床前模型中改善了 GM1 神经节苷脂沉积症
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI180724
Matsushima Saki Kondo, Shimada Yohta, Kinoshita Masafumi, Nagashima Takashi, Okamoto Shinichiro, Iizuka Sayoko, Takagi Haruna, Iizuka Shunsuke, Higuchi Takashi, Hioki Hiroyuki, Watabe Ayako M, Sonoda Hiroyuki, Ohashi Toya, Kobayashi Hiroshi