Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophy
AAV介导的ARSA替代疗法治疗异染性脑白质营养不良的跨物种疗效
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI185001
Ramachandran, Shyam; Ardinger, Jeffery; Bu, Jie; Ramos, MiAngela; Guo, Lilu; Ghosh, Dhiman; Hossain, Mahmud; Chou, Shih-Ching; Chen, Yao; Wischhof, Erik; Ayloo, Swathi; Trullo, Roger; Luo, Yuxia; Hogestyn, Jessica M; DuBreuil, Daniel M; Crosier, Emily; Flyer-Adams, Johanna G; Richards, Amy M; Tsabar, Michael; Gaglia, Giorgio; Nass, Shelley; Nambiar, Bindu; Woodcock, Denise; O'Riordan, Catherine; Tang, Qi; Elmer, Bradford; Zhang, Bailin; Goulet, Martin; Mueller, Christian