Low-Dose AAV9-SMN1 with CNS-Selective Expression Delivers Efficacy and Favorable Safety in Spinal Muscular Atrophy.

DOI:10.1016/j.ymthe.2026.02.050
作者：Yongguo Yu,Xiji Qin,Mengxia Jing,Carol Huang,Xin Zhou,Yanjie Fan,Jia Rao,Hongping Xia,Weiqian Dai,Xiaomei Luo,Hui Liu,Shihua Cao,Yanjun Shen,Yao Qiu,Qianru Zhang,Shuqi Li,Ling Su,Yi Wang,Jay Hou,Kun S
文献类型：期刊论文
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