Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study
单剂量GC101基因疗法治疗II型和III型脊髓性肌萎缩症:一项开放标签单臂研究
期刊:World Journal of Pediatrics
影响因子:4.5
doi:10.1007/s12519-025-00955-x
Ma, Xiu-Wei; Jiang, Xin-Yang; Li, Zhong-Qiu; Dong, Xiao-Yan; Ma, Wen-Hao; Wang, Yong-Xia; Yu, Shuang-Qing; Gu, Rui-Jie; Wang, Xiao-Dong; Zhong, Bai-Mao; He, Fang; Xu, Juan; Mao, Ying-Ying; Zhang, Yan-Ping; Zhang, Shan; Li, Ting; Li, Chong-Yang; Zhang, Sheng; Yang, Xiao; Zhu, Li-Na; Wu, Zhi-Jie; Zhou, Bing; Zhuang, Lu; Li, Qiu-Ping; Wu, Xiao-Bing; Feng, Zhi-Chun