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。
Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study.
作者信息
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
机构信息
Department of Pediatrics, Faculty of Pediatrics, The Seventh Medical Center of the Chinese PLA General Hospital, Beijing 100700, China.
National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China.
出版信息
World J Pediatr. 2025 Sep;21(9):935-940. doi: 10.1007/s12519-025-00955-x. Epub 2025 Aug 4.
Abstract
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