Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study
采用单剂量AAV9介导的基因疗法,通过鞘内注射GC101治疗1型脊髓性肌萎缩症(SMA)患儿:一项开放标签、单臂研究
期刊:Chinese Medical Journal
影响因子:7.3
doi:10.1097/CM9.0000000000003210
Ma, Xiuwei; Zhuang, Lu; Ma, Wenhao; Li, Jun; Wang, Xiaodong; Li, Zhongqiu; Jiang, Xinyang; Wang, Yongxia; Du, Ying; Zhang, Yingqian; He, Fang; Zhu, Zhiming; Du, Shaopeng; Xu, Juan; Gu, Ruijie; Zhang, Yanping; Zhang, Shan; Li, Ting; Yang, Xiao; Zhang, Sheng; Zhu, Lina; Li, Qiuping; Dong, Xiaoyan; Wu, Xiaobing; Feng, Zhichun