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

Author:

Ma Xiuwei1,Zhuang Lu12,Ma Wenhao3,Li Jun2,Wang Xiaodong4,Li Zhongqiu2,Jiang Xinyang2,Wang Yongxia1,Du Ying4,Zhang Yingqian4,He Fang1,Zhu Zhiming4,Du Shaopeng1,Xu Juan5,Gu Ruijie1,Zhang Yanping2,Zhang Shan6,Li Ting1,Yang Xiao2,Zhang Sheng2,Zhu Lina2,Li Qiuping6,Dong Xiaoyan4,Wu Xiaobing3,Feng Zhichun16

Affiliation:

1. Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China

2. National Engineering Laboratory for Birth Defects Prevention and Control of Key Technology, Beijing 100700, China

3. Research and Development Division, Genecradle Therapeutics Inc., Beijing 100176, China

4. Medical Division, Genecradle Therapeutics Inc., Beijing 100176, China

5. Department of Pharmacy, Medical Supplies Center of Chinese PLA General Hospital, Beijing 100700, China

6. Beijing Key Laboratory of Pediatric Organ Failure, Beijing 100700, China

Publisher

Ovid Technologies (Wolters Kluwer Health)

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