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.
作者信息
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
机构信息
Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of 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
Chin Med J (Engl). 2024 Aug 20;137(16):1976-1978. doi: 10.1097/CM9.0000000000003210. Epub 2024 Jul 9.
Abstract
摘要
Zotero 插件