• Chinese medical journal · Aug 2024

    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.

    • Xiuwei Ma, Lu Zhuang, Wenhao Ma, Jun Li, Xiaodong Wang, Zhongqiu Li, Xinyang Jiang, Yongxia Wang, Ying Du, Yingqian Zhang, Fang He, Zhiming Zhu, Shaopeng Du, Juan Xu, Ruijie Gu, Yanping Zhang, Shan Zhang, Ting Li, Xiao Yang, Sheng Zhang, Lina Zhu, Qiuping Li, Xiaoyan Dong, Xiaobing Wu, and Zhichun Feng.
    • Department of Neurodevelopment, Faculty of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing 100700, China.
    • Chin. Med. J. 2024 Aug 20; 137 (16): 197619781976-1978.

    no abstract available

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