Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study

• 期刊论文

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• 发表日期：

  2025年8月4日

• Xiu-Wei Ma,
• Xin-Yang Jiang,
• Zhong-Qiu Li,
• Xiao-Yan Dong,
• Wen-Hao Ma,
• Yong-Xia Wang,
• Shuang-Qing Yu,
• Rui-Jie Gu,
• Xiao-Dong Wang,
• Bai-Mao Zhong,
• Fang He,
• Juan Xu,
• Ying-Ying Mao,
• Yan-Ping Zhang,
• Shan Zhang,
• Ting Li,
• Chong-Yang Li,
• Sheng Zhang,
• Xiao Yang,
• Li-Na Zhu,
• Zhi-Jie Wu,
• Bing Zhou,
• Lu Zhuang,
• Qiu-Ping Li,
• Xiao-Bing Wu,
• Zhi-Chun Feng