Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates.
AAV9-coSMN1 基因疗法治疗脊髓性肌萎缩症的临床前评估：小鼠模型和非人灵长类动物的疗效和安全性
期刊:Molecular Medicine
影响因子:6.4
doi:10.1186/s10020-025-01207-4
Ma Wenhao, Wu Zhijie, Zhao Tianyi, Xia Yan, Qin Jing, Tian Xue, Li Xin, He Jun, Zhang Yan, Zhang Lina, Li Li, Dong Zheyue, Feng Zhichun, Dong Xiaoyan, Sheng Wang, Wu Xiaobing
