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    Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates

    Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates by Wenhao Ma, Zhijie Wu, Tianyi Zhao, Yan Xia, Jing Qin, Xue Tian, Xin Li, Jun He, Yan Zhang, Lina Zhang, Li Li, Zheyue Dong, Zhichun Feng, Xiaoyan Dong, Wang Sheng, Xiaobing Wu

    Published 2025-04-01
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