Evaluation of efficacy and safety of AAV8-ÎC4ATP7B gene therapy in a mutant mouse model of Wilson's disease.
在威尔逊病突变小鼠模型中评估 AAV8-ΔC4ATP7B 基因疗法的疗效和安全性
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101435
Zeng Chunhua, Lin Yunting, Lu Xinshuo, Chen Shehong, Xia Yan, Zhang Kangdi, Shao Yongxian, Guan Zhihong, Du Rong, Liu Zongcai, Zhao Mingqi, Jiang Xiaoling, Cai Yanna, Li Taolin, Su Xueying, Chen Yaoyong, Dong Xiaoyan, Zhang Wen, Liu Li, Zhou Wenhao
