Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy
利用共价封闭末端AAV载体进行脊髓性肌萎缩症基因治疗
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.06.028
Duan, Haolin; Zhang, Ciliu; Zhang, Zhongliang; Wang, Xiaole; Zhang, Junping; Yang, Lifen; He, Fang; Mao, Leilei; Yang, Li; Pan, Zou; Han, Renzhi; Wang, Weiming; Pan, Dao; Yin, Fei; Xiao, Weidong; Peng, Jing