AAV8-based gene replacement therapy for hereditary spastic paraplegia type 5.
基于 AAV8 的基因替代疗法治疗遗传性痉挛性截瘫 5 型
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101531
Wiora Linus, Yuan Qinggong, Hook Sebastian, Kraft Melanie, Björkhem Ingemar, Ott Michael, Hauser Stefan, Schöls Ludger
