Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.
开发基于 AAV 的基因疗法治疗弗里德赖希共济失调的眼部表型。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.10.048
Tang Heyu, Gupte Siddhant, Xu Emily, Calabro Kaitlyn R, Friend Hannah, Crosson Sean M, Fajardo Diego, Kostamo Zachary, Zhang Hangning, Peterson James J, Lin Fangyu, Kozmik Zbynek, Lutz Cathleen M, Boye Sanford L, Boye Shannon E
