First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial
首次人体高剂量AAV9鞘内基因疗法治疗儿童CLN7疾病:一项1期、开放标签、单次递增剂量、非随机临床试验
期刊:EBioMedicine
影响因子:10.8
doi:10.1016/j.ebiom.2025.106044
Greenberg, Benjamin M; Minassian, Berge; Messahel, Souad; Edgar, Veronica Bordes; Lowden, Andrea; Dahshi, Hamza; Nettesheim, Emily R; Nguyen, Hoang H; Hughes, Samuel; Muthukumar, Alagar R; Srinivasan, Kalayarasan; Iannaccone, Susan; Varadarajan, Ganapathy; Gray, Steven J; Kayani, Saima N