Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
采用双重给药途径和免疫调节技术，对一名卡纳万病患者进行腺相关病毒介导的基因治疗。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2023.06.001
Corti, Manuela; Byrne, Barry J; Gessler, Dominic J; Thompson, Grace; Norman, Samantha; Lammers, Jenna; Coleman, Kirsten E; Liberati, Cristina; Elder, Melissa E; Escolar, Maria L; Tuna, Ibrahim S; Mesaros, Clementina; Kleiner, Gary I; Barbouth, Deborah S; Gray-Edwards, Heather L; Clement, Nathalie; Cleaver, Brian D; Gao, Guangping
