Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies

por Chen, Xin, Dong, Thomas, Hu, Yuhui, De Pace, Raffaella, Mattera, Rafael, Eberhardt, Kathrin, Ziegler, Marvin, Pirovolakis, Terry, Sahin, Mustafa, Bonifacino, Juan S., Ebrahimi-Fakhari, Darius, Gray, Steven J.
Publicado 2023