1区 · 医学
Article
作者: Bringas, John R. ; Ávila, Jesús ; Casas, Josefina ; Soto-Huelin, Beatriz ; Bankiewicz, Krystof S. ; Jurado-Arjona, Jerónimo ; Cheng, Seng H. ; Hadaczek, Piotr ; Sudhakar, Vivek ; Samaranch, Lluis ; Chen, Haifeng ; Pérez-Cañamás, Azucena ; Schuchman, Edward H. ; Ledesma, María Dolores ; He, Xingxuan ; Forsayeth, John
Gene therapy for Niemann-Pick type A disease is safe in nonhuman primates and has therapeutic effects in a mouse model.