Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

por Davidson, Cristin D, Gibson, Alana L, Gu, Tansy, Baxter, Laura L, Deverman, Benjamin E, Beadle, Keith, Incao, Arturo A, Rodriguez-Gil, Jorge L, Fujiwara, Hideji, Jiang, Xuntian, Chandler, Randy J, Ory, Daniel S, Gradinaru, Viviana, Venditti, Charles P, Pavan, William J
Publicado 2021