The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery. by Eckenfelder, A, Tordo, J, Babbs, A, Davies, K, Goyenvalle, A, Danos, O

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease by Hughes, MP, Smith, DA, Morris, L, Fletcher, C, Colaco, A, Huebecker, M, Tordo, J, Palomar, N, Massaro, G, Henckaerts, E, Waddington, SN, Platt, FM, Rahim, AA