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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
Published 2018Journal article