AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease

Niemann-Pick type C disease (NP-C) is a fatal neurodegenerative lysosomal storage disorder. It is caused in 95% of cases by a mutation in the NPC1 gene that encodes NPC1, an integral transmembrane protein localised to the limiting membrane of the lysosome. There is no cure for NP-C but there is a di...

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Bibliographische Detailangaben
Hauptverfasser: 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
Format: Journal article
Veröffentlicht: Oxford University Press 2018

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