AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease

Niemann-Pick type C (NPC) disease is a fatal inherited neurodegenerative disorder caused by loss-of-function mutations in the NPC1 or NPC2 gene. There is no effective way to treat NPC disease. In this study, we used adeno-associated virus (AAV) serotype 9 (AAV9) to deliver a functional NPC1 gene sys...

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Detalhes bibliográficos
Publicado no:J Lipid Res
Main Authors: Xie, Chang, Gong, Xue-Min, Luo, Jie, Li, Bo-Liang, Song, Bao-Liang
Formato: Artigo
Idioma:Inglês
Publicado em: The American Society for Biochemistry and Molecular Biology 2017
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5335581/
https://ncbi.nlm.nih.gov/pubmed/28053186
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1194/jlr.M071274
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