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

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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Bibliographic Details
Main Authors: Chang Xie, Xue-Min Gong, Jie Luo, Bo-Liang Li, Bao-Liang Song
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Journal of Lipid Research
Subjects:
cholesterol
gene therapy
Niemann-Pick type C disease
lysosomal storage diseases
adeno-associated virus serotype 9
Niemann-Pick type C1
Online Access:http://www.sciencedirect.com/science/article/pii/S0022227520304454

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http://www.sciencedirect.com/science/article/pii/S0022227520304454

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