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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Autores principales:	Chang Xie, Xue-Min Gong, Jie Luo, Bo-Liang Li, Bao-Liang Song
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2017-03-01
Colección:	Journal of Lipid Research
Materias:	cholesterol gene therapy Niemann-Pick type C disease lysosomal storage diseases adeno-associated virus serotype 9 Niemann-Pick type C1
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S0022227520304454
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