Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid

A mutation in NPC1 leads to sequestration of unesterified cholesterol in the late endosomal/lysosomal compartment of every cell culminating in the development of pulmonary, hepatic, and neurodegenerative disease. Acute administration of 2-hydroxypropyl-β-cyclodextrin (CYCLO) rapidly overcomes this t...

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Detalhes bibliográficos

Principais autores:	Benny Liu, Charina M. Ramirez, Anna M. Miller, Joyce J. Repa, Stephen D. Turley, John M. Dietschy
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2010-05-01
coleção:	Journal of Lipid Research
Assuntos:	Niemann-Pick type C neurodegeneration liver lung lysosome macrophage
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S002222752041048X
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