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

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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Bibliographic Details
Main Authors: Benny Liu, Charina M. Ramirez, Anna M. Miller, Joyce J. Repa, Stephen D. Turley, John M. Dietschy
Format: Article
Language:English
Published: Elsevier 2010-05-01
Series:Journal of Lipid Research
Subjects:
Niemann-Pick type C
neurodegeneration
liver
lung
lysosome
macrophage
Online Access:http://www.sciencedirect.com/science/article/pii/S002222752041048X

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http://www.sciencedirect.com/science/article/pii/S002222752041048X

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