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: Liu, Benny, Ramirez, Charina M., Miller, Anna M., Repa, Joyce J., Turley, Stephen D., Dietschy, John M.
Format: Artigo
Language:Inglês
Published: The American Society for Biochemistry and Molecular Biology 2010
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2853461/
https://ncbi.nlm.nih.gov/pubmed/19965601
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1194/jlr.M000257
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