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Article

Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life.

Pediatric research - 1 Oct 2010

Ramirez Charina M, Liu Benny, Taylor Anna M, Repa Joyce J, Burns Dennis K, Weinberg Arthur G, Turley Stephen D, Dietschy John M

Abstract excerpt

Niemann-Pick type C1 (NPC1) disease arises from a mutation inactivating NPC1 protein that normally moves unesterified cholesterol from the late endosomal/lysosomal complex of cells to the cytosolic compartment for processing. As a result, cholesterol accumulates in every tissue of the body causing liver, lung, and CNS disease. Treatment of the murine model of this disease, the npc1 mouse, s.c. with β-cyclodextrin...

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Identifiers

PMID: 20581737
DOI: 10.1203/PDR.0b013e3181ee4dd2
PMCID: PMC3065173

Genes

NPC1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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