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Article

Identification of a pharmaceutical compound that partially corrects the Niemann-Pick C phenotype in cultured cells.

Journal of lipid research - 1 Oct 2002

Liscum Laura, Arnio Emily, Anthony Monique, Howley Andrea, Sturley Stephen L, Agler Michele

Abstract excerpt

Niemann-Pick C (NPC) is an autosomal recessive lysosomal lipid storage disease characterized by progressive central nervous system degeneration. In cultured human NPC fibroblasts, LDL-derived cholesterol accumulates in lysosomes and endosomes, LDL-cholesterol transport from endocytic compartments to other cellular compartments is delayed, and LDL does not elicit normal homeostatic responses. Currently, there is...

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Identifiers

PMID: 12364555
DOI: 10.1194/jlr.m200179-jlr200

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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