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Article

Chemical screen to reduce sterol accumulation in Niemann-Pick C disease cells identifies novel lysosomal acid lipase inhibitors.

Biochimica et biophysica acta - 1 Dec 2009

Rosenbaum Anton I, Rujoi Madalina, Huang Amy Y, Du Hong, Grabowski Gregory A, Maxfield Frederick R

Abstract excerpt

Niemann-Pick C disease (NPC) is a lysosomal storage disorder causing abnormal accumulation of unesterified free cholesterol in lysosomal storage organelles. High content phenotypic microscopy chemical screens in both human and hamster NPC-deficient cells have identified several compounds that partially revert the NPC phenotype. Cell biological and biochemical studies show that several of these molecules inhibit...

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Identifiers

PMID: 19699313
DOI: 10.1016/j.bbalip.2009.08.005
PMCID: PMC2783675

Genes

NPC1

Provenance

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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