Evidence map›Paper›PMID 19699313›Full record

ArticleBiochimica et biophysica acta2009

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

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

Open access · greenAbstract read
In one paragraph

Article in Biochimica et biophysica acta, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers.

0numbers the graph read from it
0cells of the map it votes in
38citing papers in PubMed
2.7field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

38 citing papers in PubMed, 58 citations in OpenAlex.

  1. Generation and characterization of human iPSC-derivedbioRxiv : the preprint server for biology · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 2 institutions in 1 country.

Anton I RosenbaumDepartment of Biochemistry, Weill Cornell Medical College, 1300 York Avenue, New York, NY 10065, USA.
Madalina Rujoi
Amy Y Huang
Hong Du
Gregory A Grabowski
Frederick R Maxfield
Cornell University · USCincinnati Children's Hospital Medical Center · US

Funding

RECEPTOR-MEDIATED ENDOCYTOSIS: MECHANISM AND FUNCTIONR37DK027083 · NIDDK · WEILL MEDICAL COLL OF CORNELL UNIV · PI MAXFIELD, FREDERICK R. · 1993 to 2014
$6.5M
RECEPTOR-MEDIATED ENDOCYTOSIS: MECHANISM AND FUNCTIONR01DK027083 · NIDDK · WEILL MEDICAL COLLEGE OF CORNELL UNIV · PI MAXFIELD, FREDERICK R. · 1986 to 2004
$2.0M
NIDDK NIH HHS DK27083NIDDK NIH HHS R01 DK027083NIDDK NIH HHS R37 DK027083
6 · The paper itself

Abstract

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 lysosomal acid lipase, the enzyme that hydrolyzes LDL-derived triacylglycerol and cholesteryl esters. The effects of reduced lysosomal acid lipase activity in lowering cholesterol accumulation in NPC mutant cells were verified by RNAi-mediated knockdown of lysosomal acid lipase in NPC1-deficient human fibroblasts. This work demonstrates the utility of phenotypic cellular screens as a means to identify molecular targets for altering a complex process such as intracellular cholesterol trafficking and metabolism.

Indexed as

AnimalsCattleCHO CellsCricetinaeCricetulusDrug Evaluation, PreclinicalEnzyme InhibitorsHumansLipoprotein LipaseMilkMutationNiemann-Pick Disease, Type CReproducibility of ResultsRNA InterferenceRNA, Small InterferingSmall Molecule LibrariesEnzyme InhibitorsLipoprotein LipaseRNA, Small InterferingSmall Molecule LibrariesSterol EsteraseSterols

Identifiers

PMID19699313
PMCPMC2783675
OpenAlexW2089085630

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.