Evidence map›Paper›PMID 28193631›Full record

ArticleJournal of lipid research2017

Histone deacetylase inhibitors correct the cholesterol storage defect in most Niemann-Pick C1 mutant cells.

Nina H Pipalia, Kanagaraj Subramanian, Shu Mao, Harold Ralph, Darren M Hutt, Samantha M Scott, William E Balch, Frederick R Maxfield

Erratum issuedOpen access · hybridAbstract read
In one paragraph

Article in Journal of lipid research, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 40 papers.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
5.0field-weighted citation impact, top 4% 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

40 citing papers in PubMed, 61 citations in OpenAlex.

  1. The histone deacetylase family in health and disease.Signal transduction and targeted therapy · 2026
    Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Niemann-Pick Disease Type C (NPDC) by Mutation ofAntioxidants (Basel, Switzerland) · 2023
    Review
  8. Review
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors at 2 institutions in 2 countries.

Nina H PipaliaDepartment of Biochemistry, Weill Cornell Medical College, New York, NY 10065; and Department of Chemical Physiology and Cell and Molecular Biology.
Kanagaraj SubramanianThe Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA, 92037.
Shu MaoDepartment of Biochemistry, Weill Cornell Medical College, New York, NY 10065; and Department of Chemical Physiology and Cell and Molecular Biology.
Harold RalphDepartment of Biochemistry, Weill Cornell Medical College, New York, NY 10065; and Department of Chemical Physiology and Cell and Molecular Biology.
Darren M HuttThe Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA, 92037.
Samantha M ScottThe Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA, 92037.
William E BalchThe Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA, 92037. Electronic address: webalch@scripps.edu.
Frederick R MaxfieldDepartment of Biochemistry, Weill Cornell Medical College, New York, NY 10065; and Department of Chemical Physiology and Cell and Molecular Biology,. Electronic address: frmaxfie@med.cornell.edu.
Cornell University · USScripps Research Institute · US

Funding

Modulation of Lung Disease by Genetic/Epigenetic ProfilingR01HL095524 · NHLBI · SCRIPPS RESEARCH INSTITUTE, THE · PI William Edward Balch · 2010 to 2026
$7.5M
RECEPTOR-MEDIATED ENDOCYTOSIS: MECHANISM AND FUNCTIONR37DK027083 · NIDDK · WEILL MEDICAL COLL OF CORNELL UNIV · PI MAXFIELD, FREDERICK R. · 1993 to 2014
$6.5M
Support for the Rose F. Kennedy IDDRCU54HD090260 · NICHD · ALBERT EINSTEIN COLLEGE OF MEDICINE, INC · PI MOLHOLM, SOPHIE, WALKLEY, STEVEN UPSHAW · 2016 to 2020
$6.0M
Histone Deacetylase Inhibitors for Treatment of Niemann-Pick C1 DiseaseR01NS092653 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI MAXFIELD, FREDERICK R., WALKLEY, STEVEN UPSHAW · 2015 to 2019
$2.8M
A Phase 1 Dose Escalation Study of Vorinostat in Niemann-Pick C1 DiseaseU01HD079065 · NICHD · WASHINGTON UNIVERSITY · PI JIANG, XUNTIAN, MAXFIELD, FREDERICK R. · 2014 to 2016
$1.2M
NHLBI NIH HHS R01 HL095524NICHD NIH HHS U01 HD079065NICHD NIH HHS U54 HD090260NIDDK NIH HHS R37 DK027083NINDS NIH HHS R01 NS092653
6 · The paper itself

Abstract

Niemann-Pick C (NPC) disease is an autosomal recessive disorder that leads to excessive storage of cholesterol and other lipids in late endosomes and lysosomes. The large majority of NPC disease is caused by mutations in NPC1, a large polytopic membrane protein that functions in late endosomes. There are many disease-associated mutations in NPC1, and most patients are compound heterozygotes. The most common mutation, NPC1

Indexed as

Carrier ProteinsCell LineCholesterolEndoplasmic Reticulum-Associated DegradationEndosomesFibroblastsHistone Deacetylase InhibitorsHumansHydroxamic AcidsIndolesIntracellular Signaling Peptides and ProteinsLysosomesMembrane GlycoproteinsMutationNiemann-Pick C1 ProteinNiemann-Pick Disease, Type CCarrier ProteinsCholesterolHistone Deacetylase InhibitorsHydroxamic AcidsIndolesIntracellular Signaling Peptides and ProteinsMembrane GlycoproteinsNiemann-Pick C1 ProteinNPC1 protein, humanPanobinostatRNA, Small InterferingVorinostatcellular cholesterolcholesterol traffickingdrug therapyinborn errors of metabolismlipid transportNPC1

Identifiers

PMID28193631
PMCPMC5392745
OpenAlexW2952207682

What OpenQuestion holds

Textmetadata
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.