Evidence map›Paper›PMID 27895116›Full record

ArticleThe Journal of biological chemistry2017

Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation Product.

Peter M Haggie, Puay-Wah Phuan, Joseph-Anthony Tan, Haijin Xu, Radu G Avramescu, Doranda Perdomo, Lorna Zlock, Dennis W Nielson, Walter E Finkbeiner, Gergely L Lukacs and 1 more

Open access · hybridAbstract read
In one paragraph

Article in The Journal of biological chemistry, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 65 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
65citing papers in PubMed, 1 pooled it
5.9field-weighted citation impact, top 3% 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

65 citing papers in PubMed, 1 synthesis or guideline pooled it, 90 citations in OpenAlex.

  1. Pooled it
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  14. A W1282X cystic fibrosis mouse allows the study of pharmacological and gene-editing therapeutics to restore CFTR function.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
    Article
  15. Article
  16. Systematic deletion of symmetricalNAR molecular medicine · 2024
    Article
  17. Article
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  20. Review

5 more citing papers are in PubMed but not listed here.

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

11 authors at 2 institutions in 2 countries.

Peter M HaggieFrom the Departments of Medicine, peter.haggie@ucsf.edu.
Puay-Wah PhuanFrom the Departments of Medicine.
Joseph-Anthony TanFrom the Departments of Medicine.
Haijin Xuthe Department of Physiology, McGill University, Montreal, Quebec H3G 1Y6, Canada.
Radu G Avramescuthe Department of Physiology, McGill University, Montreal, Quebec H3G 1Y6, Canada.
Doranda Perdomothe Department of Physiology, McGill University, Montreal, Quebec H3G 1Y6, Canada.
Lorna ZlockPathology, and.
Dennis W NielsonPediatrics, University of California, San Francisco, California 94143-0521 and.
Walter E FinkbeinerPathology, and.
Gergely L Lukacsthe Department of Physiology, McGill University, Montreal, Quebec H3G 1Y6, Canada gergely.lukacs@mcgill.ca.
Alan S VerkmanFrom the Departments of Medicine, alan.verkman@ucsf.edu.
University of California, San Francisco · USMcGill University · CA

Funding

Translational InformaticsP30CA082103 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Jennifer Leigh Clarke · 1999 to 2026
$209.7M
SynthesisP30DK072517 · NIDDK · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI VERKMAN, ALAN S · 2005 to 2022
$18.1M
Translational Approaches to Develop Drug Therapy for DiarrheaR24DK099803 · NIDDK · JOHNS HOPKINS UNIVERSITY · PI DONOWITZ, MARK · 2014 to 2019
$8.9M
Functional Role of Aquaporins in Eye PhysiologyR01EY013574 · NEI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI VERKMAN, ALAN S · 2002 to 2022
$7.6M
Application of Novel Optical Methods to Cell DynamicsR37EB000415 · NIBIB · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI VERKMAN, ALAN S · 2007 to 2016
$4.1M
Correction of defective functional expression of CFTR in cystic fibrosisR01DK075302 · NIDDK · MCGILL UNIVERSITY · PI LUKACS, GERGELY L. · 2006 to 2020
$3.3M
Application of Novel Optical Methods to Cell DynamicsR01EB000415 · NIBIB · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI VERKMAN, ALAN S · 2002 to 2006
$1.7M
Moving On: Interactive Guide to Breast Cancer SurvivorsR44CA091661 · NCI · HEALTHMARK MULTIMEDIA, LLC · PI BRINK, SUSAN G · 2003 to 2004
$816k
Host-Targeted Homoserine Lactone Inhibitor as Adjunctive Antipseudomonal TherapyR21AI111634 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI HAGGIE, PETER MICHAEL · 2014 to 2015
$428k
CIHRNCI NIH HHS P30 CA082103NCI NIH HHS R44 CA091661NEI NIH HHS R01 EY013574NIAID NIH HHS R21 AI111634NIBIB NIH HHS R01 EB000415NIBIB NIH HHS R37 EB000415NIDDK NIH HHS P30 DK072517NIDDK NIH HHS R01 DK075302NIDDK NIH HHS R24 DK099803
6 · The paper itself

Abstract

W1282X is the fifth most common cystic fibrosis transmembrane regulator (CFTR) mutation that causes cystic fibrosis. Here, we investigated the utility of a small molecule corrector/potentiator strategy, as used for ΔF508-CFTR, to produce functional rescue of the truncated translation product of the W1282X mutation, CFTR

Indexed as

Mutation, MissenseAmino Acid SubstitutionAminophenolsAnimalsCells, CulturedCystic Fibrosis Transmembrane Conductance RegulatorHumansPiperazinesQuinolonesRatsRats, Inbred F344AminophenolsCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorivacaftorPiperazinesQuinolonescystic fibrosiscystic fibrosis transmembrane conductance regulator (CFTR)drug discoverydrug screeningsmall molecule

Identifiers

PMID27895116
PMCPMC5247652
OpenAlexW2558608270

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.