Evidence map›Paper›PMID 33616476›Full record

ArticleAmerican journal of respiratory cell and molecular biology2021

Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations.

Venkateshwar Mutyam, Jyoti Sharma, Yao Li, Ning Peng, Jianguo Chen, Li Ping Tang, Emily Falk Libby, Ashvani K Singh, Katja Conrath, Steven M Rowe

Open access · greenAbstract read
In one paragraph

Article in American journal of respiratory cell and molecular biology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
2.3field-weighted citation impact, top 11% 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

14 citing papers in PubMed, 19 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. 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
  5. Article
  6. Article
  7. Receptor-mediated activation of CFTR via prostaglandin signaling pathways in the airway.American journal of physiology. Lung cellular and molecular physiology · 2022
    Article
  8. Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis.Proceedings of the National Academy of Sciences of the United States of America · 2022
    Article
  9. Pharmacological Responses of the G542X-CFTR to CFTR Modulators.Frontiers in molecular biosciences · 2022
    Article
  10. Article
  11. New Therapies to Correct the Cystic Fibrosis Basic Defect.International journal of molecular sciences · 2021
    Review
  12. A Green Light for Stop Mutations.American journal of respiratory cell and molecular biology · 2021
    Article
  13. Article
  14. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 3 institutions in 2 countries.

Venkateshwar MutyamDepartment of Medicine.
Jyoti SharmaDepartment of Medicine.
Yao LiDepartment of Medicine.
Ning PengDepartment of Medicine.
Jianguo ChenDepartment of Medicine.
Li Ping TangDepartment of Medicine.
Emily Falk LibbyDepartment of Medicine.
Ashvani K SinghAbbVie Inc., North Chicago, Illinois; and.
Katja ConrathGalapagos NV, Mechelen, Belgium.
Steven M RoweDepartment of Medicine.ORCID 0000-0001-9045-0133
AbbVie (United States) · USGalapagos (Belgium) · BEUniversity of Alabama at Birmingham · US

Funding

UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0M
Translational Program in CFTR-Related Airway DiseasesR35HL135816 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI ROWE, STEVEN MARK · 2017 to 2022
$6.3M
NHLBI NIH HHS R35 HL135816NIDDK NIH HHS P30 DK072482
6 · The paper itself

Abstract

Premature-termination codons (PTCs) in CFTR (cystic fibrosis [CF] transmembrane conductance regulator) result in nonfunctional CFTR protein and are the proximate cause of ∼11% of CF-causing alleles, for which no treatments exist. The CFTR corrector lumacaftor and the potentiator ivacaftor improve CFTR function with terminal PTC mutations and enhance the effect of readthrough agents. Novel correctors GLPG2222 (corrector 1 [C1]), GLPG3221 (corrector 2 [C2]), and potentiator GLPG1837 compare favorably with lumacaftor and ivacaftor

Indexed as

AminophenolsAminopyridinesAnimalsBenzoatesBenzodioxolesBenzopyransCell LineChloridesCodon, NonsenseCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorEpithelial CellsHumansIon TransportProtein BiosynthesisPyransAminophenolsAminopyridinesBenzoatesBenzodioxolesBenzopyransCFTR protein, humanChloridesCodon, NonsenseCystic Fibrosis Transmembrane Conductance RegulatorGLPG1837GLPG2222ivacaftorlumacaftorPyransPyrazolesQuinolonesCFTRcorrectors and potentiatorpremature-termination codon mutationsreadthrough

Identifiers

PMID33616476
PMCPMC8086047
OpenAlexW3131130467

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.