Evidence map›Paper›PMID 34067708›Full record

ArticleInternational journal of molecular sciences2021

Partial Rescue of F508del-CFTR Stability and Trafficking Defects by Double Corrector Treatment.

Valeria Capurro, Valeria Tomati, Elvira Sondo, Mario Renda, Anna Borrelli, Cristina Pastorino, Daniela Guidone, Arianna Venturini, Alessandro Giraudo, Sine Mandrup Bertozzi and 6 more

Open access · goldAbstract read
In one paragraph

Article in International journal of molecular sciences, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed, 1 pooled it
7.9field-weighted citation impact, top 2% 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

48 citing papers in PubMed, 1 synthesis or guideline pooled it, 69 citations in OpenAlex.

  1. Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2024
    Pooled it
  2. Article
  3. Article
  4. Review
  5. Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  6. Distinct Responses of Cystic Fibrosis Epithelial Cells to SARS-CoV-2 and Influenza A Virus.American journal of respiratory cell and molecular biology · 2025
    Article
  7. Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. Targeting ubiquitination machinery in cystic fibrosis: Where do we stand?Cellular and molecular life sciences : CMLS · 2024
    Review
  13. Article
  14. Review
  15. Article
  16. Advances in the Study of Common and RareJournal of personalized medicine · 2024
    Review
  17. Article
  18. Article
  19. Article
  20. Article
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

16 authors at 3 institutions in 1 country.

Valeria CapurroU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.ORCID 0000-0002-9740-1171
Valeria TomatiU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Elvira SondoU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Mario RendaTelethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Anna BorrelliTelethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Cristina PastorinoU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Daniela GuidoneTelethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Arianna VenturiniTelethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.ORCID 0000-0003-4265-1517
Alessandro GiraudoD3-PharmaChemistry, Fondazione Istituto Italiano di Tecnologia, 16163 Genova, Italy.
Sine Mandrup BertozziAnalytical Chemistry Lab, Istituto Italiano di Tecnologia, Via Morego 30, 16163 Genova, Italy.
Ilaria MusanteU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Fabio BertozziD3-PharmaChemistry, Fondazione Istituto Italiano di Tecnologia, 16163 Genova, Italy.
Tiziano BandieraD3-PharmaChemistry, Fondazione Istituto Italiano di Tecnologia, 16163 Genova, Italy.
Federico ZaraU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Luis J V GaliettaTelethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Nicoletta PedemonteU.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.ORCID 0000-0002-5161-1720
Istituto Giannina Gaslini · ITTelethon Institute Of Genetics And Medicine · ITItalian Institute of Technology · IT

Funding

Fondazione per la Ricerca sulla Fibrosi Cistica FFC6/2019Fondazione per la Ricerca sulla Fibrosi Cistica FFC9/2019Fondazione per la Ricerca sulla Fibrosi Cistica Task Force for Cystic FibrosisFondazione Telethon TMLGCBX16TT
6 · The paper itself

Abstract

Deletion of phenylalanine at position 508 (F508del) in the CFTR chloride channel is the most frequent mutation in cystic fibrosis (CF) patients. F508del impairs the stability and folding of the CFTR protein, thus resulting in mistrafficking and premature degradation. F508del-CFTR defects can be overcome with small molecules termed correctors. We investigated the efficacy and properties of VX-445, a newly developed corrector, which is one of the three active principles present in a drug (Trikafta

Indexed as

AminophenolsAminopyridinesBenzodioxolesBronchiChloride ChannelsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsEpithelial CellsHumansIndolesProtein FoldingPyrazolesPyridinesPyrrolidinesQuinolinesAminophenolsAminopyridinesBenzodioxolesCFTR protein, humanChloride ChannelsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftorelexacaftor, ivacaftor, tezacaftor drug combinationIndolesPyrazolesPyridinesPyrrolidinesQuinolinestezacaftorallosteric folding correctionchloride secretionconformational stabilityelexacaftorprimary bronchial cellsVX-445

Identifiers

PMID34067708
PMCPMC8156943
OpenAlexW3161541109

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.