Evidence map›Paper›PMID 41235117›Full record

ArticleFrontiers in pharmacology2025

Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR.

Iwona Pranke, Valeria Capurro, Benoit Chevalier, Emanuela Pesce, Valeria Tomati, Cristina Pastorino, Mairead Kelly-Aubert, Aurelie Hatton, Elise Dreano, Mariateresa Lena and 54 more

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Article in Frontiers in pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Article
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4 · The record

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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

64 authors.

Iwona Pranke *INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Valeria Capurro *UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Benoit Chevalier *INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Emanuela Pesce *UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Valeria TomatiUOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Cristina PastorinoUOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Mairead Kelly-AubertINSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Aurelie HattonINSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Elise DreanoINSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Mariateresa LenaUOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Renata BocciardiUOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Federico ZaraUOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Stefano PantanoUOSD CRR Fibrosi Cistica, Atri, Italy.
Vito TerlizziDepartment of Pediatric Medicine, Meyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Center, Florence, Italy.
Cristina LucantoCentro Hub Fibrosi Cistica, Azienda Ospedaliera Universitaria Policlinico G. Martino, Messina, Italy.
Stefano CostaDepartment of Pediatrics, University Hospital "G Martino", Messina, Italy.
Laura ClautDepartment of Pediatrics, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Valeria DaccòDepartment of Pediatrics, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Piercarlo PoliDepartment of Pediatrics, Cystic Fibrosis Regional Support Center, University of Brescia, ASST Spedali Civili Brescia, Brescia, Italy.
Massimo MaschioInstitute for Maternal and Child Health-IRCCS "Burlo Garofolo", Trieste, Italy.
Benedetta FabrizziCystic Fibrosis Regional Centre, Unit of Emerging and Immunosuppressed Infectious Diseases, Department of Gastroenterology and Transplantation, Azienda, Italy.
Nicole CaporelliCystic Fibrosis Regional Centre, Unit of Emerging and Immunosuppressed Infectious Diseases, Department of Gastroenterology and Transplantation, Azienda, Italy.
Marco CipolliCystic Fibrosis Center of Verona, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
Sonia VolpiCystic Fibrosis Center of Verona, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
Frederique ChedevergneCystic Fibrosis National Pediatric Reference Center, Pneumo-Allergologie Pédiatrique, Hôpital Necker Enfants Malades, AP-HP, Paris, France.
Laure CossonCentre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital de Clocheville, Tours, France.
Julie MaceyCentre de Ressources et de Compétence de la Mucoviscidose, CHU Pellegrin, Bordeaux, France.
Sophie RamelCentre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre de Perharidy, Roscoff, France.
Laurence WeissCentre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, CHU, Strasbourg, France.
Dominique GrenetCentre de Ressources et de Compétence de la Mucoviscidose, Hôpital Foch, Suresnes, France.
Laurence Le Clainche-VialaCentre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, Hôpital Robert Debré, Paris, France.
Benoit DouvryCentre de Ressources et de Compétence de la Mucoviscidose Mixte, CHIC, Créteil, France.
Bruno RavoninjatovoCentre de Ressources et de Compétence de la Mucoviscidose, American Memoral Hospital, Reims, France.
Camille AudoussetCentre de Ressources et de Compétence de la Mucoviscidose, Institut Cœur Poumons, Lille, France.
Aurélie TatopoulosCHU de Nancy - Hôpitaux de Brabois, Nancy, France.
Bénédicte Richaud-ThiriezCentre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre Hospitalier Jean Minjoz, Besancon, France.
Melissa BaravalleCentre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital d'Enfants de la Timone, Marseille, France.
Guillaume ThouveninCentre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital Trousseau, Paris, France.
Guillaume LabbéCentre de Ressources et de Compétence de la Mucoviscidose, CHU Estaing, Clermont-Ferrand, France.
Marie MittaineCentre de ressources et de compétences pour la mucoviscidose, Hôpital des enfants, CHU Toulouse, Toulouse, France.
Philippe ReixCentre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, Hospices Civils de Lyon, Bron, France.
Isabelle DurieuCentre de Référence Adulte de la Mucoviscidose, Hospices Civils de Lyon, Université de Lyon, Lyon, France.
Julie MankikianCentre hospitalier régional universitaire Bretonneau, Tours, France.
Stéphanie BuiUniversité de Bordeaux, CRCM pédiatrique, center de Recherche Cardio-thoracique de Bordeaux, INSERM U1045, Bordeaux Imaging Center, Bordeaux, France.
Thao Nguyen-KhoaINSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Karim KhoukhPharmacie Delpech, Paris, France.
Clémence MartinInstitut Cochin, Inserm U1016, Paris, France.
Jennifer Da SilvaRespiratory Medicine and Cystic Fibrosis National Reference Center, Hôpital Cochin, AP-HP. Centre Université Paris Cité, Paris, France.
Paola De CarliVaincre La Mucoviscidose, Paris, France.
Carlo CastellaniIRCCS IstitutoGianninaGaslini, CysticFibrosis Center, Genoa, Italy.
Federico CrestaIRCCS IstitutoGianninaGaslini, CysticFibrosis Center, Genoa, Italy.
Luis GaliettaTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.
Anne GuillemautCentre de ressources et de compétences pour la mucoviscidose Adultes, Centre hospitalier régional universitaire de Nancy, Vandœuvre-Lès-Nancy, France.
Emmanuelle GirodonINSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Natacha RemusCentre de Ressources et de Compétence de la Mucoviscidose Mixte, CHIC, Créteil, France.
Mathis BulcaenLaboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium.
Marjolein EnsinckLaboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium.
Miroslaw ZajacDepartment of Physics and Biophysics, Institute of Biology, Warsaw University of Life Sciences, Warsaw, Poland.
Marianne CarlonLaboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium.
Jean LeBihanCentre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre de Perharidy, Roscoff, France.
Pierre-Régis BurgelUniversité Paris-Cité, Paris, France.
Isabelle Sermet-Gaudelus *INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Alexandre Hinzpeter *INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.
Nicoletta Pedemonte *UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Rationale: Respiratory status of people with Cystic Fibrosis (pwCF) carrying N1303K is improved by Elexacaftor/Tezacaftor/Ivacaftor (ETI) but, contrary to other mutations, the impact on sweat test results is limited. Methods: To explore this discrepancy, we implemented new sweat gland and respiratory cell lines stably expressing Wild type (WT)-, F508del- and N1303K-CFTR. CFTR dependent chloride (Cl Results: In the airway and the sweat gland cells expressing F508del-CFTR, ETI induced maturation of CFTR and increased Cl Conclusion: N1303K-CFTR shows tissue specific correction and suboptimal response to ETI which can be improved by API.

Indexed as

airway epitheliumCFCFTR (cystic fibrosis transmembrane conductance regulator)CFTR modulatorcystic fibrosiselexacaftor/tezacaftor/ivacaftor (ETI)N1303K-CFTRsweat gland

Identifiers

PMID41235117
PMCPMC12605165

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