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

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Title: Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR.
Authors: Pranke I; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Capurro V; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy., Chevalier B; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Pesce E; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy., Tomati V; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy., Pastorino C; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy., Kelly-Aubert M; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Hatton A; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Dreano E; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Lena M; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genoa, Genova, Italy., Bocciardi R; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genoa, Genova, Italy., Zara F; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genoa, Genova, Italy., Pantano S; UOSD CRR Fibrosi Cistica, Atri, Italy., Terlizzi V; Department of Pediatric Medicine, Meyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Center, Florence, Italy., Lucanto C; Centro Hub Fibrosi Cistica, Azienda Ospedaliera Universitaria Policlinico G. Martino, Messina, Italy., Costa S; Department of Pediatrics, University Hospital 'G Martino', Messina, Italy., Claut L; Department of Pediatrics, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy., Daccò V; Department of Pediatrics, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy., Poli P; Department of Pediatrics, Cystic Fibrosis Regional Support Center, University of Brescia, ASST Spedali Civili Brescia, Brescia, Italy., Maschio M; Institute for Maternal and Child Health-IRCCS 'Burlo Garofolo', Trieste, Italy., Fabrizzi B; Cystic Fibrosis Regional Centre, Unit of Emerging and Immunosuppressed Infectious Diseases, Department of Gastroenterology and Transplantation, Azienda, Italy.; Ospedaliero-Universitaria 'Ospedali Riuniti', Ancona, Italy., Caporelli N; Cystic Fibrosis Regional Centre, Unit of Emerging and Immunosuppressed Infectious Diseases, Department of Gastroenterology and Transplantation, Azienda, Italy.; Ospedaliero-Universitaria 'Ospedali Riuniti', Ancona, Italy., Cipolli M; Cystic Fibrosis Center of Verona, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Volpi S; Cystic Fibrosis Center of Verona, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Chedevergne F; Cystic Fibrosis National Pediatric Reference Center, Pneumo-Allergologie Pédiatrique, Hôpital Necker Enfants Malades, AP-HP, Paris, France., Cosson L; Centre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital de Clocheville, Tours, France., Macey J; Centre de Ressources et de Compétence de la Mucoviscidose, CHU Pellegrin, Bordeaux, France., Ramel S; Centre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre de Perharidy, Roscoff, France., Weiss L; Centre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, CHU, Strasbourg, France., Grenet D; Centre de Ressources et de Compétence de la Mucoviscidose, Hôpital Foch, Suresnes, France., Le Clainche-Viala L; Centre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, Hôpital Robert Debré, Paris, France., Douvry B; Centre de Ressources et de Compétence de la Mucoviscidose Mixte, CHIC, Créteil, France., Ravoninjatovo B; Centre de Ressources et de Compétence de la Mucoviscidose, American Memoral Hospital, Reims, France., Audousset C; Centre de Ressources et de Compétence de la Mucoviscidose, Institut Cœur Poumons, Lille, France., Tatopoulos A; CHU de Nancy - Hôpitaux de Brabois, Nancy, France., Richaud-Thiriez B; Centre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre Hospitalier Jean Minjoz, Besancon, France., Baravalle M; Centre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital d'Enfants de la Timone, Marseille, France., Thouvenin G; Centre de Ressources et de Compétence de la Mucoviscidose Enfants, Hôpital Trousseau, Paris, France., Labbé G; Centre de Ressources et de Compétence de la Mucoviscidose, CHU Estaing, Clermont-Ferrand, France., Mittaine M; Centre de ressources et de compétences pour la mucoviscidose, Hôpital des enfants, CHU Toulouse, Toulouse, France., Reix P; Centre de Ressources et de Compétence de la Mucoviscidose Pédiatrique, Hospices Civils de Lyon, Bron, France., Durieu I; Centre de Référence Adulte de la Mucoviscidose, Hospices Civils de Lyon, Université de Lyon, Lyon, France., Mankikian J; Centre hospitalier régional universitaire Bretonneau, Tours, France., Bui S; Université de Bordeaux, CRCM pédiatrique, center de Recherche Cardio-thoracique de Bordeaux, INSERM U1045, Bordeaux Imaging Center, Bordeaux, France., Nguyen-Khoa T; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France.; Laboratory of Biochemistry, Hôpital Universitaire Necker Enfants Malades AP-HP Centre, Paris, France., Khoukh K; Pharmacie Delpech, Paris, France., Martin C; Institut Cochin, Inserm U1016, Paris, France.; Respiratory Medicine and Cystic Fibrosis National Reference Center, Hôpital Cochin, AP-HP. Centre Université Paris Cité, Paris, France.; ERN-Lung CF network, Frankfurt, Germany., Da Silva J; Respiratory Medicine and Cystic Fibrosis National Reference Center, Hôpital Cochin, AP-HP. Centre Université Paris Cité, Paris, France.; ERN-Lung CF network, Frankfurt, Germany., De Carli P; Vaincre La Mucoviscidose, Paris, France., Castellani C; IRCCS IstitutoGianninaGaslini, CysticFibrosis Center, Genoa, Italy., Cresta F; IRCCS IstitutoGianninaGaslini, CysticFibrosis Center, Genoa, Italy., Galietta L; Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy., Guillemaut A; Centre de ressources et de compétences pour la mucoviscidose Adultes, Centre hospitalier régional universitaire de Nancy, Vandœuvre-Lès-Nancy, France., Girodon E; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France.; Service de Médecine Génomique des Maladies de Système et d'Organe, Hôpital Cochin, Paris, France., Remus N; Centre de Ressources et de Compétence de la Mucoviscidose Mixte, CHIC, Créteil, France., Bulcaen M; Laboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium., Ensinck M; Laboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium., Zajac M; Department of Physics and Biophysics, Institute of Biology, Warsaw University of Life Sciences, Warsaw, Poland., Carlon M; Laboratory of Respiratory Diseases and Thoracic Surgery, KU Leuven, Belgium., LeBihan J; Centre de Ressources et de Compétence de la Mucoviscidose Adulte, Centre de Perharidy, Roscoff, France., Burgel PR; Université Paris-Cité, Paris, France.; Institut Cochin, Inserm U1016, Paris, France.; Respiratory Medicine and Cystic Fibrosis National Reference Center, Hôpital Cochin, AP-HP. Centre Université Paris Cité, Paris, France.; ERN-Lung CF network, Frankfurt, Germany., Sermet-Gaudelus I; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France.; Cystic Fibrosis National Pediatric Reference Center, Pneumo-Allergologie Pédiatrique, Hôpital Necker Enfants Malades, AP-HP, Paris, France.; ERN-Lung CF network, Frankfurt, Germany., Hinzpeter A; INSERM, CNRS, Institut Necker Enfants Malades, Paris, France.; Université Paris-Cité, Paris, France., Pedemonte N; UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Source: Frontiers in pharmacology [Front Pharmacol] 2025 Oct 29; Vol. 16, pp. 1661417. Date of Electronic Publication: 2025 Oct 29 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Frontiers Media] Country of Publication: Switzerland NLM ID: 101548923 Publication Model: eCollection Cited Medium: Print ISSN: 1663-9812 (Print) Linking ISSN: 16639812 NLM ISO Abbreviation: Front Pharmacol Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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ISSN:1663-9812
DOI:10.3389/fphar.2025.1661417