Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR
N1303K-CFTR shows tissue specific correction and suboptimal response to ETI which can be improved by API.
Authors
- Renata Bocciardi (ORCID: https://orcid.org/0000-0001-8415-3802)
- N. Caporelli
- Camille Audousset (ORCID: https://orcid.org/0000-0003-2554-8286)
- Sophie Ramel (ORCID: https://orcid.org/0000-0002-9195-2183)
- Melissa Baravalle
- Stefano Costa (ORCID: https://orcid.org/0000-0001-5640-1901)
- Mairead Kelly (ORCID: https://orcid.org/0000-0001-8729-6948)
- I. Durieu (ORCID: https://orcid.org/0000-0002-3874-5580)
- M. Ensinck (ORCID: https://orcid.org/0000-0003-0580-0819)
- Mirosław Zając (ORCID: https://orcid.org/0000-0002-4287-4377)
- Alexandre Hinzpeter (ORCID: https://orcid.org/0000-0001-6920-7761)
- Isabelle Sermet‐Gaudelus (ORCID: https://orcid.org/0000-0001-5537-9482)
- Marianne Sylvia Carlon (ORCID: https://orcid.org/0000-0002-8263-0350)
- Piercarlo Poli (ORCID: https://orcid.org/0000-0002-6417-4216)
- Emanuela Pesce (ORCID: https://orcid.org/0000-0002-9070-4479)
- Laure Cosson (ORCID: https://orcid.org/0000-0001-9469-9796)
- Philippe Reix (ORCID: https://orcid.org/0000-0002-9192-8335)
- S. Bui (ORCID: https://orcid.org/0000-0003-3576-2518)
- M. Bulcaen
- Julie Macey (ORCID: https://orcid.org/0000-0001-6961-7170)
- B. Chevalier (ORCID: https://orcid.org/0000-0003-0863-6022)
- Benoît Douvry
- Federico Cresta (ORCID: https://orcid.org/0000-0002-2483-5374)
- Aurélie Tatopoulos
- Marco Cipolli (ORCID: https://orcid.org/0000-0001-5652-7248)
- Cristina Pastorino (ORCID: https://orcid.org/0000-0001-8565-4393)
- F. Chedevergne
- Valeria Daccò (ORCID: https://orcid.org/0000-0002-7497-9165)
- Vito Terlizzi (ORCID: https://orcid.org/0000-0003-1106-4424)
- Aurélie Hatton (ORCID: https://orcid.org/0000-0002-8985-9625)
- Cristina Lucanto
- Jennifer Da Silva (ORCID: https://orcid.org/0000-0001-6897-3616)
- Sonia Volpi (ORCID: https://orcid.org/0000-0003-4979-5564)
- Laurence Le Clainche-Viala
- Nicoletta Pedemonte (ORCID: https://orcid.org/0000-0002-5161-1720)
- Laura Claut
- Federico Zara (ORCID: https://orcid.org/0000-0001-9744-5222)
- Marie Mittaine (ORCID: https://orcid.org/0000-0003-2385-495X)
- Elise Dréano
- Luis J. V. Galietta (ORCID: https://orcid.org/0000-0001-8686-3461)
- Bénédicte Richaud-Thiriez
- Bruno Ravoninjatovo
- Emmanuelle Girodon (ORCID: https://orcid.org/0000-0001-7913-4682)
- Guillaume Thouvenin (ORCID: https://orcid.org/0000-0003-0528-5458)
- Iwona Pranke (ORCID: https://orcid.org/0000-0003-1734-7425)
- Clémence Martin (ORCID: https://orcid.org/0000-0003-4385-5888)
- Stefano Pantano
- Mariateresa Lena (ORCID: https://orcid.org/0000-0001-9661-2885)
- Valeria Tomati (ORCID: https://orcid.org/0000-0003-3761-1916)
- Dominique Grenet
- Pierre‐Régis Burgel (ORCID: https://orcid.org/0000-0003-0903-9828)
- P. De Carli
- Benedetta Fabrizzi (ORCID: https://orcid.org/0000-0003-2358-5470)
- Thao Nguyen‐Khoa (ORCID: https://orcid.org/0000-0003-3738-7299)
- Valeria Capurro (ORCID: https://orcid.org/0000-0002-9740-1171)
- Carlo Castellani (ORCID: https://orcid.org/0000-0003-1916-0963)
- Jean Lebihan
- Karim Khoukh
- Anne Guillemaut
- Massimo Maschio
- Laurence Weiss (ORCID: https://orcid.org/0000-0003-4064-6650)
- Natacha Rémus
- Guillaume Labbé
- Julie Mankikian
Institutions
- Université Claude Bernard Lyon 1 (FR)
- Hôpital Necker-Enfants Malades (FR)
- Centre National de la Recherche Scientifique (FR)
- Université de Bordeaux (FR)
- Inserm (FR)
- Warsaw University of Life Sciences (PL)
- Université Paris Cité (FR)
- IRCCS Materno Infantile Burlo Garofolo (IT)
- Istituto Giannina Gaslini (IT)
- Martin University (US)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Sorbonne Université (FR)
- Institut Necker Enfants Malades (FR)
- Hôpital Armand-Trousseau (FR)
- Centre Hospitalier Universitaire de Besançon (FR)
- Hôpital Cochin (FR)
- Azienda Ospedaliera Universitaria Integrata Verona (IT)
- Vaincre la Mucoviscidose (FR)
- Centre Hospitalier Universitaire de Tours (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
- Centre Hospitalier Régional et Universitaire de Nancy (FR)
- Hospices Civils de Lyon (FR)
- Cystic Fibrosis Research Foundation (IT)
- Hôpital d'Enfants (FR)
- Azienda Ospedaliero Universitaria Ospedali Riuniti (IT)
- Hôpital Pellegrin (FR)
- Meyer Children's Hospital (IT)
- Health Services and Performance Research Laboratory (FR)
- Hôpital Robert-Debré (FR)
- Institute of Biochemistry and Biophysics, Polish Academy of Sciences (PL)
- Centre Hospitalier Universitaire de Clermont-Ferrand (FR)
- Azienda Ospedaliera Universitaria Policlinico "G. Martino" (IT)
- Institut Cochin (FR)
- Hôpital des Enfants (FR)
- Telethon Institute Of Genetics And Medicine (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Centre de Recherche Cardio-Thoracique de Bordeaux (FR)
- Hôpital Foch (FR)
- Research on healthcare performance (FR)
- University of Brescia (IT)
- University of Genoa (IT)
- KU Leuven (BE)
Publication Details
- Journal
- Frontiers in Pharmacology
- Published
- 2025-10-29
- DOI
- https://doi.org/10.3389/fphar.2025.1661417
- Primary Topic
- Atrial Fibrillation Management and Outcomes
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- American Breast Cancer Foundation
- Ministero della Salute
- Fondazione per la Ricerca sulla Fibrosi Cistica