Bienvenu(e) sur le site de l'Institut Necker Enfants Malades

Etudier les mécanismes responsable de la mucoviscidose et proposer les nouvelle thérapies

de la science fondamentale :
  • CFTR
  • canaux ioniques
à la recherche translationnelle :
  • mucoviscidose
  • inflammation
Isabelle Sermet Gaudelus
Mucoviscidose et autres maladies épithéliales respiratoires par défaut de repliement protéique

Isabelle Sermet-Gaudelus is MD, PhD, Professor of Pediatrics and head of the Pediatric Cystic Fibrosis Center. Hopital Necker-Enfants Malades, which is one of the biggest in Europe. She is presently the co-director with A Edelman of Team 2/INSERM U 1151: Epithelial channellopathies, Cystic Fibrosis and other diseases. Her translational research is based on phenotype-genotype studies for Cystic Fibrosis using different epithelial electrophysiological measurements and biochemical tests both in vivo and in tissues derived from patients . Basic research focuses on impact of selected interacting proteins on CFTR (dys)function and identification of partner proteins as novel targets for therapies.

Aleksander Edelman, CNRS Research Director (DR1), Head of the team 2 (Epithelial Channel diseases: cystic fibrosis and other diseases) at INSERM U1151, Paris France. Aleksander Edelman is an expert in epithelial physiology, proteomics and protein biochemistry. The team he is managing investigates the mechanisms underlying protein misfolded diseases (PMDs), such as cystic fibrosis and alpha 1 ant-trypsin deficiency focusing on protein-protein interactions, gene regulation, inflammation and ion transport. The final aim of AE research is to unmask novel targets for PMDs. Aleksander Edelman is the scientific director of proteomic core facility at Necker site in Paris.


We investigate misfolded protein biogenesis and correction, taking the CFTR protein as a model. We focus on the role of intermediary filaments in assisting CFTR trafficking to the apical membrane. Our translational research aims to implement personalized therapy in patients with cystic fibrosis.


Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene coding for the CFTR protein. The most frequent mutation is the deletion of the 508th aminoacid (F508del), which affects the folding and trafficking to the membrane of the protein and results in ion transport abnormalities and defective resolution of inflammation in the airways. Recent studies have demonstrated that CFTR mutation-targeted therapies constitute an efficient treatment for CF.

Our research focuses on 3 different aspects:

  1. Development of new correctors for F508delCFTR
    Using in silico molecular docking, we have identified compounds which interrupt the interaction between an intermediary filament, keratin 8 (K8), and F508del-CFTR. Inhibition of this interaction restores F508del-CFTR activity. This strategy is extended to other misfolded proteins such as alpha 1 anti-trypsin (AAT-Z mutant) which is involved in A1AT deficiency, another misfolding lossof-function genetic lung disease.
  2. Premature Stop codon (PTC) mutations
    The PTCs of the CFTR gene are associated with severe forms as a consequence of truncated, non-functional polypeptides synthesis in concert with the reduction of transcript level by nonsense-mediated decay (NMD). Therapeutic approach aiming at promoting translational readthrough is ineffective so far.
  3. Impact of various CFTR mutations and correction on airway epithelial functions.
    This includes in vitro and in vivo regulation of ion transport, airway surface liquid layer height and pH and specialized pro-resolution mediators (lipoxins and resolvins) production.


Research objectives

The first objective is to investigate in deep the mechanisms underlying K8 interaction with misfolded proteins (F508del-CFTR and AAT-Z mutant). The sites of interaction with K8 are actively searched to modulate misfolded proteins trafficking.

The second objective is to optimize the efficacy of the most active correctors by a molecular modeling approach and biophysical technics to get insight into the dynamic structure of the isolated F508del-CFTR 3D structure. We aim to unravel correctors binding sites by directed mutagenesis and to investigate efficacy of these correctors on biogenesis and activity of F508del-CFTR.

The third objective is to develop a more efficacious therapy for nonsense mutations by targeting the molecular pathogenesis of PTCs in CF.

The fourth objective is to establish tools to implement a national program for personalized therapy in CF including a national biobank and characterization of novel biomarkers.

5 main publications

  • Odolczyk N, Fritsch J, Norez C, Servel N, da Cunha MF, Bitam S, et al. Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain. EMBO Mol Med. 2013;5(10):1484-501.
  • Bakouh N, Bienvenu T, Thomas A, Ehrenfeld J, Liote H, Roussel D, et al. Characterization of SLC26A9 in patients with CF-like lung disease.Hum Mutat. 2013;34(10):1404-14.
  • Faure G, Bakouh N, Lourdel S, Odolczyk N, Premchandar A, Servel N, et al. Rattlesnake Phospholipase A2 Increases CFTR-Chloride Channel Current and Corrects ∆F508CFTR Dysfunction: Impact in Cystic Fibrosis. J Mol Biol. 2016;428(14):2898-915.
  • Le Henaff C, Faria Da Cunha M, Hatton A, Tondelier D, Marty C, Collet C, et al. Genetic deletion of keratin 8 corrects the altered bone formation and osteopenia in a mouse model of cystic fibrosis. Hum Mol Genet. 2016;25(7):1281-93.
  • Premchandar A, Kupniewska A, Bonna A, Faure G, Fraczyk T, Roldan A, et al. New insights into interactions between the nucleotide-binding domain of CFTR and keratin 8. Protein Sci. 2017;26(2):343-354.
30 dernières publications


  • Pharmacokinetic and Pharmacodynamic Optimization of Antibiotic Therapy in Cystic Fibrosis Patients: Current Evidences, Gaps in Knowledge and Future Directions. Magréault S, Roy C, Launay M, Sermet-Gaudelus I, Jullien V. Clin Pharmacokinet. 2021 Jan 24. doi: 10.1007/s40262-020-00981-0. Online ahead of print. PMID: 33486720 Review.
  • Modulators of CFTR. Updates on clinical development and future directions. Bardin E, Pastor A, Semeraro M, Golec A, Hayes K, Chevalier B, Berhal F, Prestat G, Hinzpeter A, Gravier-Pelletier C, Pranke I, Sermet-Gaudelus I. Eur J Med Chem. 2021 Mar 5;213:113195. doi: 10.1016/j.ejmech.2021.113195. Epub 2021 Jan 16. PMID: 33524685
  • "Il faut continuer à poser des questions" patient reported outcome measures in cystic fibrosis: An anthropological perspective. Coucke R, Chansard A, Bontemps V, Grenet D, Hubert D, Martin C, Lammertyn E, Bardin E, Bulteel V, Chedevergne F, Bourgeois ML, Burgel PR, Honore I, de Keyser H, Kirszenbaum M, de Carli P, Sermet-Gaudelus I, Hayes K; European Cystic Fibrosis Society-Clinical Trials Network Patient Advisory Group. J Cyst Fibros. 2021 Feb 26:S1569-1993(21)00044-8. doi: 10.1016/j.jcf.2021.02.009. Online ahead of print. PMID: 33648900
  • New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation. Bitam S, Elbahnsi A, Creste G, Pranke I, Chevalier B, Berhal F, Hoffmann B, Servel N, Tondelier D, Hatton A, Moquereau C, Faria Da Cunha M, Pastor A, Lepissier A, Hinzpeter A, Mornon J-P, Prestat G, Edelman A, Callebaut I, Gravier-Pelletier C, Sermet-Gaudelus I. Scientific Report. Accepted for publication January 2021. (IF 4)
  • Airway Surface Liquid pH Regulation in Airway Epithelium Current Understandings and Gaps in Knowledge – Miroslaw Zajac 1,[M1] Elise Dreano 2,3, Aurelie Edwards 4, Gabrielle Planelles [M2] 3,5,6 and Isabelle Sermet-Gaudelus[M3] 2,3,7,8,*
  • Involvement of CFTR in the pathogenesis of pulmonary arterial hypertension. Le Ribeuz H, To L, Ghigna MR, Martin C, Nagaraj C, Dreano E, Rucker-Martin C, Girerd B, Bouliguan J, Pechoux C, Lambert M, Boet A, Issard J, Mercier O, Hoetzenecker K, Manoury B, Becq F, Burgel PR, Cottart CH, Olschewski A, Sermet-Gaudelus I, Perros F, Humbert M, Montani D, Antigny F. Eur Respir J. 2021 May 28:2000653. doi: 10.1183/13993003.00653-2020. Online ahead of print. PMID: 33926975
  • Achromobacter xylosoxidans airway infection is associated with lung disease severity in children with cystic fibrosis. Marsac C, Berdah L, Thouvenin G, Sermet-Gaudelus I, Corvol H. ERJ Open Res. 2021 May 31;7(2):00076-2021. doi: 10.1183/23120541.00076-2021. eCollection 2021 Apr. PMID: 34084788
  • Prior infection by seasonal coronaviruses, as assessed by serology, does not prevent SARS-CoV-2 infection and disease in children, France, April to June 2020. Sermet-Gaudelus I, Temmam S, Huon C, Behillil S, Gajdos V, Bigot T, Lurier T, Chrétien D, Backovic M, Delaunay-Moisan A, Donati F, Albert M, Foucaud E, Mesplées B, Benoist G, Faye A, Duval-Arnould M, Cretolle C, Charbit M, Aubart M, Auriau J, Lorrot M, Kariyawasam D, Fertitta L, Orliaguet G, Pigneur B, Bader-Meunier B, Briand C, Enouf V, Toubiana J, Guilleminot T, van der Werf S, Leruez-Ville M, Eloit M. Euro Surveill. 2021 Apr;26(13):2001782. doi: 10.2807/1560-7917.ES.2021.26.13.2001782. PMID: 33797390
  • Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation. Oren YS, Irony-Tur Sinai M, Golec A, Barchad-Avitzur O, Mutyam V, Li Y, Hong J, Ozeri-Galai E, Hatton A, Leibson C, Carmel L, Reiter J, Sorscher EJ, Wilton SD, Kerem E, Rowe SM, Sermet-Gaudelus I, Kerem B. J Cyst Fibros. 2021 Jul 2:S1569-1993(21)01287-X. doi: 10.1016/j.jcf.2021.06.003. Online ahead of print. PMID: 34226157
  • Towards harmonization of solutions used for cystic fibrosis diagnosis by nasal potential difference measurements: a formulation approach with CHESS® software. Yayé HS, Caudron E, Sermet-Gaudelus I, Nicolis I, Prognon P, Pradeau D. Ann Pharm Fr. 2021 May 13:S0003-4509(21)00071-7. doi: 10.1016/j.pharma.2021.05.002. Online ahead of print. PMID: 33992643
  • Sweat Chloride Testing and Nasal Potential Difference (NPD) Are Primary Outcome Parameters in Treatment with Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators by Isabelle Sermet-Gaudelus, Thao Nguyen-Khoa, Aurélie Hatton, Kate Hayes, Iwona Pranke Academic Editors: Cornelis K. van der Ent and Harry G. M. Heijerman J. Pers. Med. 2021, 11(8), 729; Received: 8 June 2021 / Revised: 15 July 2021 / Accepted: 20 July 2021 / Published: 27 July 2021
  • 2020



    Suppressing 'nonsense' in cystic fibrosis. Hinzpeter A, Sermet-Gaudelus I, Sheppard DN. J Physiol. 2020 Feb;598(3):429-430. doi: 10.1113/JP279267. Epub 2020 Jan 16. PMID: 31869855 No abstract available

    Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF).  Konstan MW, VanDevanter DR, Rowe SM, Wilschanski M, Kerem E, Sermet-Gaudelus I, DiMango E, Melotti P, McIntosh J, De Boeck K; ACT CF Study Group. J Cyst Fibros. 2020 Jul;19(4):595-601. doi: 10.1016/j.jcf.2020.01.007. Epub 2020 Jan 23. PMID: 31983658

    Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. Burgel PR, Munck A, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Lemonnier L, Dehillotte C, Da Silva J, Paillasseur JL, Hubert D; French Cystic Fibrosis Reference Network Study Group. Am J Respir Crit Care Med. 2020 Jan 15;201(2):188-197. doi: 10.1164/rccm.201906-1227OC. PMID: 31601120

    Genetic diagnosis in practice: From cystic fibrosis to CFTR-related disorders. Pagin A, Sermet-Gaudelus I, Burgel PR. Arch Pediatr. 2020 Feb;27 Suppl 1:eS25-eS29. doi: 10.1016/S0929-693X(20)30047-6. PMID: 32172933 Review.

    Targeting different binding sites in the CFTR structures allows to synergistically potentiate channel activity.  Froux L, Elbahnsi A, Boucherle B, Billet A, Baatallah N, Hoffmann B, Alliot J, Zelli R, Zeinyeh W, Haudecoeur R, Chevalier B, Fortuné A, Mirval S, Simard C, Lehn P, Mornon JP, Hinzpeter A, Becq F, Callebaut I, Décout JL. Eur J Med Chem. 2020 Mar 15;190:112116. doi: 10.1016/j.ejmech.2020.112116. Epub 2020 Feb 9. PMID: 32078860

    Current and future diagnosis of cystic fibrosis: Performance and limitations. Bienvenu T, Nguyen-Khoa T. Arch Pediatr. 2020 Feb;27 Suppl 1:eS19-eS24. PMID: 32172931 Review.

    Mucoviscidosis: fisiopatología, genética, aspectos clínicos y terapéuticos S. Noël, I. Sermet-Gaudelus

    EMC Pediatr. 2020 Mar; 55(1): 1–23. Spanish. Published online 2020 Mar 4. doi: 10.1016/S1245-1789(20)43427-4

    Penetrance is a critical parameter for assessing the disease liability of CFTR variants. Boussaroque A, Audrézet MP, Raynal C, Sermet-Gaudelus I, Bienvenu T, Férec C, Bergougnoux A, Lopez M, Scotet V, Munck A, Girodon E. J Cyst Fibros. 2020 Nov;19(6):949-954. doi: 10.1016/j.jcf.2020.03.019. Epub 2020 Apr 20. PMID: 32327388

    Impact of COVID-19 on people with cystic fibrosis. Carla Colombo, Pierre-Régis Burgel, Silvia Gartner, Silke van Koningsbruggen-Rietschel, Lutz Naehrlich, Isabelle Sermet-Gaudelus, Kevin W Southern Lancet Respir Med. 2020 May; 8(5): e35–e36. Published online 2020 Apr 15. doi: 10.1016/S2213-2600(20)30177-6

    Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function. Burgel PR, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Biouhee T, Hubert D, Munck A, Lemonnier L, Dehillotte C, Silva JD, Paillasseur JL, Martin C; French Cystic Fibrosis Reference Network study group. J Cyst Fibros. 2020 Jun 23:S1569-1993(20)30752-9. doi: 10.1016/j.jcf.2020.06.012. Online ahead of print. PMID: 32591294

    Guidelines for the management of children at risk of secondary bone fragility: Expert opinion of a French working group. Edouard T, Guillaume-Czitrom S, Bacchetta J, Sermet-Gaudelus I, Dugelay E, Martinez-Vinson C, Salles JP, Linglart A. Arch Pediatr. 2020 Oct;27(7):393-398. doi:  10.1016/j.arcped.2020.07.008. Epub 2020 Sep 10. PMID: 32921532

    A phare 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation. Davies JC, Sermet-Gaudelus I, Naehrlich L, Harris RS, Campbell D, Ahluwalia N, Short C, Haseltine E, Panorchan P, Saunders C, Owen CA, Wainwright CE; VX16-661-115 Investigator Group. J Cyst Fibros. 2021 Jan;20(1):68-77. doi: 10.1016/j.jcf.2020.07.023. Epub 2020 Sep 21. PMID: 32967799

    Urinary Exosomes of Patients with Cystic Fibrosis Unravel CFTR-Related Renal Disease. Sebastien Gauthier, Iwona Pranke, Vincent Jung, Loredana Martignetti, Véronique Stoven, Thao Nguyen-Khoa, Michaela Semeraro, Alexandre Hinzpeter, Aleksander Edelman, Ida Chiara Guerrera, Isabelle Sermet-Gaudelus Int J Mol Sci. 2020 Sep; 21(18): 6625. Published online 2020 Sep 10. doi: 10.3390/ijms21186625

    Immunological and virological profile of children with chilblain‐like lesions and SARS‐CoV‐2   L. Fertitta, A. Welfringer, A. Ouedrani, L. Polivka, S. Chhun, L. Chatenoud, J. Fourgeaud, S. Hadj‐Rabia, S. Temmam, M. Eloit, Sermet‐Gaudelus I, C. Bodemer. J Eur Acad Dermatol Venereol. 2020 Oct 3 : 10.1111/jdv.16972. doi: 10.1111/jdv.16972 [Epub ahead of print]

    Updated guidance on the management of children with cystic fibrosis transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID). Barben J, Castellani C, Munck A, Davies JC, de Winter-de Groot KM, Gartner S, Kashirskaya N, Linnane B, Mayell SJ, McColley S, Ooi CY, Proesmans M, Ren CL, Salinas D, Sands D, Sermet-Gaudelus I, Sommerburg O, Southern KW; European CF Society Neonatal Screening Working Group (ECFS NSWG). J Cyst Fibros. 2020 Nov 27:S1569-1993(20)30909-7. doi:  10.1016/j.jcf.2020.11.006. Online ahead of print. PMID: 33257262

    Pseudo-engelures de l’enfant et SARS-CoV-2 : quel lien ? L. Fertitta, A. Welfringer, L. Polivka, S. Chhun, A. Ouedrani, L. Chatenoud, S. Hadj-Rabia, I. Sermet-Gaudelus, C. Bodemer, Ann Dermatol Venereol. 2020 Dec; 147(12): A196–A197. French. Publication en ligne 2020 nov. 25. DOI : 10.1016/j.annder.2020.09.235

    Arterial abnormalities identified in kidneys transplanted into children during the COVID-19 pandemic.  Berteloot L, Berthaud R, Temmam S, Lozach C, Zanelli E, Blanc T, Heloury Y, Capito C, Chardot C, Sarnacki S, Garcelon N, Lacaille F, Charbit M, Pastural M, Rabant M, Boddaert N, Leruez-Ville M, Eloit M, Sermet-Gaudelus I, Dehoux L, Boyer O. Am J Transplant. 2020 Dec 21. doi: 10.1111/ajt.16464. Online ahead of print. PMID: 33346946

    Exon identity influences splicing induced by exonic variants and in silico prediction efficacy. Martin N, Bergougnoux A, Baatallah N, Chevalier B, Varilh J, Baux D, Costes B, Fanen P, Raynal C, Sermet-Gaudelus I, Girodon E, Taulan-Cadars M, Hinzpeter A. J Cyst Fibros. 2020 Dec 16:S1569-1993(20)30935-8. doi: 10.1016/j.jcf.2020.12.003. Online ahead of print. PMID: 33341408


  • Burgel PR, Munck A, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Lemonnier L, Dehilotte C, Da Silva J, Paillasseur JL, Hubert D; French Cystic Fibrosis Reference Network study group.Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. Am J Respir Crit Care Med. 2019 Oct 11. doi: 10.1164/rccm.201906-1227OC. [Epub ahead of print]
  • Chedevergne F, Sermet-Gaudelus I.Prevention of osteoporosis in cystic fibrosis. Curr Opin Pulm Med. 2019 Nov;25(6):660-665. doi: 10.1097/MCP.0000000000000624.
  • Taccetti G, Denton M, Hayes K; ECFS-CTN Microbiology Group, Drevinek P, Sermet-Gaudelus I. A critical review of definitions used to describe Pseudomonas aeruginosa microbiological status in patients with cystic fibrosis for application in clinical trials. J Cyst Fibros. 2019 Sep 13. pii: S1569-1993(19)30867-7. doi: 10.1016/j.jcf.2019.08.014. [Epub ahead of print]
  • Simonin J, Bille E, Crambert G, Noel S, Dreano E, Edwards A, Hatton A, Pranke I, Villeret B, Cottart CH, Vrel JP, Urbach V, Baatallah N, Hinzpeter A, Golec A, Touqui L, Nassif X, Galietta LJV, Planelles G, Sallenave JM, Edelman A, Sermet-Gaudelus I. Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis. Sci Rep. 2019 Apr 24;9(1):6516. doi: 10.1038/s41598-019-42751-4.
  • Rollet-Cohen V, Roux AL, Le Bourgeois M, Sapriel G, El Bahri M, Jais JP, Heym B, Mougari F, Raskine L, Véziris N, Gaillard JL, Sermet-Gaudelus I. Mycobacterium bolletii Lung Disease in Cystic Fibrosis. Chest. 2019 Aug;156(2):247-254. doi: 10.1016/j.chest.2019.03.019. Epub 2019 Mar 29.
  • Pranke I, Golec A, Hinzpeter A, Edelman A, Sermet-Gaudelus I. Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine. Front Pharmacol. 2019 Feb 27;10:121. doi: 10.3389/fphar.2019.00121. eCollection 2019.
  • Tan X, Coureuil M, Ramond E, Euphrasie D, Dupuis M, Tros F, Meyer J, Nemazanyy I, Chhuon C, Guerrera IC, Ferroni A, Sermet-Gaudelus I, Nassif X, Charbit A, Jamet A. Chronic Staphylococcus aureus lung infection correlates with proteogenomic and metabolic adaptations leading to an increased intracellular persistence. Clin Infect Dis. 2019 Feb 7. doi: 10.1093/cid/ciz106. [Epub ahead of print]
  • 2018

  • Masson A, Schneider-Futschik EK, Baatallah N, Nguyen-Khoa T, Girodon E, Hatton A, Flament T, Le Bourgeois M, Chedevergne F, Bailly C, Kyrilli S, Achimastos D, Hinzpeter A, Edelman A, Sermet-Gaudelus I. Predictive factors for lumacaftor/ivacaftor clinical response. J Cyst Fibros. 2019 May;18(3):368-374. doi: 10.1016/j.jcf.2018.12.011. Epub 2018 Dec 28.
  • Sermet-Gaudelus I, Clancy JP, Nichols DP, Nick JA, De Boeck K, Solomon GM, Mall MA, Bolognese J, Bouisset F, den Hollander W, Paquette-Lamontagne N, Tomkinson N, Henig N, Elborn JS, Rowe SM. Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis. J Cyst Fibros. 2019 Jul;18(4):536-542. doi: 10.1016/j.jcf.2018.10.015. Epub 2018 Nov 19.
  • Scudieri P, Musante I, Caci E, Venturini A, Morelli P, Walter C, Tosi D, Palleschi A, Martin-Vasallo P, Sermet-Gaudelus I, Planelles G, Crambert G, Galietta LJ. Increased expression of ATP12A proton pump in cystic fibrosis airways. JCI Insight. 2018 Oct 18;3(20). pii: 123616. doi: 10.1172/jci.insight.123616.
  • Pranke I, Hatton A, Masson A, Flament T, Le Bourgeois M, Chedevergne F, Bailly C, Urbach V, Hinzpeter A, Edelman A, Sermet-Gaudelus I. Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response? Am J Respir Crit Care Med. 2019 Jan 1;199(1):123-126. doi: 10.1164/rccm.201808-1436LE.
  • Veit G, Xu H, Dreano E, Avramescu RG, Bagdany M, Beitel LK, Roldan A, Hancock MA, Lay C, Li W, Morin K, Gao S, Mak PA, Ainscow E, Orth AP, McNamara P, Edelman A, Frenkiel S, Matouk E, Sermet-Gaudelus I, Barnes WG, Lukacs GL. Structure-guided combination therapy to potently improve the function of mutant CFTRs. Nat Med. 2018 Nov;24(11):1732-1742. doi: 10.1038/s41591-018-0200-x. Epub 2018 Oct 8.
  • Solomon GM, Bronsveld I, Hayes K, Wilschanski M, Melotti P, Rowe SM, Sermet-Gaudelus I. Standardized Measurement of Nasal Membrane Transepithelial Potential Difference (NPD). J Vis Exp. 2018 Sep 13;(139). doi: 10.3791/57006.
  • Sermet-Gaudelus I, Renouil M, Fajac A, Bidou L, Parbaille B, Pierrot S, Davy N, Bismuth E, Reinert P, Lenoir G, Lesure JF, Rousset JP, Edelman A. Correction to: In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study.BMC Med. 2018 Aug 25;16(1):159. doi: 10.1186/s12916-018-1138-z.
  • Pesce E, Sondo E, Ferrera L, Tomati V, Caci E, Scudieri P, Musante I, Renda M, Baatallah N, Servel N, Hinzpeter A, di Bernardo D, Pedemonte N, Galietta LJV. The Autophagy Inhibitor Spautin-1 Antagonizes Rescue of Mutant CFTR Through an Autophagy-Independent and USP13-Mediated Mechanism. Front Pharmacol. 2018 Dec 13;9:1464. doi: 10.3389/fphar.2018.01464. eCollection 2018.
  • Noel S, Sermet-Gaudelus I, Sheppard DN. N1303K: Leaving no stone unturned in the search for transformational therapeutics. J Cyst Fibros. 2018 Sep;17(5):555-557. doi: 10.1016/j.jcf.2018.07.009. Epub 2018 Aug 17. No abstract available.
  • Rollet-Cohen V, Bourderioux M, Lipecka J, Chhuon C, Jung VA, Mesbahi M, Nguyen-Khoa T, Guérin-Pfyffer S, Schmitt A, Edelman A, Sermet-Gaudelus I, Guerrera IC. Comparative proteomics of respiratory exosomes in cystic fibrosis, primary ciliary dyskinesia and asthma. J Proteomics. 2018 Aug 15;185:1-7. doi: 10.1016/j.jprot.2018.07.001. Epub 2018 Jul 3.
  • Castellani C, Duff AJA, Bell SC, Heijerman HGM, Munck A, Ratjen F, Sermet-Gaudelus I, Southern KW, Barben J, Flume PA, Hodková P, Kashirskaya N, Kirszenbaum MN, Madge S, Oxley H, Plant B, Schwarzenberg SJ, Smyth AR, Taccetti G, Wagner TOF, Wolfe SP, Drevinek P. ECFS best practice guidelines: the 2018 revision. J Cyst Fibros. 2018 Mar;17(2):153-178. doi: 10.1016/j.jcf.2018.02.006. Epub 2018 Mar 3. Review.
  • Pranke I, Bidou L, Martin N, Blanchet S, Hatton A, Karri S, Cornu D, Costes B, Chevalier B, Tondelier D, Girodon E, Coupet M, Edelman A, Fanen P, Namy O, Sermet-Gaudelus I, Hinzpeter A. Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons. ERJ Open Res. 2018 Feb 23;4(1). pii: 00080-2017. doi: 10.1183/23120541.00080-2017. eCollection 2018 Jan. Erratum in: ERJ Open Res. 2018 Jul 13;4(3)
  • Baatallah N, Bitam S, Martin N, Servel N, Costes B, Mekki C, Chevalier B, Pranke I, Simonin J, Girodon E, Hoffmann B, Mornon JP, Callebaut I, Sermet-Gaudelus I, Fanen P, Edelman A, Hinzpeter A. Cis variants identified in F508del complex alleles modulate CFTR channel rescue by small molecules. Hum Mutat. 2018 Apr;39(4):506-514. doi: 10.1002/humu.23389. Epub 2018 Jan 16.
  • Ringholz FC, Higgins G, Hatton A, Sassi A, Moukachar A, Fustero-Torre C, Hollenhorst M, Sermet-Gaudelus I, Harvey BJ, McNally P, Urbach V. Resolvin D1 regulates epithelial ion transport and inflammation in cystic fibrosis airways. Guidelines for the clinical management and follow-up of infants with inconclusive cystic fibrosis diagnosis through newborn screening. J Cyst Fibros. 2018 Sep;17(5):607-615. doi: 10.1016/j.jcf.2017.11.017. Epub 2017 Dec 8.
  • Masson A, Kirszenbaum M, Sermet-Gaudelus I. Pain is an underestimated symptom in cystic fibrosis. Curr Opin Pulm Med. 2017 Nov;23(6):570-573. doi: 10.1097/MCP.0000000000000427.
  • Saint-Criq V, Villeret B, Bastaert F, Kheir S, Hatton A, Cazes A, Xing Z, Sermet-Gaudelus I, Garcia-Verdugo I, Edelman A, Sallenave JM. Pseudomonas aeruginosa LasB protease impairs innate immunity in mice and humans by targeting a lung epithelial cystic fibrosis transmembrane regulator-IL-6-antimicrobial-repair pathway. Thorax. 2018 Jan;73(1):49-61.
  • 2017

  • Pranke IM, Hatton A, Simonin J, Jais JP, Le Pimpec‐Barthes F, Carsin A, Bonnette P, Fayon M, Stremler‐Le Bel N, Grenet D, Thumerel M, Mazenq J, Urbach V, Mesbahi M, Girodon‐Boulandet E, Hinzpeter A, Edelman A, Sermet‐Gaudelus I. Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. Sci Rep. 2017 Aug 7;7(1):7375. doi: 10.1038/s41598‐017‐07504‐1. PubMed PMID: 28785019; PubMed Central PMCID: PMC5547155.
  • Saint‐Criq V, Villeret B, Bastaert F, Kheir S, Hatton A, Cazes A, Xing Z, Sermet‐Gaudelus I, Garcia‐Verdugo I, Edelman A, Sallenave JM. Pseudomonas aeruginosa LasB protease impairs innate immunity in mice and humans by targeting a lung epithelial cystic fibrosis transmembrane
    regulator‐IL‐6‐antimicrobial‐repair pathway. Thorax. 2017 Aug 8. pii: thoraxjnl‐2017‐210298. doi: 10.1136/thoraxjnl‐2017‐210298. [Epub ahead of print] PubMed PMID: 28790180.
  • Higgins G, Fustero Torre C, Tyrrell J, McNally P, Harvey BJ, Urbach V. Lipoxin A4 prevents tight junction disruption and delays the colonization of cystic fibrosis bronchial epithelial cells by Pseudomonas aeruginosa. Am J Physiol Lung Cell Mol Physiol. 2016 Jun 1;310(11):L1053‐61. doi: 10.1152/ajplung.00368.2015. Epub 2016 Apr 15. PubMed PMID: 27084849.
  • Farrell PM, White TB, Howenstine MS, Munck A, Parad RB, Rosenfeld M, Sommerburg O, Accurso FJ, Davies JC, Rock MJ, Sanders DB, Wilschanski M, Sermet-Gaudelus I, Blau H, Gartner S, McColley SA. Diagnosis of Cystic Fibrosis in Screened Populations. J Pediatr. 2017 Feb;181S:S33-S44.e2. doi: 10.1016/j.jpeds.2016.09.065.
  • Farrell PM, White TB, Ren CL, Hempstead SE, Accurso F, Derichs N, Howenstine M, McColley SA, Rock M, Rosenfeld M, Sermet-Gaudelus I, Southern KW, Marshall BC, Sosnay PR. Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-S15.e1. doi: 10.1016/j.jpeds.2016.09.064.
  • Nevo N, Thomas L, Chhuon C, Andrzejewska Z, Lipecka J, Guillonneau F, Bailleux A, Edelman A, Antignac C, Guerrera IC. Impact of cystinosin glycosylation on protein stability by differential dynamic SILAC. Mol Cell Proteomics. 2017 Jan 12. pii: mcp.M116.063867. doi: 10.1074/mcp.M116.063867. [Epub ahead of print]
  • 2016

  • Premchandar A, Kupniewska A, Bonna A, Faure G, Fraczyk T, Roldan A, Hoffmann B, Faria da Cunha M, Herrmann H, Lukacs GL, Edelman A, Dadlez M. New insights into interactions between the nucleotide-binding domain of CFTR and keratin 8. Protein Sci. 2016 Nov 21. doi: 10.1002/pro.3086. [Epub ahead of print]
  • da Cunha MF, Simonin J, Sassi A, Freund R, Hatton A, Cottart CH, Elganfoud N, Zoubairi R, Dragu C, Jais JP, Hinzpeter A, Edelman A, Sermet-Gaudelus I. Analysis of nasal potential in murine cystic fibrosis models. Int J Biochem Cell Biol. 2016 Nov;80:87-97.
  • Sermet-Gaudelus I, Namy O. New Pharmacological Approaches to Treat Patients with Cystic Fibrosis with Nonsense Mutations. Am J Respir Crit Care Med. 2016 Nov 1;194(9):1042-1044.
  • Sermet-Gaudelus I, Delion M, Durieu I, Jacquot J, Hubert D. Bone demineralization is improved by ivacaftor in patients with cystic fibrosis carrying the p.Gly551Asp mutation. J Cyst Fibros. 2016 Nov;15(6):e67-e69.
  • Delion M, Braux J, Jourdain ML, Guillaume C, Bour C, Gangloff S, Pimpec-Barthes FL, Sermet-Gaudelus I, Jacquot J, Velard F. Overexpression of RANKL in osteoblasts: a possible mechanism of susceptibility to bone disease in cystic fibrosis. J Pathol. 2016 Sep;240(1):50-60.
  • Chhuon C, Pranke I, Borot F, Tondelier D, Lipecka J, Fritsch J, Chanson M, Edelman A, Ollero M, Guerrera IC. Changes in lipid raft proteome upon TNF-α stimulation of cystic fibrosis cells. J Proteomics. 2016 Aug 11;145:246-53.
  • Chhuon C, Pranke I, Borot F, Tondelier D, Lipecka J, Fritsch J, Chanson M, Edelman A, Ollero M, Guerrera IC. Dataset of differential lipid raft and global proteomes of SILAC-labeled cystic fibrosis cells upon TNF -α stimulation. Data Brief. 2016 Aug 20;9:51-6.
  • Faure G, Bakouh N, Lourdel S, Odolczyk N, Premchandar A, Servel N, Hatton A, Ostrowski MK, Xu H, Saul FA, Moquereau C, Bitam S, Pranke I, Planelles G, Teulon J, Herrmann H, Roldan A, Zielenkiewicz P, Dadlez M, Lukacs GL, Sermet-Gaudelus I, Ollero M, Corringer PJ, Edelman A. Rattlesnake Phospholipase A2 Increases CFTR-Chloride Channel Current and Corrects ∆F508CFTR Dysfunction: Impact in Cystic Fibrosis. J Mol Biol. 2016 Jul 17;428(14):2898-915.
  • Lipecka J, Chhuon C, Bourderioux M, Bessard MA, van Endert P, Edelman A, Guerrera IC. Sensitivity of mass spectrometry analysis depends on the shape of the filtration unit used for filter aided sample preparation (FASP). Proteomics. 2016 Jul;16(13):1852-7.
  • Le Henaff C, Faria Da Cunha M, Hatton A, Tondelier D, Marty C, Collet C, Zarka M, Geoffroy V, Zatloukal K, Laplantine E, Edelman A, Sermet-Gaudelus I, Marie PJ. Genetic deletion of keratin 8 corrects the altered bone formation and osteopenia in a mouse model of cystic fibrosis. Hum Mol Genet. 2016 Apr 1;25(7):1281-93.
  • Neau E, Delannoy J, Marion C, Cottart CH, Labellie C, Holowacz S, Butel MJ, Kapel N, Waligora-Dupriet AJ. Identification of three novel candidate probiotic strains with prophylactic properties in a murine model of cow's milk allergy. Appl Environ Microbiol. 2016 Jan 4. pii: AEM.03440-15. [Epub ahead of print]
  • Vidović D, Carlon MS, da Cunha MF, Dekkers JF, Hollenhorst MI, Bijvelds MJ, Ramalho AS, Van den Haute C, Ferrante M, Baekelandt V, Janssens HM, De Boeck K, Sermet-Gaudelus I, de Jonge HR, Gijsbers R, Beekman JM, Edelman A, Debyser Z. rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice. Am J Respir Crit Care Med. 2016 Feb 1;193(3):288-98. doi: 10.1164/rccm.201505-0914OC.
  • Bucher J, Boelle PY, Hubert D, Lebourgeois M, Stremler N, Durieu I, Bremont F, Deneuville E, Delaisi B, Corvol H, Bassinet L, Grenet D, Remus N, Vodoff MV, Boussaud V, Troussier F, Leruez-Ville M, Treluyer JM, Launay O, Sermet-Gaudelus I. Lessons from a French collaborative case-control study in cystic fibrosis patients during the 2009 A/H1N1 influenza pandemy. BMC Infect Dis. 2016 Feb 1;16:55.
  • 2015

  • Chatin B, Mével M, Devallière J, Dallet L, Haudebourg T, Peuziat P, Colombani T, Berchel M, Lambert O, Edelman A, Pitard B. Liposome-based Formulation for Intracellular Delivery of Functional Proteins. Mol Ther Nucleic Acids. 2015 Jun 23;4:e244. doi: 10.1038/mtna.2015.17.
  • Endale Ahanda ML, Bienvenu T, Sermet-Gaudelus I, Mazzolini L, Edelman A, Zoorob R, Davezac N. The hsa-miR-125a/hsa-let-7e/hsa-miR-99b cluster is potentially implicated in Cystic Fibrosis pathogenesis. J Cyst Fibros. 2015 Sep;14(5):571-9.
  • El Khatib N, Ferroni A, Le Bourgeois M, Chedevergne F, Clairicia M, Avril H, Guiso N, Sermet-Gaudelus I. Persistent Bordetella bronchiseptica infection in a child with cystic fibrosis: Relationship to bacterial phenotype. J Cyst Fibros. 2015 Sep;14(5):E13-5.
  • Bourderioux M, Nguyen-Khoa T, Chhuon C, Jeanson L, Tondelier D, Walczak M, Ollero M, Bekri S, Knebelmann B, Escudier E, Escudier B, Edelman A, Guerrera IC. A new workflow for proteomic analysis of urinary exosomes and assessment in cystinuria patients. J Proteome Res. 2015 Jan 2;14(1):567-77.
  • Endale Ahanda ML, Bienvenu T, Sermet-Gaudelus I, Mazzolini L, Edelman A, Zoorob R, Davezac N. The hsa-miR-125a/hsa-let-7e/hsa-miR-99b cluster is potentially implicated in Cystic Fibrosis pathogenesis. J Cyst Fibros. 2015 Mar 20. pii: S1569-1993(15)00053-3. doi: 10.1016/j.jcf.2015.02.011. [Epub ahead of print] PubMed PMID: 25800681.
  • Velard F, Delion M, Lemaire F, Tabary O, Guillaume C, Le Pimpec Barthès F, Touqui L, Gangloff S, Sermet-Gaudelus I, Jacquot J. Cystic fibrosis bone disease: is the CFTR corrector C18 an option for therapy? Eur Respir J. 2015 Mar;45(3):845-8.
  • Munck A, Kheniche A, Alberti C, Hubert D, Martine RG, Nove-Josserand R, Pin I, Bremont F, Chiron R, Couderc L, Dalphin ML, Darviot E, Delaisi B, Dominique S, Durieu I, Fanton A, Fayon M, Gérardin M, Giniès JL, Giraut C, Grenet D, Guillot M, Huet F, Le Bourgeois M, Murris-Epin M, Ramel S, Sardet A, Sermet-Gaudelus I, Varaigne F, Wanin S, Weiss L, Hurtaud MF. Central venous thrombosis and thrombophilia in cystic fibrosis: A prospective study. J Cyst Fibros. 2015 Jan;14(1):97-103.
  • Higgins G, Ringholz F, Buchanan P, McNally P, Urbach V. Physiological impact of abnormal lipoxin A₄ production on cystic fibrosis airway epithelium and therapeutic potential. Biomed Res Int. 2015;2015:781087. doi: 10.1155/2015/781087. Epub 2015 Mar 19. Review. PubMed PMID: 25866809; PubMed Central PMCID: PMC4383482.
  • Bitam S, Pranke I, Hollenhorst M, Servel N, Moquereau C, Tondelier D, Hatton A, Urbach V, Sermet-Gaudelus I, Hinzpeter A, Edelman A. An unexpected effect of TNF-α on F508del-CFTR maturation and function. Version 2. F1000Res. 2015 Jul 10 [revised 2015 Sep 2];4:218.
  • Verkman AS, Edelman A, Amaral M, Mall MA, Beekman JM, Meiners T, Galietta LJ, Bear CE. Finding new drugs to enhance anion secretion in cystic fibrosis: Toward suitable systems for better drug screening. Report on the pre-conference meeting to the 12th ECFS Basic Science Conference, Albufeira, 25-28 March 2015. J Cyst Fibros. 2015 Nov;14(6):700-5.
  • Premchandar A, Kupniewska A, Tarnowski K, Mücke N, Mauermann M, Kaus-Drobek M, Edelman A, Herrmann H, Dadlez M. Analysis of distinct molecular assembly complexes of keratin K8 and K18 by hydrogen-deuterium exchange. J Struct Biol. 2015 Dec;192(3):426-40.
  • 2014

  • Edelman A, Sallenave JM. Cystic fibrosis, a multi-systemic mucosal disease: 25 years after the discovery of CFTR. Int J Biochem Cell Biol. 2014 Jul;52:2-4.
  • Smyth AR, Bell SC, Bojcin S, Bryon M, Duff A, Flume P, Kashirskaya N, Munck A, Ratjen F, Schwarzenberg SJ, Sermet-Gaudelus I, Southern KW, Taccetti G, Ullrich G, Wolfe S; European Cystic Fibrosis Society. European Cystic Fibrosis Society Standards of Care: Best Practice guidelines. J Cyst Fibros. 2014 May;13 Suppl 1:S23-42.
  • Pranke IM, Sermet-Gaudelus I. Biosynthesis of cystic fibrosis transmembrane conductance regulator. Int J Biochem Cell Biol. 2014 Jul;52:26-38.
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    S.A.R. la Princesse Caroline de Hanovre qui, à travers la Fondation Princesse Grace, soutient déjà la recherche medicale et tout ce qui contribue à soulager les enfants malades en France et dans le monde, a accepté de s'engager à nos cotés pour que ce centre de medecine moleculaire, tourné entre autres vers les pathologies des enfants, prenne de vitesse les maladies et continue à relever les defis actuels.

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