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Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL. Sermet-Gaudelus I, et al. Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. doi: 10.1164/rccm.201001-0137OC. Epub 2010 Jul 9. Am J Respir Crit Care Med. 2010. PMID: 20622033 Clinical Trial.
An unexpected effect of TNF-α on F508del-CFTR maturation and function.
Bitam S, Pranke I, Hollenhorst M, Servel N, Moquereau C, Tondelier D, Hatton A, Urbach V, Sermet-Gaudelus I, Hinzpeter A, Edelman A. Bitam S, et al. F1000Res. 2015 Jul 10;4:218. doi: 10.12688/f1000research.6683.2. eCollection 2015. F1000Res. 2015. PMID: 26594334 Free PMC article.
Rattlesnake Phospholipase A2 Increases CFTR-Chloride Channel Current and Corrects ∆F508CFTR Dysfunction: Impact in Cystic Fibrosis.
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. Faure G, et al. J Mol Biol. 2016 Jul 17;428(14):2898-915. doi: 10.1016/j.jmb.2016.05.016. Epub 2016 May 27. J Mol Biol. 2016. PMID: 27241308
Changes of CFTR functional measurements and clinical improvements in cystic fibrosis patients with non p.Gly551Asp gating mutations treated with ivacaftor.
Mesbahi M, Shteinberg M, Wilschanski M, Hatton A, Nguyen-Khoa T, Friedman H, Cohen M, Escabasse V, Le Bourgeois M, Lucidi V, Sermet-Gaudelus I, Bassinet L, Livnat G. Mesbahi M, et al. J Cyst Fibros. 2017 Jan;16(1):45-48. doi: 10.1016/j.jcf.2016.08.006. Epub 2016 Sep 20. J Cyst Fibros. 2017. PMID: 27659740 Free article.
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators.
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. Pranke IM, et al. Sci Rep. 2017 Aug 7;7(1):7375. doi: 10.1038/s41598-017-07504-1. Sci Rep. 2017. PMID: 28785019 Free PMC article.
Pain is an underestimated symptom in cystic fibrosis.
Masson A, Kirszenbaum M, Sermet-Gaudelus I. Masson A, et al. Curr Opin Pulm Med. 2017 Nov;23(6):570-573. doi: 10.1097/MCP.0000000000000427. Curr Opin Pulm Med. 2017. PMID: 28806187 Review.
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