SEARCH

SEARCH BY CITATION

References

  • Becq F, Mall MA, Sheppard DN, Conese M, Zegarra-Moran O (2011) Pharmacological therapy for cystic fibrosis: from bench to bedside. J Cyst Fibros 10 (Suppl 2), S129-S145
  • Berdiev BK, Qadri YJ, Benos DJ (2009) Assessment of the CFTR and ENaC association. Mol Biosyst 5: 123-127
  • Carlile GW, Robert R, Zhang D, Teske KA, Luo Y, Hanrahan JW, Thomas DY (2007) Correctors of protein trafficking defects identified by a novel high-throughput screening assay. Chembiochem 8: 1012-1020
  • Cheng SH, Rich DP, Marshall J, Gregory RJ, Welsh MJ, Smith AE (1991) Phosphorylation of the R domain by cAMP-dependent protein kinase regulates the CFTR chloride channel. Cell 66: 1027-1036
  • Colas J, Faure G, Saussereau E, Trudel S, Rabeh WM, Bitam S, Guerrera IC, Fritsch J, Sermet-Gaudelus I, Davezac N, et al (2012) Disruption of cytokeratin-8 interaction with F508del-CFTR corrects its functional defect. Hum Mol Genet 21: 623-634
  • Dalemans W, Barbry P, Champigny G, Jallat S, Dott K, Dreyer D, Crystal RG, Pavirani A, Lecocq JP, Lazdunski M (1991) Altered chloride ion channel kinetics associated with the delta F508 cystic fibrosis mutation. Nature 354: 526-528
  • Denning GM, Anderson MP, Amara JF, Marshall J, Smith AE, Welsh MJ (1992) Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature 358: 761-764
  • Dormer RL, Harris CM, Clark Z, Pereira MM, Doull IJ, Norez C, Becq F, McPherson MA (2005) Sildenafil (Viagra) corrects DeltaF508-CFTR location in nasal epithelial cells from patients with cystic fibrosis. Thorax 60: 55-59
  • Duan Y, Sun Y, Zhang F, Zhang WK, Wang D, Wang Y, Cao X, Hu W, Xie C, Cuppoletti J, et al (2012) Keratin K18 increases cystic fibrosis transmembrane conductance regulator (CFTR) surface expression by binding to its C-terminal hydrophobic patch. J Biol Chem 287: 40547-40559
  • Englebienne P, Moitessier N (2009) Docking ligands into flexible and solvated macromolecules. 4. Are popular scoring functions accurate for this class of proteins? J Chem Inf Model 49: 1568-1580
  • Hamdaoui N, Baudoin-Legros M, Kelly M, Aissat A, Moriceau S, Vieu DL, Colas J, Fritsch J, Edelman A, Planelles G (2011) Resveratrol rescues cAMP-dependent anionic transport in the cystic fibrosis pancreatic cell line CFPAC1. Br J Pharmacol 163: 876-886
  • He L, Kota P, Aleksandrov AA, Cui L, Jensen T, Dokholyan NV, Riordan JR (2013) Correctors of DeltaF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein. FASEB J 27: 536-545
  • Hinzpeter A, Lipecka J, Brouillard F, Baudoin-Legros M, Dadlez M, Edelman A, Fritsch J (2006) Association between Hsp90 and the ClC-2 chloride channel upregulates channel function. Am J Physiol Cell Physiol 290: C45-C56
  • Hutt DM, Herman D, Rodrigues AP, Noel S, Pilewski JM, Matteson J, Hoch B, Kellner W, Kelly JW, Schmidt A, et al (2010) Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis. Nat Chem Biol 6: 25-33
  • Jensen TJ, Loo MA, Pind S, Williams DB, Goldberg AL, Riordan JR (1995) Multiple proteolytic systems, including the proteasome, contribute to CFTR processing. Cell 83: 129-135
  • Kalid O, Mense M, Fischman S, Shitrit A, Bihler H, Ben-Zeev E, Schutz N, Pedemonte N, Thomas PJ, Bridges RJ, et al (2010) Small molecule correctors of F508del-CFTR discovered by structure-based virtual screening. J Comput Aided Mol Des 24: 971-991
  • Kim Chiaw P, Wellhauser L, Huan LJ, Ramjeesingh M, Bear CE (2010) A chemical corrector modifies the channel function of F508del-CFTR. Mol Pharmacol 78: 411-418
  • Ko SB, Zeng W, Dorwart MR, Luo X, Kim KH, Millen L, Goto H, Naruse S, Soyombo A, Thomas PJ, et al (2004) Gating of CFTR by the STAS domain of SLC26 transporters. Nat Cell Biol 6: 343-350
  • Kreyszig E (1993) Advanced Engineering Mathematics, Wiley, New York, USA
  • Lazar P, Schwartz D (1967) Éléments de Probabilités et Statistique à L'usage Des Étudiants en Biologie Humaine et Générale, Éditions médicales Flammarion, Paris, France
  • Leach AR, Shoichet BK, Peishoff CE (2006) Prediction of protein-ligand interactions. Docking and scoring: successes and gaps. J Med Chem 49: 5851-5855
  • Lipecka J, Norez C, Bensalem N, Baudouin-Legros M, Planelles G, Becq F, Edelman A, Davezac N (2006) Rescue of DeltaF508-CFTR (cystic fibrosis transmembrane conductance regulator) by curcumin: involvement of the keratin 18 network. J Pharmacol Exp Ther 317: 500-505
  • Loo TW, Bartlett MC, Clarke DM (2005) Rescue of DeltaF508 and other misprocessed CFTR mutants by a novel quinazoline compound. Mol Pharm 2: 407-413
  • Marivingt-Mounir C, Norez C, Derand R, Bulteau-Pignoux L, Nguyen-Huy D, Viossat B, Morgant G, Becq F, Vierfond JM, Mettey Y (2004) Synthesis, SAR, crystal structure, and biological evaluation of benzoquinoliziniums as activators of wild-type and mutant cystic fibrosis transmembrane conductance regulator channels. J Med Chem 47: 962-972
  • McKone EF, Emerson SS, Edwards KL, Aitken ML (2003) Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study. Lancet 361: 1671-1676
  • Mendoza JL, Schmidt A, Li Q, Nuvaga E, Barrett T, Bridges RJ, Feranchak AP, Brautigam CA, Thomas PJ (2012) Requirements for efficient correction of DeltaF508 CFTR revealed by analyses of evolved sequences. Cell 148: 164-174
  • Moran O, Galietta LJ, Zegarra-Moran O (2005) Binding site of activators of the cystic fibrosis transmembrane conductance regulator in the nucleotide binding domains. Cell Mol Life Sci 62: 446-460
  • Mornon JP, Lehn P, Callebaut I (2008) Atomic model of human cystic fibrosis transmembrane conductance regulator: membrane-spanning domains and coupling interfaces. Cell Mol Life Sci 65: 2594-2612
  • Moustakas DT, Lang PT, Pegg S, Pettersen E, Kuntz ID, Brooijmans N, Rizzo RC (2006) Development and validation of a modular, extensible docking program: DOCK 5. J Comput Aided Mol Des 20: 601-619
  • Nayal M, Honig B (2006) On the nature of cavities on protein surfaces: application to the identification of drug-binding sites. Proteins 63: 892-906
  • Norez C, Noel S, Wilke M, Bijvelds M, Jorna H, Melin P, DeJonge H, Becq F (2006) Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS Lett 580: 2081-2086
  • Ollero M, Brouillard F, Edelman A (2006) Cystic fibrosis enters the proteomics scene: new answers to old questions. Proteomics 6: 4084-4099
  • Pedemonte N, Diena T, Caci E, Nieddu E, Mazzei M, Ravazzolo R, Zegarra-Moran O, Galietta LJ (2005a) Antihypertensive 1,4-dihydropyridines as correctors of the cystic fibrosis transmembrane conductance regulator channel gating defect caused by cystic fibrosis mutations. Mol Pharmacol 68: 1736-1746
  • Pedemonte N, Lukacs GL, Du K, Caci E, Zegarra-Moran O, Galietta LJ, Verkman AS (2005b) Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening. J Clin Invest 115: 2564-2571
  • Rabeh WM, Bossard F, Xu H, Okiyoneda T, Bagdany M, Mulvihill CM, Du K, di Bernardo S, Liu Y, Konermann L, et al (2012) Correction of both NBD1 energetics and domain interface is required to restore DeltaF508 CFTR folding and function. Cell 148: 150-163
  • Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, et al (2011) A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 365: 1663-1672
  • Riordan JR (2008) CFTR function and prospects for therapy. Annu Rev Biochem 77: 701-726
  • Riordan JR, Rommens JM, Kerem B, Alon N, Rozmahel R, Grzelczak Z, Zielenski J, Lok S, Plavsic N, Chou JL, et al (1989) Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 245: 1066-1073
  • Robert R, Carlile GW, Liao J, Balghi H, Lesimple P, Liu N, Kus B, Rotin D, Wilke M, de Jonge HR, et al (2010) Correction of the Delta phe508 cystic fibrosis transmembrane conductance regulator trafficking defect by the bioavailable compound glafenine. Mol Pharmacol 77: 922-930
  • Robert R, Carlile GW, Pavel C, Liu N, Anjos SM, Liao J, Luo Y, Zhang D, Thomas DY, Hanrahan JW (2008) Structural analog of sildenafil identified as a novel corrector of the F508del-CFTR trafficking defect. Mol Pharmacol 73: 478-489
  • Rode B, Dirami T, Bakouh N, Rizk-Rabin M, Norez C, Lhuillier P, Lores P, Jollivet M, Melin P, Zvetkova I, et al (2012) The testis anion transporter TAT1 (SLC26A8) physically and functionally interacts with the cystic fibrosis transmembrane conductance regulator channel: a potential role during sperm capacitation. Hum Mol Genet 21: 1287-1298
  • Sampson HM, Robert R, Liao J, Matthes E, Carlile GW, Hanrahan JW, Thomas DY (2011) Identification of a NBD1-binding pharmacological chaperone that corrects the trafficking defect of F508del-CFTR. Chem Biol 18: 231-242
  • Sermet-Gaudelus I, Girodon E, Roussel D, Deneuville E, Bui S, Huet F, Guillot M, Aboutaam R, Renouil M, Munck A, et al (2010) Measurement of nasal potential difference in young children with an equivocal sweat test following newborn screening for cystic fibrosis. Thorax 65: 539-544
  • Sondo E, Tomati V, Caci E, Esposito AI, Pfeffer U, Pedemonte N, Galietta LJ (2011) Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling. Am J Physiol Cell Physiol 301: C872-C885
  • Tanguy G, Drevillon L, Arous N, Hasnain A, Hinzpeter A, Fritsch J, Goossens M, Fanen P (2008) CSN5 binds to misfolded CFTR and promotes its degradation. Biochim Biophys Acta 1783: 1189-1199
  • Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, et al (2011) Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 108: 18843-18848
  • Van Goor F, Straley KS, Cao D, Gonzalez J, Hadida S, Hazlewood A, Joubran J, Knapp T, Makings LR, Miller M, et al (2006) Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Lung Cell Mol Physiol 290: L1117-L1130
  • Wang Y, Loo TW, Bartlett MC, Clarke DM (2007) Modulating the folding of P-glycoprotein and cystic fibrosis transmembrane conductance regulator truncation mutants with pharmacological chaperones. Mol Pharmacol 71: 751-758
  • Ward CL, Omura S, Kopito RR (1995) Degradation of CFTR by the ubiquitin-proteasome pathway. Cell 83: 121-127
  • Wellhauser L, Kim Chiaw P, Pasyk S, Li C, Ramjeesingh M, Bear CE (2009) A small-molecule modulator interacts directly with deltaPhe508-CFTR to modify its ATPase activity and conformational stability. Mol Pharmacol 75: 1430-1438
  • Wieczorek G, Zielenkiewicz P (2008) DeltaF508 mutation increases conformational flexibility of CFTR protein. J Cyst Fibros 7: 295-300
  • Younger JM, Chen L, Ren HY, Rosser MF, Turnbull EL, Fan CY, Patterson C, Cyr DM (2006) Sequential quality-control checkpoints triage misfolded cystic fibrosis transmembrane conductance regulator. Cell 126: 571-582
  • Yu H, Burton B, Huang CJ, Worley J, Cao D, Johnson JP Jr, Urrutia A, Joubran J, Seepersaud S, Sussky K, et al (2012) Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 11: 237-245
  • Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, Skiadopoulos MH, Dang YL, Vogel LN, McKay T, Mengos A, et al (2009) CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium. PLoS Biol 7: e1000155