Phenylglycine and sulfonamide correctors of defective ΔF508 and G551D cystic fibrosis transmembrane conductance regulator chloride-channel gating

Molecular Pharmacology

Volumn 67, Issue 5, 2005, Pages 1797-1807

  Pedemonte, Nicoletta ^a,b   Sonawane, N D ^a   Taddei, Alessandro ^b   Hu, Jie ^a   Zegarra Moran, Olga ^b   Suen, Yat Fan ^c   Robins, Lori I ^c   Dicus, Christopher W ^c   Willenbring, Dan ^c   Nantz, Michael H ^c   Kurth, Mark J ^c   Galietta, Luis J V ^b   Verkman, Alan S ^a,d  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b G GASLINI INSTITUTE   (Italy)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 [(2 1H INDOL 3 YLACETYL)METHYLAMINO] N (4 ISOPROPYLPHENYL) 2 PHENYLACETAMIDE; 6 (ETHYLPHENYLSULFAMOYL) 4 OXO 1,4 DIHYDROQUINOLINE 3 CARBOXYLIC ACID CYCLOHEPTYLAMIDE; ACETANILIDE DERIVATIVE; ASPARTIC ACID; CARBOXYLIC ACID DERIVATIVE; CHLORIDE CHANNEL; FLUORESCENT DYE; FORSKOLIN; GENISTEIN; GLYCINE; HALIDE; IODIDE; PHENYLGLYCINE; SODIUM CHLORIDE; SULFONAMIDE; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; YELLOW FLUORESCENT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CHANNEL GATING; CONTROLLED STUDY; CYSTIC FIBROSIS; DRUG MECHANISM; DRUG METABOLISM; ELECTROPHYSIOLOGY; EPITHELIUM CELL; GENE MUTATION; HUMAN; HUMAN CELL; LIVER METABOLISM; LIVER MICROSOME; MALE; NONHUMAN; PATCH CLAMP; PATHOGENESIS; PRIORITY JOURNAL; RAT; STRUCTURE ACTIVITY RELATION;

ANIMALS; CHLORIDE CHANNELS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOSE-RESPONSE RELATIONSHIP, DRUG; GLYCINE; HUMANS; ION CHANNEL GATING; RATS; RATS, INBRED F344; SULFONAMIDES;

EID: 20944442087     PISSN: 0026895X     EISSN: None     Source Type: Journal    
DOI: 10.1124/mol.105.010959     Document Type: Article

References (34)

1. Blus K (1999) Synthesis and properties of acid dyes derived from 7-amino-1-hydroxynaphthalene-3-sulfonic acid. Dyes Pigments 41:149-157.
2. Bobadilla J, Macek M, Fine JP, and Farrell PM (2002) Cystic fibrosis: a worldwide analysis of CFTR mutations-correlation with incidence data and application to screening. Hum Mutat 19:575-606.
3. Cashman SM, Patino A, Delgado MG, Byrne L, Denham B, and Dearce M (1995) The Irish cystic fibrosis database. J Med Genet 32:972-975.
4. Dalemans W, Barbry P, Champigny G, Jallat S, Dott K, Dreyer D, Crystal RG, Pavirani A, Lecocq JP, and Lazdunski M (1991) Altered chloride ion channel kinetics associated with the ΔF508 cystic fibrosis mutation. Nature (Lond) 354:526-528.
5. Denning GM, Anderson MP, Amara JF, Marshall J, Smith AE, and Welsh MJ (1992) Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature (Lond) 358:761-764.
6. Derand R, Bulteau-Pignoux L, and Becq F (2002) The cystic fibrosis mutation G551D alters the non-Michaelis-Menten behavior of the cystic fibrosis transmembrane conductance regulator (CFTR) channel and abolishes the inhibitory genistein binding site. J Biol Chem 277:35999-36004.
7. Drumm ML, Wilkinson DJ, Smit LS, Worrell RT, Strong TV, Frizzell RA, Dawson DC, and Collins FS (1991) Chloride conductance expressed by ΔF508 and other mutant CFTRs in Xenopus oocytes. Science (Wash DC) 254:1797-1799.
8. Galietta LJV, Haggle PM, and Verkman AS (2001a) Green fluorescent protein-based halide indicators with improved chloride and iodide affinities. FEBS Lett 499:220-224.
9. Galietta LJV, Jayaraman S, and Verkman AS (2001c) Cell-based assay for high-throughput quantitative screening of CFTR chloride transport agonists. Am J Physiol 281:C1734-C1742.
10. Galietta LJV, Musante L, Romio L, Caruso U, Fantasia A, Gazzolo A, Romano L, Sacco O, Rossi GA, Varesio L, et al. (1998) An electrogenic amino acid transporter in the apical membrane of cultured human bronchial epithelial cells. Am J Physiol 275:L917-L923.
11. Galietta LJV, Springsteel MF, Eda M, Niedzinski EJ, By K, Haddadin MJ, Kurth MJ, Nantz MH, and Verkman AS (2001b) Novel CFTR chloride channel activators identified by screening of combinatorial libraries based on flavone and benzoquinolizinium lead compounds. J Biol Chem 276:19723-19728.
12. Gregory RJ, Rich DP, Cheng SH, Souza DW, Paul S, Manavalan P, Anderson MP, Welsh MJ, and Smith AE (1991) Maturation and function of cystic fibrosis transmembrane conductance regulator variants bearing mutations in putative nucleotide-biuding domains 1 and 2. Mol Cell Biol 11:3886-3893.
13. Hamosh A, King TM, Rosenstein BJ, Corey M, Levison H, Durie P, Tsui LC, McIntosh I, Keston M, Brock DJH, et al. (1992) Cystic fibrosis patients bearing the common missense mutation Gly to Asp at codon 551 and the ΔF508 mutation are clinically indistinguishable from ΔF508 homozygotes, except for a decreased risk of meconium ileus. Am J Hum Genet 51:245-250.
14. Haws CM, Nepomuceno IB, Krouse ME, Wakelee H, Law T, Xia Y, Nguyen H, and Wine JJ (1996) ΔF508-CFTR channels: kinetics, activation by forskolin and potentiation by xanthines. Am J Physiol 270:C1544-C1555.
15. Hwang T-C, Wang F, Yang I, and Reenstra WW (1997) Genistein potentiates wild-type and ΔF508-CFTR channel activity. Am J Physiol 273:C988-C998.
16. Hyde SC, Emsley P, Hartshorn MJ, Mimmack MM, Gileadi U, Pearce SR, Gallagher MP, Gill DR, Hubbard RE, and Higgins CF (1990) Structural model of ATP-binding proteins associated with cystic fibrosis, multidrug resistance and bacterial transport. Nature (Lond) 346:362-365.
17. Illek B, Zhang L, Lewis NC, Moss RB, Dong JY, and Fischer H (1999) Defective function of the cystic fibrosis-causing mutation missense mutation G551D is recovered by genistein. Am J Physiol 277:C833-C839.
18. Jensen TJ, Loo MA, Find S, Williams DB, Goldberg AL, and Riordan JR (1995) Multiple proteolytic systems, including the proteasome, contribute to CFTR processing. Cell 83:129-135.
19. Kopito RR (1999) Biosynthesis and degradation of CFTR. Physiol Rev 79:S167-S173.
20. Logan J, Hiestand D, Daram P, Huang Z, Muccio DD, Hartman J, Haley B, Cook WJ, and Sorscher EJ (1994) Cystic fibrosis transmembrane conductance regulator mutations that disrupt nucleotide binding. J Clin Investig 94:228-236.
21. Ma T, Thiagarajah JR, Yang H, Sonawane ND, Folli C, Galietta LJ, and Verkman AS (2002a) Thiazolidinone CFTR inhibitor identified by high-throughput screening blocks cholera-toxin induced intestinal fluid secretion. J Clin Investig 110:1651-1658.
22. Ma T, Vetrivel L, Yang H, Pedemonte N, Zegarra-Moran O, Galietta LJV, and Verkman AS (2002b) High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening. J Biol Chem 277:37235-37241.
23. Penque D, Mendes F, Beck S, Farinha C, Pacheco P, Nogueira P, Lavinha J, Malho R, and Amaral MD (2000) Cystic fibrosis F508del patients have apically localized CFTR in a reduced number of airway cells. Lab Investig 80:857-868.
24. Pilewski JM and Frizzell RA (1999) Role of CFTR in airway disease. Physiol Rev 79:S215-S255.
25. Rasola A, Galietta LJV, Gruenert DC, and Romeo G (1994) Volume-sensitive chloride currents in four epithelial cell lines are not directly correlated to the expression of the MDR-1 gene. J Biol Chem 269:1432-1436.
26. Sermet-Gaudelus I, Vallee B, Urbin I, Torossi T, Marianovski R, Fajac A, Feuillet MN, Bresson JL, Lenoir G, et al. (2002) Normal function of the cystic fibrosis conductance regulator protein can be associated with homozygous ΔF508 mutation. Pediatr Res 52:628-635.
27. Sheppard DN and Welsh MJ (1999) Structure and function of the CFTR chloride channel. Physiol Rev 79:S23-S45.
28. Sonawane ND, Muanprasat C, Nagatani R, Song Y, and Verkman AS (2004) In vivo pharmacology and antidiarrheal efficacy of a thiazolidinone CFTR inhibitor in rodents. J Pharm Sci 94:134-143.
29. Taddei A, Folli C, Zegarra-Moran O, Fanen P, Verkman AS, and Galietta LJV (2004) Altered channel gating mechanism for CFTR inhibition by a high-affinity thiazolidinone blocker. FEBS Lett 558:52-56.
30. Vankeerberghen A, Wei L, Teng H, Jaspers M, Cassiman JJ, Nilius B, and Cuppens H (1998) Characterization of mutations located in exon 18 of the CFTR gene. FEBS Lett 437:1-4.
31. Ward CL, Omura S, and Kopito RR (1995) Degradation of CFTR by the ubiquitin-proteasome pathway. Cell 83:121-127.
32. Yang H, Shelat AA, Guy RK, Gopinath VS, Ma T, Du K, Lukacs GL, Taddei A, Folli C, Pedemonte N, et al. (2003) Nanomolar affinity small molecule correctors of defective ΔF508-CFTR chloride channel gating. J Biol Chem 278:35079-35085.
33. Zegarra-Moran O, Romio L, Folli C, Caci E, Becq F, Vierfond JM, Mettey Y, Cabrini G, Fanen P, and Galietta LJV (2002) Correction of G551D-CFTR transport defect in epithelial monolayers by genisteiu but not by CPX or MPB-07. Br J Pharmacol 137:504-512.
34. Zhang JH, Chung TD, and Oldenburg KR (1999) A simple statistical parameter for use in valuation and validation of high throughput screening assays. J Biomol Screen 4:67-73.