Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the α-glucosidase inhibitor miglustat

FEBS Letters

Volumn 580, Issue 8, 2006, Pages 2081-2086

  Norez, Caroline ^a   Noel, Sabrina ^a   Wilke, Martina ^b   Bijvelds, Marcel ^b   Jorna, Huub ^b   Melin, Patricia ^a   Dejonge, Hugo ^b   Becq, Frederic ^a  

^a UNIVERSITÉ DE POITIERS   (France)

^b ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

Calnexin; Castanospermine; Chloride channels; Cystic fibrosis transmembrane conductance regulator; delF508 mice; Miglustat

Indexed keywords

ALPHA GLUCOSIDASE INHIBITOR; CALNEXIN; CASTANOSPERMINE; CYCLIC AMP; MIGLUSTAT; ORPHAN DRUG; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ANIMAL CELL; ANIMAL TISSUE; ARTICLE; CELL MATURATION; CELLULAR DISTRIBUTION; CHLORIDE CURRENT; CONTROLLED STUDY; CYSTIC FIBROSIS; DEGLYCOSYLATION; DRUG INHIBITION; ENZYME INHIBITION; GAUCHER DISEASE; HUMAN; HUMAN CELL; ILEUM MUCOSA; IMMUNOHISTOCHEMISTRY; INTESTINE CRYPT; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN DEGRADATION; PROTEIN INTERACTION;

1-DEOXYNOJIRIMYCIN; ALPHA-GLUCOSIDASES; ANIMALS; CALNEXIN; CERCOPITHECUS AETHIOPS; COS CELLS; CRICETINAE; CRICETULUS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EPITHELIAL CELLS; HUMANS; INTESTINAL MUCOSA; MICE; MICE, INBRED CFTR; MICE, KNOCKOUT; PROTEIN BINDING; PROTEIN PROCESSING, POST-TRANSLATIONAL; PROTEIN TRANSPORT;

ANIMALIA;

EID: 33645211759     PISSN: 00145793     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.febslet.2006.03.010     Document Type: Article

References (35)

1. J.R. Riordan Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA Sci. 245 1989 1066 1073
2. A. Vankeerberghen, H. Cuppens, and J.J. Cassiman The cystic fibrosis transmembrane conductance regulator: an intriguing protein with pleiotropic functions J. Cyst. Fibros. 1 2002 13 29
3. J.A. Tabcharani, X.B. Chang, J.R. Riordan, and J.W. Hanrahan Phosphorylation-regulated Cl-channel in CHO cells stably expressing the cystic fibrosis gene Nature 352 1991 628 631
4. N. Kartner, O. Augustinas, T.J. Jensen, A.L. Naismith, and J.R. Riordan Mislocalization of delta F508 CFTR in cystic fibrosis sweat gland Nat. Genet. 1 1992 321 327
5. S. Pind, J.R. Riordan, and D.B. Williams Participation of the endoplasmic reticulum chaperone calnexin (p88, IP90) in the biogenesis of the cystic fibrosis transmembrane conductance regulator J. Biol. Chem. 269 1994 12784 12788
6. S.H. Cheng, R.J. Gregory, J. Marshall, S. Paul, D.W. Souza, G.A. White, C.R. O'Riordan, and A.E. Smith Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis Cell 63 1990 827 834
7. L. Ellgaard, and A. Helenius Quality control in the endoplasmic reticulum Nat. Rev. Mol. Cell Biol. 4 2003 181 191
8. A. Helenius, and M. Aebi Roles of N-linked glycans in the endoplasmic reticulum Annu. Rev. Biochem. 73 2004 1019 1049
9. F.E. Ware, A. Vassilakos, P.A. Peterson, M.R. Jackson, M.A. Lehrman, and D.B. Williams The molecular chaperone calnexin binds Glc1Man9GlcNAc2 oligosaccharide as an initial step in recognizing unfolded glycoproteins J. Biol. Chem. 270 1995 4697 4704
10. A. Zapun, S.M. Petrescu, P.M. Rudd, R.A. Dwek, D.Y. Thomas, and J.J. Bergeron Conformation-independent binding of monoglucosylated ribonuclease B to calnexin Cell 88 1997 29 38
11. C. Norez, F. Antigny, F. Becq and C. Vandebrouck (2006) Maintaining low Ca2+ level in the endoplasmic reticulum restores abnormal endogenous F508del-CFTR trafficking in airway epithelial cells. Traffic 7, in press.
12. M.E. Egan Curcumin, a major constituent of turmeric, corrects cystic fibrosis defects Science 304 2004 600 602
13. T. Okiyoneda Delta F508 CFTR pool in the endoplasmic reticulum is increased by calnexin overexpression Mol. Biol. Cell 15 2004 563 574
14. T. Okiyoneda Calnexin delta 185-520 partially reverses the misprocessing of the Delta F508 cystic fibrosis transmembrane conductance regulator FEBS Lett. 526 2002 87 92
15. T.M. Cox The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement J. Inherit. Metab. Dis. 26 2003 513 526
16. R.A. Dwek, T.D. Butters, F.M. Platt, and N. Zitzmann Targeting glycosylation as a therapeutic approach Nat. Rev. Drug Discov. 1 2002 65 75
17. D.M. Jefferson Expression of normal and cystic fibrosis phenotypes by continuous airway epithelial cell lines Am. J. Physiol. 259 1990 L496 L505
18. W. Kammouni, B. Moreau, F. Becq, A. Saleh, A. Pavirani, C. Figarella, and M.D. Merten A cystic fibrosis tracheal gland cell line, CF-KM4. Correction by adenovirus-mediated CFTR gene transfer Am. J. Respir. Cell. Mol. Biol. 20 1999 684 691
19. O. Eidelman, C. Guay-Broder, P.J. van Galen, K.A. Jacobson, C. Fox, R.J. Turner, Z.I. Cabantchik, and H.B. Pollard A1 adenosine-receptor antagonists activate chloride efflux from cystic fibrosis cells Proc. Natl. Acad. Sci. USA 89 1992 5562 5566
20. P. Melin, V. Thoreau, C. Norez, F. Bilan, A. Kitzis, and F. Becq The cystic fibrosis mutation G1349D within the signature motif LSHGH of NBD2 abolishes the activation of CFTR chloride channels by genistein Biochem. Pharmacol. 67 2004 2187 2196
21. F. Becq, B. Verrier, X.B. Chang, J.R. Riordan, and J.W. Hanrahan cAMP- and Ca2+-independent activation of cystic fibrosis transmembrane conductance regulator channels by phenylimidazothiazole drugs J. Biol. Chem. 271 1996 16171 16179
22. P.J. French A delta F508 mutation in mouse cystic fibrosis transmembrane conductance regulator results in a temperature-sensitive processing defect in vivo J. Clin. Invest. 98 1996 1304 1312
23. B.J. Scholte, D.J. Davidson, M. Wilke, and H.R. De Jonge Animal models of cystic fibrosis J. Cyst. Fibros. 3 Suppl. 2 2004 183 190
24. H.R. De Jonge, M. Ballmann, H. Veeze, I. Bronsveld, F. Stanke, B. Tummler, and M. Sinaasappel Ex vivo CF diagnosis by intestinal current measurements (ICM) in small aperture, circulating Ussing chambers J. Cyst. Fibros. 3 Suppl. 2 2004 159 163
25. P. Dickinson The severe G480C cystic fibrosis mutation, when replicated in the mouse, demonstrates mistrafficking, normal survival and organ-specific bioelectrics Hum. Mol. Genet. 11 2002 243 251
26. G.M. Denning, M.P. Anderson, J.F. Amara, J. Marshall, A.E. Smith, and M.J. Welsh Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive Nature 358 1992 761 764
27. T. Ma, J.R. Thiagarajah, H. Yang, N.D. Sonawane, C. Folli, L.J. Galietta, and A.S. Verkman Thiazolidinone CFTR inhibitor identified by high-throughput screening blocks cholera toxin-induced intestinal fluid secretion J. Clin. Invest. 110 2002 1651 1658
28. H. De Jonge, M. Wilke, A. Bot, H. Jorna, N. Charizopoulou, and B. Tummler What can we learn from mouse models? Pediatr. Pulmonol. Suppl. 27 2004 93 94
29. B. Burke, K. Matlin, E. Bause, G. Legler, N. Peyrieras, and H. Ploegh Inhibition of N-linked oligosaccharide trimming does not interfere with surface expression of certain integral membrane proteins Embo J. 3 1984 551 556
30. C. Hammond, I. Braakman, and A. Helenius Role of N-linked oligosaccharide recognition, glucose trimming, and calnexin in glycoprotein folding and quality control Proc. Natl. Acad. Sci. USA 91 1994 913 917
31. M. Aridor, and L.A. Hannan Traffic jams II: an update of diseases of intracellular transport Traffic 3 2002 781 790
32. C.T. Esapa, R.A. McIlhinney, and D.J. Blake Fukutin-related protein mutations that cause congenital muscular dystrophy result in ER-retention of the mutant protein in cultured cells Hum. Mol. Genet. 14 2005 295 305
33. G. Baroudi, V. Pouliot, I. Denjoy, P. Guicheney, A. Shrier, and M. Chahine Novel mechanism for Brugada syndrome: defective surface localization of an SCN5A mutant (R1432G) Circ. Res. 88 2001 E78 E83
34. J.A. Burrows, L.K. Willis, and D.H. Perlmutter Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: a potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency Proc. Natl. Acad. Sci. USA 97 2000 1796 1801
35. J.Q. Fan, S. Ishii, N. Asano, and Y. Suzuki Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor Nat. Med. 5 1999 112 115