Specific inhibition of epithelial Na+ channels by antisense oligonucleotides for the treatment of Na+ hyperabsorption in cystic fibrosis

Journal of Gene Medicine

Volumn 11, Issue 9, 2009, Pages 813-823

  Sobczakl, Katja ^a   Segal, Andrei ^b   Bangel Ruland, Nadine ^a   Semmler, Judith ^a   Van Driessche, Willy ^b   Lindemann, Hermann ^c   Heermann, Ralf ^d   Weber, Wolf Michael ^a  

^a UNIVERSITY OF MÜNSTER   (Germany)

^b UNIVERSITY OF LEUVEN   (Belgium)

^c UNIVERSITY HOSPITAL GIESSEN   (Germany)

^d HNO Zentrum Muensterland   (Germany)

Author keywords

Antisense oligonucleotides; Cystic fibrosis; Epithelial Na+ channel; Na+ hyperabsorption

Indexed keywords

AMILORIDE; ANTISENSE OLIGONUCLEOTIDE; EPITHELIAL SODIUM CHANNEL;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; CYSTIC FIBROSIS; FEMALE; HUMAN; HUMAN CELL; NONHUMAN; NOSE MUCOSA; OOCYTE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SODIUM ABSORPTION; SODIUM CONDUCTANCE; SODIUM CURRENT; WESTERN BLOTTING; XENOPUS LAEVIS;

ANIMALS; BASE SEQUENCE; CYSTIC FIBROSIS; DNA PRIMERS; EPITHELIAL SODIUM CHANNEL; HUMANS; MICROELECTRODES; MICROSCOPY, FLUORESCENCE; NASAL MUCOSA; OLIGONUCLEOTIDES, ANTISENSE; RNA, MESSENGER; SODIUM; XENOPUS LAEVIS;

XENOPUS LAEVIS;

EID: 70349484831     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1363     Document Type: Article

References (52)

1. Alvarez de la Rosa D, Canessa CM, Fyfe GK, Zhang P. Structure and regulation of amiloride-sensitive sodium channels. Annu Rev Physiol 2000; 62: 573-594.
2. Kellenberger S, Schild L. Epithelial sodium channel/degenerin family of ion channels: a variety of functions for a shared structure. Phys Rev 2002; 82: 735-767.
3. Rossier BC, Pradervand S, Schild L, Hummler E. Epithelial sodium channel and the control of sodium balance: interaction between genetic and environmental factors. Annu Rev Physiol 2002; 64: 877-897.
4. + channel is made of three homologous subunits. Nature 1994; 367: 463-467.
5. + channel. J Biol Chem 1995; 270: 27411-27414.
6. Ji H-L, Benos DJ. Degenerin sites mediate proton activation of δβγ -epithelial sodium channel. J Biol Chem 2004; 279: 26939-26947.
7. Bangel N, Kusche-Vihrog K, Langhorst H, Weber W-M. Cloning of the epithelial sodium channel δ subunit in human nasal epithelium. Acta Physiol 2008; 188: S107.
8. Firsov D, Gautschi I, Merillat A-M, Rossier BC, Schild L. The heterotetrameric architecture of the epithelial sodium channel (ENaC). EMBO J 1998; 17: 344-352.
9. + channel subunit stoichiometry. Biophys J 2005; 88: 3966-3975.
10. Snyder PM, Cheng C, Prince LS, Rogers JC, Welsh MJ. Electropyhsiological and biochemical evidence that DEG/ENaC cation channels are composed of nine subunits. J Biol Chem 1998; 273: 681-684.
11. + channel mechanism revealed by the acid-sensing ion channel 1 structure. IUBMB Life 2008; 60: 620-628.
12. + channels by β-subunit mutation. Am J Physiol 1996; 270: C208-C213.
13. Schild L. The epithelial sodium channel: from molecule to disease. Rev Physiol Biochem Pharmacol 2004; 151: 93-107.
14. - secretion in non-cystic fibrosis and cystic fibrosis human nasal epithelium. Cell Physiol Biochem 1999; 9: 1-11.
15. Berdiev BK, Ismailow II. Epithelial sodium channels in cystic fibrosis. Curr Top Membr 1999; 47: 351-380.
16. + absorption produces cystic fibrosis-like lung disease in mice. Nature Med 2004; 10: 487-493.
17. Bangel N, Dahlhoff C, Sobczak K, Weber WM, Kusche-Vihrog K. Upregulated expression of ENaC in human CF nasal epithelium. J Cyst Fibros 2008; 7: 197-205.
18. Hofmann T, Stutts MJ, Ziersch A, et al. Effects of topically applied benzamil and amiloride on nasal potential difference and sodium absorption in cystic fibrosis respiratory epithelium. Am J Respir Crit Care Med 1998; 157: 1844-1849.
19. Zhou Z, Treis D, Schubert SC, et al. Preventive but not late amiloride therapy reduces morbidity and mortality of lung disease in betaENaC-overexpressing mice. Am J Respir Crit Care Med 2008; 178: 1245-1256.
20. Tomkiewicz RP, App EM, Zayas JG, et al. Amiloride inhalation therapy in cystic fibrosis. Am Rev Respir Dis 1993; 148: 1002-1007.
21. Hofmann T, Stutts MJ, Ziersch A, et al. Effects of topically delivered benzamil and amiloride on nasal potential difference and sodium absorption in cystic fibrosis respiratory epithelium. Am J Respir Crit Care Med 1998; 157: 1844-1849.
22. Phillips MI, Gyurko R. Antisense oligonucleotides: new tools for physiology. News Physiol Sci 1997; 12: 99-105.
23. Jain L, Chen XJ, Malik B, Al-Khalili O, Eaton DC. Antisense oligonucleotides against the alpha-subunit of ENaC decrease lung epithelial cation-channel activity. Am J Physiol 1999; 276: L1046-L1051.
24. Ma H-P, Al-Khalili O, Ramosevac S, et al. Steroids and exogenous γ-ENaC subunit modulate cation channels formed by α-ENaC in human B lymphocytes. J Biol Chem 2004; 279: 33206-33212.
25. + channels. Biochem Biophys Res Com 1997; 237: 488-491.
26. Gruenert DC, Finkbeiner WE, Widdicombe JH. Culture and transformation of human airway epithelial cells. Am J Physiol 1995; 268: L347-L360.
27. Hirsh AJ, Sabater JR, Zamurs A, et al. Evaluation of second generation amiloride analogs as therapy for cystic fibrosis lung disease. J Pharmacol Exp Ther 2004; 311: 929-938.
28. Weber W-M, Clauss W, Cuppens H, Cassiman JJ, Van Driessche W. Capacitance measurements reveal different pathways for the activation of CFTR. Pflügers Arch - Eur J Physiol 1999; 438: 561-569.
29. Hofacker I, Fontana W, Stadtler PF, Bonhoeffer S, Tacker M, Schuster P. Fast folding and comparison of RNA secondary structures. Monatshefte für Chemie 1994; 125: 167-188.
30. Blank U, Clauss W, Weber W-M. Effects of benzamil in human cystic fibrosis airway epithelium. Cell Physiol Biochem 1995; 5: 385-390.
31. + absorption and reversible block by phenamil. Pflügers Arch - Eur J Physiol 1997; 434: 19-24.
32. Bradford MM. A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal Biochem 1976; 72: 248-254.
33. Abramoff MD, Magelhaes PJ, Ram SJ. Image Processing with ImageJ. Biophot Intern 2004; 11: 36-42.
34. 2+. FEBS Lett 2002; 515: 132-138.
35. Weber W-M. Ion currents of Xenopus laevis oocytes: State of the art. Biochim Biophys Acta 1999; 1421: 213-233.
36. + concentration on amiloride block and transport kinetics of rat ENaC expressed in Xenopus oocytes. Pflügers Arch - Eur J Physiol 2002; 443: 882-891.
37. + transport in cystic fibrosis respiratory epithelia. J Clin Invest 1986; 78: 1245-1252.
38. McNicholas CM, Canessa CM. Diversity of channels generated by different combinations of epithelial channel subunits. J Gen Physiol 1997; 109: 681-692.
39. Stein CA. Exploiting the potential of antisense: beyond phosphorothioate oligodeoxynucleotides. Chem Biol 1996; 3: 319-323.
40. Ho SP, Britton DHO, Stone BA, et al. Potent antisense oligonucleotides to the human multidrug resistance-1 mRNA are rationally selected by mapping RNA-accessible sites with oligonucleotide libraries. Nucleic Acid Res 1996; 24: 1901-1907.
41. Kronenwett R, Steidl U, Kirsch M, Sczakiel G, Haas R. Oligodeoxyribonucleotide uptake in primary human hematopoietic cells is enhanced by cationic lipids and depends on the hematopoietic cell subset. Blood 1998; 91: 852-862.
42. Akhtar S, Hughes MD, Khan A, et al. The delivery of antisense therapeutics. Adv Drug Deliv Rev 2000; 44: 3-21.
43. Yakubov LA, Deeva EA, Zarytova VF, et al. Mechanism of oligonucleotide uptake by cells: involvement of specific receptors? Proc Natl Acad Sci USA 1989; 86: 6454-6458.
44. Wagner RW. Gene inhibition using antisense oligodeoxynucleotides. Nature 1994; 372: 333-335.
45. Zamecnik PC, Stephenson ML. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci USA 1978; 75: 280-284.
46. Wagner RW, Matteucci MD, Grant D, Huang T, Froehler BC. Potent and selective inhibition of gene expression by an antisense heptanucleotide. Nat Biotechnol 1996; 14: 840-844.
47. Akhtar S. Antisense technology: selection and delivery of optimally acting antisense oligonucleotides. J Drug Target 1998; 5: 225-234.
48. Crooke ST. Antisense strategies. Curr Mol Med 2004; 4: 465-487.
49. Yokota T, Takeda S, Lu QL, Partridge TA, Nakamura A, Hoffman EP. A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Arch Neurol 2009; 66: 32-38.
50. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference-based therapeutics. Nature 2009; 457: 426-433.
51. + and fluid absorption in the mammalian distal lung. Am J Physiol Lung Cell Mol Physiol 2008; 294: L401-L408.
52. Elfinger M, Geiger J, Hasenpusch G, et al. Targeting of the beta(2)-adrenoceptor increases nonviral gene delivery to pulmonary epithelial cells in vitro and lungs in vivo. J Control Release 2009; 135: 234-241.