CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector

Journal of Cellular Physiology

Volumn 214, Issue 2, 2008, Pages 442-455

  Rakonczay Jr , Zoltán ^a,b   Hegyi, Péter ^a,b   Hasegawa, Mamoru ^c   Inoue, Makoto ^c   You, Jun ^c   Iida, Akihiro ^c   Ignáth, Imre ^b   Alton, Eric W F W ^d   Griesenbach, Uta ^d   Óvári, Gabriella ^e   Vag, János ^e   Da Paula, Ana C ^f   Crawford, Russell M ^h   Varga, Gàbor ^e   Amaral, Margarida D ^f,g   Mehta, Anil ^h   Lonovics, János ^b   Argent, Barry E ^a   Gray, Michael A ^a  

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b UNIVERSITY OF SZEGED   (Hungary)

^c DNAVEC Research Inc   (Japan)

^d NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^e INSTITUTE OF EXPERIMENTAL MEDICINE   (Hungary)

^f NATIONAL INSTITUTE OF HEALTH DR RICARDO JORGE   (Portugal)

^g UNIVERSITY OF LISBON   (Portugal)

^h UNIVERSITY OF DUNDEE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BICARBONATE; CARRIER PROTEIN; CHLORIDE; MESSENGER RNA; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

ARTICLE; CELL GROWTH; CONTROLLED STUDY; CYSTIC FIBROSIS; GENE TRANSFER; HUMAN; HUMAN CELL; PANCREAS DISEASE; PANCREAS DUCT; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SENDAI VIRUS; VIRUS RECOMBINANT;

BETA-GALACTOSIDASE; CELL LINE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; HYDROGEN-ION CONCENTRATION; PANCREATIC DUCTS; RNA, MESSENGER; SENDAI VIRUS;

SENDAI VIRUS;

EID: 37349084579     PISSN: 00219541     EISSN: None     Source Type: Journal    
DOI: 10.1002/jcp.21220     Document Type: Article

References (64)

1. - salvage mechanisms in model systems and the mouse pancreatic duct. J Biol Chem 276:17236-17243.
2. Akabas MH. 2000. Cystic fibrosis transmembrane conductance regulator. Structure and function of an epithelial chloride channel. J Biol Chem 275:3729-3732.
3. Alper SL, Stewart AK, Chernova MN, Zolotarev AS, Clark JS, Vandorpe DH. 2006. Anion exchangers in flux: Functional differences between human and mouse SLC26A6 polypeptides. Novartis Found Symp 273:107-119.
4. Alvarez BV, Vilas GL, Casey JR. 2005. Metabolon disruption: A mechanism that regulates bicarbonate transport. EMBO J 24:2499-2511.
5. Argent BE, Gray MA, Steward MC, Case RM. 2006. Cell physiology of pancreatic ducts. In: Johnson LR, editor. Physiology of the Gastrointestinal Tract 4th edition San Diego: Elsevier. pp. 1371-1396.
6. Bitzer M, Armeanu S, Lauer UM, Neubert WJ. 2003. Sendai virus vectors as an emerging negative-strand RNA viral vector system. J Gene Med 5:543-553.
7. Chang XB, Tabcharani JA, Hou YX, Jensen TJ, Kartner N, Alon N, Hanrahan JW, Riordan JR. 1993. Protein kinase A (PKA) still activates CFTR chloride channel after mutagenesis of all 10 PKA consensus phosphorylation sites. J Biol Chem 268:11304-11311.
8. Chernova MN, Jiang L, Shmukler BE, Schweinfest CW, Blanco P, Freedman SD, Stewart AK, Alper SL. 2003. Acute regulation of the SLC26A3 congenital chloride diarrhoea anion exchanger (DRA) expressed in Xenopus oocytes. J Physiol 549:3-19.
9. - transport in mutations associated with cystic fibrosis. Nature 410:94-97.
10. DiMagno EP, Go VL, Summerskill WH. 1973. Relations between pancreatic enzyme ouputs and malabsorption in severe pancreatic insufficiency. N Engl J Med 288:813-815.
11. Dodge JA, Turck D. 2006. Cystic fibrosis: Nutritional consequences and management. Best Pract Res Clin Gastroenterol 2006. 20 531-546.
12. Farinha CM, Mendes F, Roxo-Rosa M, Penque D, Amaral MD. 2004. A comparison of 14 antibodies for the biochemical detection of the cystic fibrosis transmembrane conductance regulator protein. Mol Cell Probes 18:235-242.
13. Farinha CM, Penque D, Roxo-Rosa M, Lukacs G, Dormer R, McPherson M, Pereira M, Bot AG, Jorna H, Willemsen R, Dejonge H, Heda GD, Marino CR, Fanen P, Hinzpeter A, Lipecka J, Fritsch J, Gentzsch M, Edelman A, Amaral MD. 2004. Biochemical methods to assess CFTR expression and membrane localization. J Cyst Fibros 3:73-77.
14. Ferrari S, Griesenbach U, Iida A, Farley R, Wright AM, Zhu J, Munkonge FM, Smith SN, You J, Ban H, Inoue M, Chan M, Singh C, Verdon B, Argent BE, Wainwright B, Jeffery PK, Geddes DM, Porteous DJ, Hyde SC, Gray MA, Hasegawa M, Alton EW. 2007. Sendai virus-mediated CFTR gene transfer to the airway epithelium. Gene Ther [Epub ahead of print] doi 10.1038/sj.gt.3302991.
15. Fuerst TR, Niles EG, Studier FW, Moss B. 1986. Eukaryotic transient-expression system based on recombinant vaccinia virus that synthesizes bacteriophage T7 RNA polymerase. Proc Natl Acad Sci USA 83:8122-8126.
16. Gray MA, Pollard CE, Harris A, Coleman L, Greenwell JR, Argent BE. 1990. Anion selectivity and block of the small-conductance chloride channel on pancreatic duct cells. Am J Physiol 259:C752-C761.
17. Gray MA, Plant S, Argent BE. 1993. cAMP-regulated whole cell chloride currents in pancreatic duct cells. Am J Physiol 264:C591-C602.
18. Gray MA, Winpenny JP, Verdon B, O'Reilly CM, Argent BE. 2002. Properties and role of calcium-activated chloride channels in pancreatic duct cells. In: Fuller CM, editor. Current topics in Membranes,Vol. 53. Calcium-activated chloride channels. San Diego: Academic Press. pp. 231-256.
19. Greeley T, Shumaker H, Wang Z, Schweinfest CW, Soleimani M. 2001. Downregulated in adenoma and putative anion transporter are regulated by CFTR in cultured pancreatic duct cells. Am J Physiol 281:G1301-G1308.
20. Griesenbach U, Inoue M, Hasegawa M, Alton EW. 2005. Sendai virus for gene therapy and vaccination. Curr Opin Mol Ther 7:346-352.
21. Griesenbach U, Geddes DM, Alton EW. 2006. Gene therapy progress and prospects: Cystic fibrosis. Gene Ther 13:1061-1167.
22. Harlow E, Lane D. 1988. Antibodies: A Laboratory Manual. Cold Spring Harbor: Cold Spring Harbor Laboratory Press.
23. Hegyi P, Rakonczay Z, Jr, Gray MA, Argent BE. 2004. Measurement of intracellular pH in pancreatic duct cells: A new method for calibrating the fluorescence data. Pancreas 28:427-434.
24. - channel activity http://central.igc.gulbenkian. pt/cftr/vr/d/ hughes_iodide_selective_electrode_measure_cftr_cl_activity.pdf.
25. 2 permeability and bicarbonate transport in microperfused interlobular ducts isolated from the guinea-pig pancreas. J Physiol 528:305-315.
26. - exchanger activity and cAMP-stimulated bicarbonate secretion in pancreatic duct. Am J Physiol 292:G447-G455.
27. Kato A, Sakai Y, Shioda T, Kondo T, Nakanishi M, Nagai Y. 1996. Initiation of Sendai virus multiplication from transfected cDNA or RNA with negative or positive sense. Genes Cells 1:569-579.
28. - transport in cystic fibrosis. EMBO J 21:5662-5672.
29. Ko SB, Zeng W, Dorwart MR, Luo X, Kim KH, Milien L, Goto H, Naruse S, Soyombo A, Thomas PJ, Muallem S. 2004. Gating of CFTR by the STAS domain of SLC26 transporters. Nat Cell Biol 6:343-350.
30. + exchange. Biochemistry 41:12336-12342.
31. - exchange in mouse submandibular and pancreatic ducts. J Biol Chem 274:14670-14677.
32. - exchange by cystic fibrosis transmembrane conductance regulator expressed in NIH 3T3 and HEK 293 cells. J Biol Chem 274:3414-3421.
33. - salvage across the luminal membrane of the main pancreatic duct. J Clin Invest 105:1651-1658.
34. Linsdell P, Tabcharani JA, Rommens JM, Hou YX, Chang XB, Tsui LC, Riordan JR, Hanrahan JW. 1997. Permeability of wild-type and mutant cystic fibrosis transmembrane conductance regulator chloride channels to polyatomic anions. J Gen Physiol 110:355-364.
35. Livak KJ, Schmittgen TD. 2001. Analysis of relative gene expression data using real-time quantitative PCR and the 2(-Delta Delta C(T)). Methods 25:402-408.
36. Lohi H, Lamprecht G, Markovich D, Heil A, Kujala M, Seidler U, Kere J. 2003. Isoforms of SLC26A6 mediate anion transport and have functional PDZ interaction domains. Am J Physiol 284:C769-C779.
37. Ma T, Thiagarajah JR, Yang H, Sonawane ND, Folli C, Galietta LJ, Verkman AS. 2002. Thiazolidinone CFTR inhibitor identified by high-throughput screening blocks cholera toxin-induced intestinal fluid secretion. J Clin Invest 110:1651-1658.
38. + exchanger in bovine main pancreatic duct. Dig Dis Sci 40:2336-2340.
39. + exchanger. J Biol Chem 274:22855-22861.
40. Mohammad-Panah R, Demolombe S, Riochet D, Leblais V, Loussouarn G, Pollard H, Baro I, Escande D. 1998. Hyperexpression of recombinant CFTR in heterologous cells alters its physiological properties. Am J Physiol 274:C310-C318.
41. Moseley RH, Hoglund P, Wu GD, Silberg DG, Haila S, de la Chapelle A, Holmberg C, Kere J. 1999. Downregulated in adenoma gene encodes a chloride transporter defective in congenital chloride diarrhea. Am J Physiol 276:G185-G192.
42. Mount DB, Romero MF. 2004. The SLC26 gene family of multifunctional anion exchangers. Pflugers Arch 447:710-721.
43. Muimo R, Hornickova Z, Riemen CE, Gerke V, Matthews H, Mehta A. 2000. Histidine phosphorylation of annexin I in airway epithelia. J Biol Chem 275:36632-36636.
44. - exchanger in rat kidney proximal tubule. Am J Physiol 285:C608-C617.
45. - transporters in CFPAC-1 human pancreatic duct cells. World J Gastroenterol 12:885-895.
46. Rosenecker J, Huth S, Rudolph C. 2006. Gene therapy for cystic fibrosis lung disease: Current status and future perspectives. Curr Opin Mol Ther 8:439-445.
47. Rowntree RK, Harris A. 2003. The phenotypic consequences of CFTR mutations. Ann Hum Genet 67:471-485.
48. Scheele GA, Fukuoka SI, Kern HF, Freedman SD. 1996. Pancreatic dysfunction in cystic fibrosis occurs as a result of impairments in luminal pH, apical trafficking of zymogen granule membranes, and solubilization of secretory enzymes. Pancreas 12:1-9.
49. Schiavi SC, Abdelkader N, Reber S, Pennington S, Narayana R, McPherson JM, Smith AE, Hoppe H IV, Cheng SH. 1996. Biosynthetic and growth abnormalities are associated with high-level expression of CFTR in heterologous cells. Am J Physiol 270:C341-351.
50. Schoumacher RA, Ram J, Iannuzzi MC, Bradbury NA, Wallace RW, Tom Hon C, Kelly DR, Schmid SM, Gelder FB, Rado TA, Frizzell RA. 1990. A cystic fibrosis pancreatic adenocarcinoma cell line. Proc Natl Acad Sci USA 87:4012-4016.
51. Shcheynikov N, Ko SB, Zeng W, Choi JY, Dorwart MR, Thomas PJ, Muallem S. 2006. Regulatory interaction between CFTR and the SLC26 transporters. Novartis Found Symp 273:177-186.
52. Shiotani A, Fukumura M, Maeda M, Hou X, Inoue M, Kanamori T, Komaba S, Washizawa K, Fujikawa S, Yamamoto T, Kadono C, Watabe K, Fukuda H, Saito K, Sakai Y, Nagai Y, Kanzaki J, Hasegawa M. 2001. Skeletal muscle regeneration after insulin-like growth factor I gene transfer by recombinant Sendai virus vector. Gene Ther 8:1043-1050.
53. Silberg DG, Wang W, Moseley RH, Traber PG. 1995. The down regulated in adenoma (dra) gene encodes an intestine-specific membrane sulfate transport protein. J Biol Chem 270:11897-11902.
54. - exchange in the villous epithelium of intact murine duodenum. Am J Physiol 288:G1241-G1251.
55. Steward MC, Ishiguro H, Case RM. 2005. Mechanisms of bicarbonate secretion in the pancreatic duct. Annu Rev Physiol 67:377-409.
56. Tamada K, Wang XP, Brunicardi FC. 2005. Molecular targeting of pancreatic disorders. World J Surg 29:325-333.
57. Thomas JA, Buchsbaum RN, Zimniak A, Racker E. 1979. Intracellular pH measurements in Ehrlich ascites tumor cells utilizing spectroscopic probes generated in situ. Biochemistry 18:2210-2218.
58. Tokusumi T, Iida A, Hirata T, Kato A, Nagai Y, Hasegawa M. 2002. Recombinant Sendai viruses expressing different levels of a foreign reporter gene. Virus Res 86:33-38.
59. Wallrapp C, Muller-Pillasch F, Solinas-Toldo S, Lichter P, Friess H, Buchler M, Fink T, Adler G, Gress TM. 1997. Characterization of a high copy number amplification at 6q24 in pancreatic cancer identifies c-myb as a candidate oncogene. Cancer Res 57:3135-3139.
60. - exchanger in the small intestine. Am J Physiol 282:G573-G579.
61. - secretion: Relevance to cystic fibrosis. EMBO J 25:5049-5057.
62. Waters DL, Dorney SFA, Gaskin KJ, Gruca MA, O'Halloran M, Wilcken B. 1990. Pancreatic function in infants identified as having cystic-fibrosis in a neonatal screening program. N Engl J Med 322:303-308.
63. 3 exchange, and Na-HCO3 cotransport. Am J Physiol 257:G317-G327.
64. Yonemitsu Y, Kitson C, Ferrari S, Farley R, Griesenbach U, Judd D, Steel R, Scheid P, Zhu J, Jeffery PK, Kato A, Hasan MK, Nagai Y, Masaki I, Fukumura M, Hasegawa M, Geddes DM, Alton EW. 2000. Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nat Biotechnol 18:970-973.