Targeting transgene expression to airway epithelia and submucosal glands, prominent sites of human CFTR expression

Molecular Therapy

Volumn 2, Issue 4, 2000, Pages 359-367

  Chow, Yu Hua ^a   Plumb, Jonathan ^a   Wen, Yanxia ^a   Steer, Brent M ^a   Lu, Zhan ^b   Buchwald, Manuel ^b,c   Hu, Jim ^a,b  

^a UNIVERSITY OF TORONTO   (Canada)

^b HOSPITAL FOR SICK CHILDREN   (Canada)

^c UNIVERSITY OF TORONTO   (Canada)

Author keywords

Cytokeratin; Gene expression; Lung epithelia; Transgenic mice; galactosidase

Indexed keywords

MUS MUSCULUS;

CFTR PROTEIN, HUMAN; KERATIN; PRIMER DNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ANIMAL; ARTICLE; BIOSYNTHESIS; CHEMISTRY; CYTOLOGY; ENHANCER REGION; EPITHELIUM CELL; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETICS; HUMAN; LACTOSE OPERON; LUNG; MALE; METABOLISM; MOUSE; MUCOSA; PHYSIOLOGY; POLYMERASE CHAIN REACTION; PROMOTER REGION; REPORTER GENE; TISSUE DISTRIBUTION; TRANSGENE; TRANSGENIC MOUSE;

ANIMALS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DNA PRIMERS; ENHANCER ELEMENTS (GENETICS); EPITHELIAL CELLS; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENES, REPORTER; HUMANS; KERATINS; LAC OPERON; LUNG; MALE; MICE; MICE, TRANSGENIC; MUCOUS MEMBRANE; POLYMERASE CHAIN REACTION; PROMOTER REGIONS (GENETICS); TISSUE DISTRIBUTION; TRANSGENES;

EID: 0034298004     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0135     Document Type: Article

References (39)

1. Welsh, M. J., Tsui, L.-C., Boat, T. F., and Beaudet, A. L. (1995). Cystic fibrosis. In The Metabolic and Molecular Bases of Inherited Disease (C. R. Scriver, A. L. Beaudet, W. S. Sly, and D. Valle, Eds.), pp. 3799-3876, McGraw-Hill, New York.
2. Rommens, J. M., Iannuzzi, M. C., Kerem, B., Drumm, M. L., Melmer, G., Dean, M., Rozmahel, R., Cole, J. L., Kennedy, D., Hidaka, N., Zsiga, M., Buchwald, M., Riordan, J. R., Tsui, L.-C., and Collins, F. S. (1989). Identification of the cystic fibrosis gene: Chromosome walking and jumping. Science 245: 1059-1065.
3. Riordan, J. R., Rommens, J. M., Kerem, B., Alon, N., Rozmahel, R., Grzelczak, Z., Zielenski, J., Lok, S., Plavsic, N., Chou, J. L., Drumm, M. L., Iannuzi, M. C., Collins, F. S., and Tsui, L. C. (1989). Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA. Science 245: 1066-1073.
4. Kerem, B., Rommens, J. M., Buchanan, J. A., Markiewicz, D., Cox, T. K., Chakravarti, A., Buchwald, M., and Tsui, L. C. (1989). Identification of the cystic fibrosis gene: genetic analysis. Science 245: 1073-1080.
5. Wine, J. J. (1999). The genesis of cystic fibrosis lung disease. J. Clin. Invest. 103: 309-312.
6. Crystal, R. G. (1995). Transfer of genes to humans: Early lessons and obstacles to success. Science 270: 404-410.
7. Boucher, R. C. (1999). Status of gene therapy for cystic fibrosis lung disease. J. Clin. Invest. 103: 441-445.
8. Moll, R., Franke, W. W., Schiller, D. L., Geiger, B., and Krepler, R. (1982). The catalog of human cytokeratins: Patterns of expression in normal epithelia, tumors and cultured cells. Cell 31: 11-24.
9. Abe, M., and Oshima, R. G. (1990). A single human keratin 18 gene is expressed in diverse epithelial cells of transgenic mice J, Cell Biol. 111: 1197-1206.
10. Kulesh, D. A., and Oshima, R. G. (1988). Cloning of the human keratin 18 gene and its expression in nonepithelial mouse cells. Mol. Cell. Biol. 8:1540-1550.
11. Neznanov, N., Thorey, I. S., Cecena, G., and Oshima, R. G. (1993). Transcriptional insulation of the human keratin 18 gene in transgenic mice. Mol. Cell. Biol. 13: 2214-2223.
12. Wilson, C., Bellen, H. J., and Gehring, W. J. (1990). Position effects on eukaryotic gene expression. Annu. Rev. Cell Biol. 6: 679-714.
13. Garrick, D., Fiering, S., Martin, D. I., and Whitelaw, E. (1998). Repeat-induced gene silencing in mammals. Nat. Genet. 18: 56-59.
14. Oshima, R. G., Abrams, L., and Kulesh, D. (1990). Activation of an intron enhancer within the keratin 18 gene by expression of c-fos and c-jun in undifferentiated F9 embryonal carcinoma cells. Genes Dev. 4: 835-848.
15. Pankov, R., Umezawa, A., Maki, R., Der, C. J., Hauser, C. A., and Oshima, R. G. (1994). Oncogene activation of human keratin 18 transcription via the Ras signal transduction pathway. Proc. Natl. Acad. Sci. USA 91: 873-877.
16. Pankov, R., Neznanov, N., Umezawa, A., and Oshima, R. G. (1994). AP-1, ETS, and transcriptional silencers regulate retinoic acid-dependent induction of keratin 18 in embryonic cells. Mol. Cell. Biol. 14: 7744-7757.
17. Chow, Y. H., O'Brodovich, H., Plumb, J., Wen, Y., Sohn, K. J., Lu, Z., Zhang, F., Lukacs, G. L., Tanswell, A. K., Hui, C. C., Buchwald, M., and Hu, J. (1997). Development of an epithelium-specific expression cassette with human DNA regulatory elements for transgene expression in lung airways. Proc. Natl. Acad. Sci. USA 94: 14695-14700.
18. Jobling, S. A., and Gehrke, L. (1987). Enhanced translation of chimaeric messenger RNAs containing a plant viral untranslated leader sequence. Nature 325: 622-625.
19. Kalderon, D., Roberts, B. L., Richardson, W. D., and Smith, A. E. (1984). A short amino acid sequence able to specify nuclear location. Cell 39: 499-509.
20. Weiss, D. J., Liggitt, D., and Clark, J. G. (1997). In situ histochemical detection of beta-galactosidase activity in lung: assessment of X-Gal reagent in distinguishing lacZ gene expression and endogenous beta-galactosidase activity. Hum. Gene Ther. 8: 1545-1554.
21. Bonnerot, C., Rocancourt, D., Briand, P., Grimber, G., and Nicolas, J. F. (1987). A beta-galactosidase hybrid protein targeted to nuclei as a marker for developmental studies. Proc Natl. Acad. Sci. USA 84: 6795-6799.
22. Pack, R. J., Al-Ugaily, L. H., Morris, G., and Widdicombe, J. G. (1980). The distribution and structure of cells in the tracheal epithelium of the mouse. Cell Tissue Res. 208: 65-84.
23. Pack, R. J., Al-Ugaily, L. H., and Morris, G. (1981). The cells of the tracheobronchial epithelium of the mouse: A quantitative light and electron microscope study. J. Anat. 132: 71-84.
24. Trezise, A. E. O., and Buchwald, M. (1991). In vivo cell-specific expression of the cystic fibrosis transmembrane conductance regulator. Nature 353: 434-437.
25. Cohn, J. A., Strong, T. V., Picciotto, M. R., Nairn, A. C., Collins, F. S., and Fitz, J. G. (1993). Localization of the cystic fibrosis transmembrane conductance regulator in human bile duct epithelial cells. Gastroenterology 105: 1857-1864.
26. Strong, T. V., Boehm, K., and Collins, F. S. (1994). Localization of cystic fibrosis transmembrane conductance regulator mRNA in the human gastrointestinal tract by in situ hybridization. J. Clin. Invest. 93: 347-354.
27. Dray-Charier, N., Paul, A., Veissiere, D., Mergey, M., Scoazec, J. Y., Capeau, J., Brahimi-Horn, C., and Housset, C. (1995). Expression of cystic fibrosis transmembrane conductance regulator in human gallbladder epithelial cells. Lab. Invest. 73: 828-836.
28. al-Shawi, R., Kinnaird, J., Burke, J., and Bishop, J. O. (1990). Expression of a foreign gene in a line of transgenic mice is modulated by a chromosomal position effect. Mol. Cell. Biol. 10: 1192-1198.
29. Kioussis, D., and Festenstein, R. (1997). Locus control regions: Overcoming heterochromatin-induced gene inactivation in mammals. Curr. Opin. Genet. Dev. 7: 614-619.
30. Grosveld, F., van Assendelft, G. B., Greaves, D. R., and Kollias, G. (1987). Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 51: 975-985.
31. Pasceri, P., Pannell, D., Wu, X., and Ellis, J. (1998). Full activity from human beta-globin locus control region transgenes requires 5′HS1, distal beta-globin promoter, and 3′ beta-globin sequences. Blood 92: 653-663.
32. Ellis, J., Tan-Un, K. C., Harper, A., Michalovich, D., Yannoutsos, N., Philipsen, S., and Grosveld, F. (1996). A dominant chromatin-opening activity in 5′ hypersensitive site 3 of the human beta-globin locus control region. EMBO J. 15: 562-568.
33. Thorey, I. S., Meneses, J. J., Neznanov, N., Kulesh, D. A., Pedersen, R. A., and Oshima, R. G. (1993). Embryonic expression of human keratin 18 and K18-beta-galactosidase fusion genes in transgenic mice. Dev. Biol. 160: 519-534.
34. Brezillon, S., Dupuit, F., Hinnrasky, J., Marchand, V., Kalin, N., Tummler, B., and Puchelle, E. (1995). Decreased expression of the CFTR protein in remodeled human nasal epithelium from non-cystic fibrosis patients. Lab. Invest. 72: 191-200.
35. Brezillon, S., Hamm, H., Heilmann, M., Schafers, H. J., Hinnrasky, J., Wagner, T. O., Puchelle, E., and Tummler, B. (1997). Decreased expression of the cystic fibrosis transmembrane conductance regulator protein in remodeled airway epithelium from lung transplanted patients. Hum. Pathol. 28: 944-952.
36. Mulberg, A. E., Resta, L. P., Wiedner, E. B., Altschuler, S. M., Jefferson, D. M., and Broussard, D. L. (1995). Expression and localization of the cystic fibrosis transmembrane conductance regulator mRNA and its protein in rat brain. J. Clin. Invest. 96: 646-652.
37. Engelhardt, J. F., Yankaskas, J. R., Ernst, S. A., Yang, Y., Marino, C. R., Boucher, R. C., Cohn, J. A., and Wilson, J. M. (1992). Submucosal glands are the predominant site of CFTR expression in the human bronchus. Nat. Genet. 2: 240-248.
38. Engelhardt, J. F., Zepeda, M., Cohn, J. A., Yankaskas, J. R., and Wilson, J. M. (1994). Expression of the cystic fibrosis gene in adult human lung. J. Clin. Invest. 93: 737-749.
39. Tizzano, E. F., O'Brodovich, H., Chitayat, D., Benichou, J. C., and Buchwald, M. (1994). Regional expression of CFTR in developing human respiratory tissues. Am. J. Respir. Cell Mol. Biol. 10: 355-362.