High-efficiency transfer of cystic fibrosis transmembrane conductance regulator cDNA into cystic fibrosis airway cells in culture using lactosylated polylysine as a vector

Human Gene Therapy

Volumn 10, Issue 4, 1999, Pages 615-622

  Kollen, Wouter J W ^a,b   Mulberg, Andrew E ^a   Wei, Xiaofang ^a,b   Sugita, Makoto ^a   Raghuram, Viswanathan ^a   Wang, Jing ^a,b   Foskett, J Kevin ^a   Glick, Mary Catherine ^a,b   Scanlin, Thomas F ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CHLORIDE CHANNEL; COMPLEMENTARY DNA; DIGOXIGENIN; GLIAL FIBRILLARY ACIDIC PROTEIN; GLYCEROL; LACTOSE; MESSENGER RNA; PEPTIDE; POLYLYSINE; TRANSMEMBRANE CONDUCTANCE REGULATOR;

AMINO TERMINAL SEQUENCE; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CORRELATION FUNCTION; CYSTIC FIBROSIS; DNA PROBE; ELECTROPHYSIOLOGY; EXON; EXPRESSION VECTOR; FUSION GENE; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IN SITU HYBRIDIZATION; PATCH CLAMP; PROTEIN EXPRESSION; RESPIRATORY EPITHELIUM; VIRUS RECOMBINANT;

BASE SEQUENCE; CELL LINE, TRANSFORMED; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DNA PRIMERS; DNA, COMPLEMENTARY; DRUG CARRIERS; GENE TRANSFER TECHNIQUES; POLYLYSINE; TRANSFECTION;

ANIMALIA;

EID: 0033105798     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950018689     Document Type: Article

References (29)

1. BIESSEN, E.A.L., BEUTING, D.M., VIETSCH, H., BIJSTERBOSCH, M.K., and VAN BERKEL, T.J.C. (1994). Specific targeting of the antiviral drug 5-iodo 2′-deoxyuridine to the parenchymal liver cell using lactosylated poly-L-lysine. Hepatology 21, 806-815.
2. BRASIER, A.R., TATE, J.E., and HABENER, J.F. (1989). Optimized use of the firefly luciferase assay as a reporter gene in mammalian cell lines. Biotechniques 10, 1116-1122.
3. CHENG, S.H., and SCHEULE, R.K. (1998). Airway delivery of cationic lipid: DNA complexes for cystic fibrosis. Adv. Drug Delivery Rev. 30, 173-184.
4. CRYSTAL, R.G. (1995). Transfer of genes to humans: Early lessons and obstacles to success. Science 270, 404-410.
5. DE WET, J., WOOD, K., DE LUCA, M., HELSINKI, D., and SUBRAMANI, S. (1987). Firefly luciferase gene: Structure and expression in mammalian cells. Mol. Cell. Biol. 7, 725-737.
6. DI STEFANO, G., BUSI, C., MATTIOLI, A., and FIUME, L. (1995). Selective delivery to the liver of antiviral nucleoside analogs coupled to a high molecular mass lactosaminated poly-L-lysine and administered to mice by intramuscular route. Biochem. Pharm. 12, 1769-1775.
7. ENGELHARDT, J.F., YANG, Y., STRATFORD-PERRICAUDET, L.D., ALLEN, E.D., KOZARSKY, K., PERRICAUDET, M., YANKASKAS, J.R., and WILSON, J.M. (1993). Direct gene transfer of human CFTR into himan bronchial epithelia of xenografts with El-deleted adenoviruses. Nature Genet. 4, 27-34.
8. ERBACHER, P., ROCHE, A.C., MONSIGNY, M., and MIDOUX, P. (1995). Glycosylated polylysine/DNA complexes: Gene transfer efficiency in relation with the size and the sugar substitution level of glycosylated polylysines and with the plasmid size. Bioconjugate Chem. 6, 401-410.
9. ERBACHER, P., BOUSSER, M.T., RAIMOND, J., MONSIGNY, M., MIDOUX, P., and ROCHE, A.C. (1996). Gene transfer by DNA/glycosylated polylysine complexes into human blood monocyte-derived macrophages. Hum. Gene Ther. 7, 721-729.
10. FASBENDER, A., ZABNER, J., ZEIHER, B.C., and WELSH, M.J. (1997). A low rate of cell proliferation and reduced DNA uptake limit cationic lipid-mediated gene transfer to primary cultures of ciliated human airway epithelia. Gene Ther. 4, 1173-1180.
11. FIUME, L., DI STEFANO, G., BUSI, C., MATTIOLI, A., GERVASI, G.B., BERTINI, M., BARTOLI, C., CATALANI, R., CACCIA, G., FARINA, C., FISSI, A., PIERONI, O., GIUSEPPETTI, R., D'UGO, E., BRUNI, R., and RAPICETTA, M. (1997). Hepatotropic conjugate of adenine monophosphate with lactosaminated poly-L-lysine. J. Hepatol. 26, 253-259.
12. JETTEN, A.M., YANKASKAS, J.R., STUTTS, M.J., WILLUMSEN, N.J., and BOUCHER, R.C. (1989). Persistence of abnormal chloride conductance regulation in transformed cystic fibrosis epithelia. Science 244, 1472-1475.
13. JOHNSON, L.G., OLSEN, J.C., SARKADI, B., MOORE, K.L., SWANSTROM, R., and BOUCHER, R.C. (1992). Efficiency of transfer for restoration of normal airway epithelial function in cystic fibrosis. Nature (London) 2, 21-25.
14. KOLLEN, W.J.W., MIDOUX, P., ERBACHER, P., YIP, A., ROCHE, A.C., MONSIGNY, M., CLICK, M.C., and SCANLIN, T.F. (1996). Gluconoylated and glycosylated polylysines as vectors for gene transfer into cystic fibrosis airway epithelial cells. Hum. Gene Ther. 7, 1577-1586.
15. KOLLEN, W.J.W., SCHEMBRI, F., GERWIG, G.J., VLIEGENTHART, J.F.G., GLICK, M.C., and SCANLIN, T.F. (1999). Enhancement of gene transfer into cystic fibrosis airway epithelial cells with lactosylated polylysine as a nonviral vector. Am. J. Respir. Cell Mol. Biol. (in press).
16. LOWRY, O., ROSEBROUGH, N., FARR, A., and RANDALL, R. (1951). Protein measurement with the folin phenol reagent. J. Biol. Chem. 193, 265-275.
17. MARTINEZ-FONG, D., MULERSMAN, J.E., PURCHIO, A.F., ARMENDARIZ-BORUNDA, J., and MARTINEZ-HERNANDEZ, A. (1994). Nonenzymatic glycosylation of poly-L-lysine: A new tool for targeted gene delivery. Hepatology 20, 1602-1608.
18. MULBERG, A.E., RESTA, L.P., WIEDNER, E.B., BAO, X., MARSHALL, J. JEFFERSON, D.M., and ALTSCHULER, S.M. (1994). Cystic fibrosis transmembrane conductance regulator protein expression in brain. NeuroReport 5, 1684-1688.
19. 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.
20. MULBERG, A.E., WEYLER, R.T., ALTSCHULER, S.M., and HYDE, T.M. (1998). Cystic fibrosis transmembrane conductance regulator expression in human hypothalamus. NeuroReport 9, 141-144.
21. OLSEN, J.C., JOHNSON, L.G., STUTTS, M.J., SARKADI, B., YANKASKAS, J.R., SWANSTROM, R., and BOUCHER, R. (1992). Correction of the apical membrane chloride permeability defect in polarized cystic fibrosis airway epithelia following retroviral-mediated transfer. Hum. Gene Ther. 3, 253-266.
22. PERALES, J.C., FERKOL, T., BEEGEN, H., RATNOFF, O.D., and HANSON R.W. (1994). Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc. Natl. Acad. Sci. USA 91, 4086-4090.
23. ROBINSON, C., and SCANLIN, T.F. (1997). Cystic fibrosis. In Pulmonary Diseases and Disorders. A.P. Fishman, ed. (McGraw-Hill, New York) pp. 1273-1294.
24. ROSENFELD, M.A., and COLLINS, F.S. (1996). Impact of basic research on tomorrow's medicine: Gene therapy for cystic fibrosis. Chest 19, 241-252.
25. - channel and CFTR-associated ATP channel: Distinct pores regulated by common gates. EMBO J. 17, 898-908.
26. VERMA, I.M., and SOMIA, N. (1997). Gene therapy - promises, problems and prospects. Nature (London) 389, 239-242.
27. WEI, X., EISMAN, R., XU, J., HARSCH, A.D., MULBERG, A.E., BEVINS, C.L., GLICK, M.C., and SCANLIN, T.F. (1996). Turnover of the cystic fibrosis transmembrane conductance regulator (CFTR): Slow degradation of wild-type and AF508 CFTR in surface membrane preparations of immortalized airway epithelial cells. J. Cell. Physiol. 168, 373-384.
28. WILSON, J.M. (1995). Gene therapy for cystic fibrosis: Challenges and future directions. J. Clin. Invest. 96, 2547-2554.
29. ZAUNER, W., OGRIS, M., and WAGNER, E. (1998). Polylysine-based transfection systems utilizing receptor-mediated delivery. Adv. Drug. Delivery Rev. 30, 97-113.