Novel shielded transferrin-polyethylene glycol-polyethylenimine/DNA complexes for systemic tumor-targeted gene transfer

Bioconjugate Chemistry

Volumn 14, Issue 1, 2003, Pages 222-231

  Kursa, Malgorzata ^b   Walker, Greg F ^a   Roessler, Vanessa ^b   Ogris, Manfred ^a   Roedl, Wolfgang ^a   Kircheis, Ralf ^b,c   Wagner, Ernst ^a  

^a UNIVERSITY OF MUNICH   (Germany)

^b BOEHRINGER INGELHEIM RCV GMBH AND CO KG   (Austria)

^c Igeneon AG   (Austria)

Author keywords

[No Author keywords available]

Indexed keywords

BIOMOLECULES; DNA; GENE EXPRESSION; GENE TRANSFER; MIXING; PARTICLE SIZE; SURFACE CHARGE; THAWING; TUMORS;

DNA COMPLEX; FREEZE-THAWING; IN-VITRO; IN-VIVO; K562 CELLS; PARTICLES SIZES; PLASMID DNA; POLY(ETHYLENIMINE); TUMOR-TARGETING; VITRO TRANSFECTION;

POLYETHYLENE GLYCOLS;

DNA; MACROGOL DERIVATIVE; POLYETHYLENEIMINE DERIVATIVE; TRANSFERRIN; TUMOR NECROSIS FACTOR ALPHA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; COMPLEX FORMATION; DRUG CONJUGATION; DRUG FORMULATION; DRUG SYNTHESIS; GENE TARGETING; GENE TRANSFER; MOUSE; NONHUMAN; PARTICLE SIZE; SURFACE CHARGE; TUMOR; TUMOR GROWTH;

ANIMALIA; MURINAE;

EID: 0142072936     PISSN: 10431802     EISSN: None     Source Type: Journal    
DOI: 10.1021/bc0256087     Document Type: Article

References (35)

1. Ogris, M., and Wagner, E. (2002) Targeting tumors with nonviral gene delivery systems. Drug Discovery Today 15, 479-485.
2. Felgner, P. L., Barenholz, Y., Behr, J. P., Cheng, S. H., Cullis, P., Huang, L., Jessee, J. A., Seymour, L., Szoka, F., Thierry, A. R., Wagner, E., and Wu, G. (1997) Nomenclature for synthetic gene delivery systems [editorial]. Hum. Gene Ther. 8, 511-512.
3. Kircheis, R, Wightman, L., Kursa, M., Ostermann E, and Wagner, E. (2002) Tumor-targeted gene delivery: an attractive strategy to use highly active effector molecules in cancer treatment. Gene Ther. 9, 731-735.
4. Anchordoquy, T. J., Dean Allison, S., Molina, M., Girouard, L. G., and Carson, T. K. (2001) Physical stabilization of DNA-based therapeutics. Drug Discovery Today 6, 463-470.
5. Boussif, O., Lezoualc'h, F., Zanta, M. A., Mergny, M. D., Scherman, D., Demeneix, B., and Behr, J. P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. U.S.A. 92, 7297-7301.
6. Ferrari, S., Moro, E., Pettenazzo, A., Behr, J. P., Zacchello, F., and Scarpa, M. (1997) ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther. 4, 1100-1106.
7. Blessing, T, Kursa, M., Holzhauser, R, Kircheis, R, and Wagner, E. (2001) Different strategies for formulation of PEGylated EGF-conjugated PEI/DNA complexes for targeted gene delivery. Bioconjugate Chem. 12, 529-537.
8. Leamon, C. P., Weigl, D., and Hendren, R. W., (1999) Folate copolymer-mediated transfection of cultured cells. Bioconjugate Chem. 10, 947-957.
9. Kircheis, R., Wightman, L., Schreiber, A., Robitza, B., Rossler, V., Kursa, M., and Wagner, E., (2001) Polyethylenimine/DNA complexes shielded by transferrin target gene expression to tumors after systemic application. Gene Ther. 8, 28-40.
10. Ogris, M, Brunner, S., Schuller, S., Kircheis, R., and Wagner, E. (1999) PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther. 6, 595-605.
11. Oupicky, D., Ogris, M., and Seymour, L., (2002) Development of long-circulating polyelectrolyte complexes for systemic delivery of genes. J. Drug Targeting 10, 93-98.
12. Kichler, A., Chillón, M., Leborgne, C., Danos, O., and Frisch, B., (2002) Intranasal gene delivery with a polyethylenimine-PEG conjugate. J. Controlled Release 379-388.
13. Kircheis, R. Schuller, S., Brunner, S., Orgris, M., Heider, K. H., Zauner, W., and Wagner, E. (1999) Polycation-based DNA complexes for tumour targeted gene delivery in vivo. J. Gene Med. 1, 111-120.
14. Wolfert, M. A., Schacht, E. H., Toncheva, V., Ulbrich, K., Nazarova, O., Seymour, L. W., Dash, P. R., and Oupicky, D. (1996) Characterization of vectors for gene therapy formed by self-assembly of DNA with synthetic block copolymers. Hum. Gene Ther. 7, 2123-2133.
15. Katayose, S. and Kataoka, K. (1997) Water Soluble Polyion Complex Associates of DNA and Poly(ethylene glycol) Poly- (L-lysine) Block Copolymer. Bioconjugate Chem. 8 (5), 702-707.
16. Toncheva, V., Wolfert, M. A., Dash, P. R., Oupicky, D., Ulbrich, K., Seymour, L. W., and Schacht, E. H. (1998) Novel vectors for gene delivery formed by self-assembly of DNA with poly(L-lysine) grafted with hydrophilic polymers. Biochim. Biophys. Acta 1380, 354-368.
17. Erbacher, P, Bettinger, T., Belguise, Valladier P., Zou, S., Coll, J. L., Behr, J. B., and Remy, J. S. (1999) Transfection and physical properties of various saccharide, poly(ethylene glycol), and antibody-derivatized polyethylenimines (PEI). J. Gene Med. 1, 210-222.
18. Nguyen, H. K., Lemieux, P., Vinogradov, S. V., Gebhart, C. L, Guérin, N., Paradis, G., Bronich, T. K., Alakhov, V. Y., and Kabanov, A. V. (2000) Evaluation of polyether-polyethyleneimine graft copolymers as gene transfer agents. Gene Ther. 7, 126-138.
19. Woodle, M. C., Scaria, P., Ganesh, S., Subramanian, K., Titmas, R., Cheng, C., Yang, J., Pan, Y., Weng, K., Gu, C., and Torkelson, S. (2001) Sterically stabilized polyplex: ligand-mediated activity. J. Controlled Release 74, 309-311.
20. Ogris, M, Steinlein, P., Carotta, S., Brunner, S., and Wagner, E. (2001) DNA/polyethylenimine transfection particles: influence of ligands, polymer size, and PEGylation on internalization and gene expression. AAPS Pharm Sci. 3, E21.
21. Sarin, V. K., Kent, S. B., Tam, J. P., and Merrifield, R. B. (1981) Quantitative monitoring of solid-phase peptide synthesis by the ninhydrin reaction. Anal. Biochem. 117, 147-157.
22. Riddles, P. W., Blakeley, R. L., and Zerner, B. (1979) Ellman's reagent: 5,5′-dithiobis(2-nitrobenzoic acid)- -a reexamination. Anal. Biochem. 94, 75-81.
23. Plank, C., Zatloukal, K., Cotten, M., Mechtler, K., and Wagner, E. (1992) Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconjugate Chem. 3, 533-539.
24. Carlsson, J., Drevin, H., and Axen, R. (1978) Protein thiolation and reversible protein - protein conjugation. N-Succinimidyl 3-(2-pyridyldithio)propionate, a new heterobifunctional reagent. Biochem. J. 173, 723-737.
25. Kircheis, R, Wightman, L., and Wagner, E. (2001) Design and gene delivery activity of modified polyethylenimines. Adv. Drug Delivery Rev. 53, 341-358.
26. Kircheis, R., Kichler, A., Wallner, G., Kursa, M., Ogris, M., Felzmann, T., Buchberger, M., and Wagner, E. (1997) Coupling of cell-binding ligands to polyethylenimine for targeted gene transfer. Gene Ther. 4, 409-418.
27. Cotten, M., Langle, Rouault, Kirlappos, H., Wagner, E., Mechtler, K., Zenke, M., Beug, H., and Birnstiel, M. L. (1990) Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc. Natl. Acad. Sci. U.S.A. 87, 4033-4037.
28. Mislick, K. A. and Baldeschwieler, J. D. (1996) Evidence for the role of proteoglycans in cation-mediated gene transfer. Proc. Natl. Acad. Sci. U.S.A. 93, 12349-12354.
29. Wagner, E., Curiel, D., and Cotten, M. (1994) Delivery of drugs, proteins and genes into cells using transferrin as a ligand for receptor-mediated endocytosis. Adv. Drug Delivery Rev. 14, 113-136.
30. Ogris, M., Steinlein, P., Kursa, M., Mechtler, K., Kircheis, R., and Wagner, E. (1998) The size of DNA/Transferrin-PEI complexes is an important factor for gene expression in cultured cells. Gene Ther. 5, 1425-1433.
31. Wightman, L., Kircheis, R., Rössler, V., Carotta, S., Ruzicka, R., Kursa, M., and Wagner, E. (2001) Different behavior of branched and linear polyethlyenimine for gene delivery in vitro and in vivo. J. Gene Med. 3, 362-372.
32. Cherng, J. Y., Wetering P., Talsma, H., Crommelin, D. J., and Hennink, W. E. (1999) Stabilization of polymer-based gene delivery systems. Int. J. Pharm. 183, 25-28.
33. Kwok, K. Y., Adami, R. C., Hester, K. C., Park, Y, Thomas, S., and Rice, K. G, (2000) Strategies for maintaining the particle size of peptide DNA condensates following freeze-drying. Int. J. Pharm. 203, 81-88.
34. Kircheis, R, Ostermann, E, Wolschek, M. F., Lichtenberger, C., Magin-Lachmann, C., Wightman, L., Kursa, M., and Wagner, E. (2002) Tumor-targeted gene delivery of tumor necrosis factor-alpha induces tumor necrosis and tumor regression without systemic toxicity. Cancer Gene Ther. 9, 673-680.
35. Hashizume, H, Baluk, P, Morikawa, S., McLean, J. W., Thurston, G., Roberge, S., Jain, R. K., and McDonald, D. M. (2000) Openings between defective endothelial cells explain tumor vessel leakiness. Am. J. Pathol. 156, 1363-1380.