Towards multifunctional synthetic vectors

Current Gene Therapy

Volumn 4, Issue 4, 2004, Pages 445-455

  Demeneix, Barbara ^a   Hassani, Zahra ^a   Behr, Jean Paul ^b  

^a MUSÉUM NATIONAL D'HISTOIRE NATURELLE   (France)

^b UNIVERSITÉ DE STRASBOURG   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ARGINYLGLYCYLASPARTYLSERINE; CATION; DETERGENT; DNA; INTEGRIN; LIPID; LIPOSOME; MACROGOL; NUCLEIC ACID; PLASMID DNA; POLYETHYLENEIMINE; POLYMER; SPERMINE; SYNDECAN;

BACTERIUM; DIMERIZATION; DRUG DELIVERY SYSTEM; DRUG DESIGN; DRUG FORMULATION; ENDOSOME; GENE TARGETING; GENE VECTOR; HUMAN; IN VIVO STUDY; MEMBRANE TRANSPORT; NONHUMAN; NUCLEAR IMPORT; NUCLEAR PORE; OSMOSIS; REVIEW; VIRUS;

EID: 8844239128     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/1566523043345940     Document Type: Review

References (90)

1. Abdallah, B., Hassan, A., Benoist, C., Goula, D., Behr, J. P., and Demeneix, B. A. (1996). A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine, Hum Gene Ther. 7: 1947-54.
2. Andreadis, S., Fuller, A. O., and Palsson, B. O. (1998). Cell cycle dependence of retroviral transduction: An issue of overlapping time scales, Biotechnol. Bioeng. 58: 272-81.
3. Aoki, K., Furuhata, S., Hatanaka, K., Maeda, M., Remy, J. S., Behr, J. P., Terada, M., and Yoshida, T. (2001). Polyethylenimine-mediated gene transfer into pancreatic tumor dissemination in the murine peritoneal cavity, Gene Ther. 8: 508-14.
4. Behr, J. P., Demeneix, B., Loeffler, J. P., and Perez-Mutul, J. (1989). Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA, Proc. Natl. Acad. Sci. USA 86: 6982-6.
5. Blessing, T., J.S. Remy, and J.P. Behr, (1998) Template oligomerization of cations bound to DNA produces calibrated nanometric particles. J. Amer. Chem. Soc. 120: p. 8519-8520.
6. Blessing, T., Remy, J, S., and Behr, J. P. (1998). Monomolecular collapse of plasmid DNA into stable virus-like particles, Proc. Natl. Acad. Sci. USA 95: 1427-31.
7. Blessing, T., Kursa, M., Holzhauser, R., Kircheis, R., and Wagner, E. (2001). Different strategies for formation of pegylated EGF-conjugated PEI/DNA complexes for targeted gene delivery, Bioconjug. Chem. 12: 529-37.
8. 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. USA 92: 7297-301.
9. Boussif, O., Zanta, M. A., and Behr, J. P. (1996). Optimized galenics improve in vitro gene transfer with cationic molecules up to 1000-fold, Gene Ther. 3: 1074-80.
10. Bragonzi, A., Dina, G., Villa, A., Calori, G., Biffi, A., Bordignon, C., Assael, B. M., and Conese, M. (2000). Biodistribution and transgene expression with nonviral cationic vector/DNA complexes in the lungs, Gene Ther. 7: 1753-60.
11. Branden, L. J., Mohamed, A. J., and Smith, C. I. (1999). A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA, Nat. Biotechnol. 17: 784-7.
12. Brunner, S., Sauer, T., Carotta, S., Cotten, M., Saltik, M., and Wagner, E. (2000). Cell cycle dependence of gene transfer by lipoplex, polyplex and recombinant adenovirus, Gene Ther. 7: 401-7.
13. Brunner, S., Furtbauer, E., Sauer, T., Kursa, M., and Wagner, E. (2002). Overcoming the nuclear barrier: cell cycle independent nonviral gene transfer with linear polyethylenimine or electroporation, Mol. Ther. 5: 80-6.
14. Ciolina, C., Byk, G., Blanche, F., Thuillier, V., Scherman, D., and Wils, P. (1999). Coupling of nuclear localization signals to plasmid DNA and specific interaction of the conjugates with importin alpha, Bioconjug. Chem. 10: 49-55.
15. Clamme, J. P., Bernacchi, S., Vuilleumier, C., Duportail, G., and Mely, Y. (2000). Gene transfer by cationic surfactants is essentially limited by the trapping of the surfactant/DNA complexes onto the cell membrane: a fluorescence investigation, Biochim. Biophys. Acta 1467: 347-61.
16. Coen, L., Osta, R., Maury, M., and Brulet, P. (1997). Construction of hybrid proteins that migrate retrogradely and transynaptically into the central nervous system, Proc. Natl. Acad. Sci. USA 94: 9400-5.
17. Coll, J. L., Chollet, P., Brambilla, E., Desplanques, D., Behr, J. P., and Favrot, M. (1999). In vivo delivery to tumors of DNA complexed with linear polyethylenimine, Hum. Gene Ther. 10 1659-66.
18. Dauty, E., Remy, J. S., Blessing, T., and Behr, J. P. (2001). Dimerizable cationic detergents with a low cmc condense plasmid DNA into nanometric particles and transfect cells in culture, J. Am. Chem. Soc. 123: 9227-34.
19. Dauty, E., Remy, J. S., Zuber, G., and Behr, J. P. (2002). Intracellular delivery of nanometric DNA particles via the folate receptor, Bioconjug. Chem. 13: 831-9.
20. Dehio, C., Freissler, E., Lanz, C., Gomez-Duarte, O. G., David, G., and Meyer, T. F. (1998). Ligation of cell surface heparan sulfate proteoglycans by antibody-coated beads stimulates phagocytic uptake into epithelial cells: a model for cellular invasion by Neisseria gonorrhoeae, Exp. Cell Res. 242: 528-39.
21. Demeneix, B., Behr, J., Boussif, O., Zanta, M. A., Abdallah, B., and Remy, J. (1998). Gene transfer with lipospermines and polyethylenimines, Adv. Drug Deliv. Rev. 30: 85-95.
22. Densmore, C. L., Orson, F. M., Xu, B., Kinsey, B. M., Waldrep, J. C., Hua, P., Bhogal, B., and Knight, V. (2000). Aerosol delivery of robust polyethyleneimine-DNA complexes for gene therapy and genetic immunization, Mol. Ther. 1: 180-8.
23. Dolivet, G., Merlin, J. L., Barberi-Heyob, M., Ramacci, C., Erbacher, P., Parache, R. M., Behr, J. P., and Guillemin, F. (2002). In vivo growth inhibitory effect of iterative wild-type p53 gene transfer in human head and neck carcinoma xenografts using glucosylated polyethylenimine nonviral vector, Cancer Gene Ther. 9: 708-14.
24. Erbacher, P., Remy, J. S., and Behr, J. P. (1999). Gene transfer with synthetic virus-like particles via the integrin-mediated endocytosis pathway, Gene Ther. 6: 138-45.
25. Erbacher, P., Bettinger, T., Beiguise-Valladier, P., Zou, S., Coll, J. L., Behr, J. P., and Remy, J. S. (1999). Transfection and physical properties of various saccharide, poly(ethylene glycol), and antibody-derivatized polyethylenimines (PEI), J. Gene Med. 1: 210-22.
26. Fabre, V., Boutrel, B., Hanoun, N., Lanfumey, L., Fattaccini, C. M., Demeneix, B., Adrien, J., Hamon, M., and Martres, M. P. (2000). Homeostatic regulation of serotonergic function by the serotonin transporter as revealed by nonviral gene transfer, J. Neurosci. 20: 5065-75.
27. Fajac, I., Allo, J. C., Souil, E., Merten, M., Pichon, C., Figarella, C., Monsigny, M., Briand, P., and Midoux, P. (2000). Histidylated polylysine as a synthetic vector for gene transfer into immortalized cystic fibrosis airway surface and airway gland serous cells, J. Gene Med. 2: 368-78.
28. Fisher, K. D., Ulbrich, K., Subr, V., Ward, C. M., Mautner, V., Blakey, D., and Seymour, L. W. (2000). A versatile system for receptor-mediated gene delivery permits increased entry of DNA into target cells, enhanced delivery to the nucleus and elevated rates of transgene expression, Gene Ther. 7: 1337-43.
29. Foglieni, C., Bragonzi, A., Cortese, M., Cantu, L., Boletta, A., Chiossone, I., Soria, M. R., and Monaco, L. (2000). Glomerular filtration is required for transfection of proximal tubular cells in the rat kidney following injection of DNA complexes into the renal artery, Gene Ther. 7: 279-85.
30. Forrest, M. L., and Pack, D. W. (2002). On the kinetics of polyplex endocytic trafficking: implications for gene delivery vector design, Mol. Ther. 6: 57-66.
31. Freissler, E., Meyer auf der Heyde, A., David, G., Meyer, T. F., and Dehio, C. (2000). Syndecan-1 and syndecan-4 can mediate the invasion of OpaHSPG-expressing Neisseria gonorrhoeae into epithelial cells, Cell Microbiol. 2: 69-82.
32. Gautam, A., Densmore, C. L., Xu, B., and Waldrep, J. C. (2000). Enhanced gene expression in mouse lung after PEI-DNA aerosol delivery, Mol. Ther. 2: 63-70.
33. Goula, D., Remy, J. S., Erbacher, P., Wasowicz, M., Levi, G., Abdallah, B., and Demeneix, B. A. (1998). Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervous system, Gene Ther. 5: 712-7.
34. Goula, D., Benoist, C., Mantero, S., Merlo, G., Levi, G., and Demeneix, B. A. (1998). Polyethylenimine-based intravenous delivery of transgenes to mouse lung, Gene Ther. 5: 1291-5.
35. Goula, D., Becker, N., Lemkine, G. F., Normandie, P., Rodrigues, J., Mantero, S., Levi, G., and Demeneix, B. A. (2000). Rapid crossing of the pulmonary endothelial barrier by polyethylenimine/DNA complexes, Gene Ther. 7: 499-504.
36. Guissouma, H., Ghorbel, M. T., Seugnet, I., Ouatas, T., and Demeneix, B. A. (1998). Physiological regulation of hypothalamic TRH transcription in vivo is T3 receptor isoform specific, FASEB J. 12: 1755-64.
37. Guissouma, H., Dupre, S. M., Becker, N., Jeannin, E., Seugnet, I., Desvergne, B., and Demeneix, B. A. (2002). Feedback on hypothalamic TRH transcription is dependent on thyroid hormone receptor N terminus, Mol. Endocrinol. 16: 1652-66.
38. Haensler, J., and Szoka, F. C., Jr. (1993). Polyamidoamine cascade polymers mediate efficient transfection of cells in culture, Bioconjug. Chem. 4: 372-9.
39. Han, S., R.I. Mahato, and S.W. Kim, (2001) Water-soluble lipopolymer for gene delivery. Bioconjug. Chem. 12(3): p. 337-45.
40. Harbottle, R. P., Cooper, R. G., Hart, S. L., Ladhoff, A., McKay, T., Knight, A. M., Wagner, E., Miller, A. D., and Coutelle, C. (1998). An RGD-oligolysine peptide: a prototype construct for integrin-mediated gene delivery, Hum. Gene Ther. 9: 1037-47.
41. Hart, S. L., Collins, L., Gustafsson, K., and Fabre, J. W. (1997). Integrin-mediated transfection with peptides containing arginine-glycine-aspartic acid domains, Gene Ther. 4: 1225-30.
42. Horbinski, C., Stachowiak, M. K., Higgins, D., and Finnegan, S. G. (2001). Polyethyleneimine-mediated transfection of cultured postmitotic neurons from rat sympathetic ganglia and adult human retina, BMC Neurosci 2: 2.
43. Kataoka, K., Harada, A., and Nagasaki, Y. (2001). Block copolymer micelles for drug delivery: design, characterization and biological significance, Adv. Drug Deliv. Rev. 47: 113-31.
44. Kichler, A., Leborgne, C., Coeytaux, E., and Danos, O. (2001). Polyethylenimine-mediated gene delivery: a mechanistic study, J. Gene Med. 3: 135-44.
45. 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.
46. Kircheis, R., Blessing, T., Brunner, S., Wightman, L., and Wagner, E. (2001). Tumor targeting with surface-shielded ligand--polycation DNA complexes, J. Control Release 72: 165-70.
47. Koping-Hoggard, M., Tubulekas, I., Guan, H., Fdwards, K., Nilsson, M., Varum, K. M., and Artursson, P. (2001). Chitosan as a nonviral gene delivery system. Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo, Gene Ther. 8: 1108-21.
48. Kreiss, P., Cameron, B., Rangara, R., Mailhe, P., Aguerre-Charriol, O., Airiau, M., Scherman, D., Crouzet, J., and Pitard, B. (1999). Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency, Nucleic Acids Res. 27: 3792-8.
49. Kukowska-Latallo, J. F., Bielinska, A. U., Johnson, J., Spindler, R., Tomalia, D. A., and Baker, J. R., Jr. (1996). Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers, Proc. Natl. Acad. Sci. USA 93: 4897-902.
50. Labat-Moleur, F., Steffan, A. M., Brisson, C., Perron, H., Feugeas, O., Furstenberger, P., Oberling, F., Brambilla, E., and Behr, J. P. (1996). An electron microscopy study into the mechanism of gene transfer with lipopolyamines, Gene Ther. 3: 1010-7.
51. Lee, H., Jeong, J. H., and Park, T. G. (2001). A new gene delivery formulation of polyethylenimine/DNA complexes coated with PEG conjugated fusogenic peptide, J. Control Release 76: 183-92.
52. Lemkine, G. F., Goula, D., Becker, N., Paleari, L., Levi, G., and Demeneix, B. A. (1999). Optimisation of polyethylenimine-based gene delivery to mouse brain, J. Drug Target 7: 305-12.
53. Lemkine, G. F., Mantero, S., Migne, C., Raji, A., Goula, D., Normandie, P., Levi, G., and Demeneix, B. A. (2002). Preferential transfection of adult mouse neural stem cells and their immediate progeny in vivo with polyethylenimine, Mol. Cell Neurosci. 19: 165-74.
54. Lin, A. J., Slack, N. L., Ahmad, A., Koltover, I., George, C. X., Samuel, C. E., and Safinya, C. R. (2000). Structure and structure-function studies of lipid/plasmid DNA complexes, J. Drug Target 8: 13-27.
55. Lisziewicz, J., Gabrilovich, D. I., Varga, G., Xu, J., Greenberg, P. D., Arya, S. K., Bosch, M., Behr, J. P., and Lori, F. (2001). Induction of potent human immunodeficiency virus type 1-specific T-cell-restricted immunity by genetically modified dendritic cells, J. Virol. 75: 7621-8.
56. Ludtke, J. J., Zhang, G., Sebestyen, M. G., and Wolff, J. A. (1999). A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA, J. Cell Sci. 112: 2033-41.
57. Lukacs, G. L., Haggie, P., Seksek, O., Lechardeur, D., Freedman, N., and Verkman, A. S. (2000). Size-dependent DNA mobility in cytoplasm and nucleus, J. Biol. Chem. 275: 1625-9.
58. Martres, M. P., Demeneix, B., Hanoun, N., Hamon, M., and Giros, B. (1998). Up- and down-expression of the dopamine transporter by plasmid DNA transfer in the rat brain, Eur. J. Neurosci. 10: 3607-16.
59. Merdan, T., Kunath, K., Fischer, D., Kopecek, J., and Kissel, T. (2002). Intracellular processing of poly(ethylene imine)/ribozyme complexes can be observed in living cells by using confocal laser scanning microscopy and inhibitor experiments, Pharm. Res. 19 140-6.
60. Merlin, J. L., Dolivet, G., Dubessy, C., Festor, E., Parache, R. M., Verneuil, L., Erbacher, P., Behr, J. P., and Guillemin, F. (2001). Improvement of nonviral p53 gene transfer in human carcinoma cells using glucosylated polyethylenimine derivatives, Cancer Gene Ther. 8 203-10.
61. Mislick, K. A., and Baldeschwieler, J. D. (1996). Evidence for the role of proteoglycans in cation-mediated gene transfer, Proc. Natl. Acad. Sci. USA 93: 12349-54.
62. Mounkes, L. C., Zhong, W., Cipres-Palacin, G., Heath, T. D., and Debs, R. J. (1998). Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo, J. Biol. Chem. 273: 26164-70.
63. Neves, C., Byk, G., Scherman, D., and Wils, P. (1999). Coupling of a targeting peptide to plasmid DNA by covalent triple helix formation, FEBS Lett. 453: 41-5.
64. Nishikawa, M., and Huang, L, (2001). Nonviral vectors in the new millennium: delivery barriers in gene transfer, Hum. Gene Ther. 12: 861-70.
65. Pasqualini, R., Koivunen, E., and Ruoslahti, E. (1997). Alpha v integrins as receptors for tumor targeting by circulating ligands, Nat. Biolechnol. 15: 542-6.
66. Petersen, H., Fechner, P. M., Martin, A. L., Kunath, K., Stolnik, S., Roberts, C. J., Fischer, D., Davies, M. C., and Kissel, T. (2002). Polyethylenimine-graft-poly(ethylene glycol) copolymers: influence of copolymer block structure on DNA complexation and biological activities as gene delivery system, Bioconjug. Chem. 13: 845-54.
67. Pollard, H., Remy, J. S., Loussouam, G., Demolombe, S., Behr, J. P., and Escande, D. (1998). Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells, J. Biol. Chem. 273: 7507-11.
68. Pollard, H., Toumaniantz, G., Amos, J. L., Avet-Loiseau, H., Guihard, G., Behr, J. P., and Escande, D. (2001). Ca2+-sensitive cytosolic nucleases prevent efficient delivery to the nucleus of injected plasmids, J. Gene Med. 3: 153-64.
69. Poulain, L., Ziller, C., Muller, C. D., Erbacher, P., Bettinger, T., Rodier, J. F., and Behr, J. P. (2000). Ovarian carcinoma cells are effectively transfected by polyethylenimine (PEI) derivatives, Cancer Gene Ther. 7: 644-52.
70. Putnam, D., Gentry, C. A., Pack, D. W., and Langer, R. (2001). Polymer-based gene delivery with low cytotoxicity by a unique balance of side-chain termini, Proc. Natl. Acad. Sci. USA 98: 1200-5.
71. Reddy, J. A., Dean, D., Kennedy, M. D., and Low, P. S. (1999). Optimization of folate-conjugated liposomal vectors for folate receptor-mediated gene therapy, J. Pharm. Sci. 88: 1112-8.
72. Remy, J. S., Sirlin, C., Vierling, P., and Behr, J. P. (1994). Gene transfer with a series of lipophilic DNA-binding molecules, Bioconjug. Chem. 5: 647-54.
73. Remy, J. S., Kichler, A., Mordvinov, V., Schuber, F., and Behr, J. P. (1995). Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial viruses, Proc. Natl. Acad. Sci. USA 92 1744-8.
74. Rolland, A. P. (1998). From genes to gene medicines: recent advances in nonviral gene delivery, Crit. Rev. Ther. Drug Carrier Syst. 15: 143-918.
75. Rudolph, C., Lausier, J., Naundorf, S., Muller, R. H., and Rosenecker, J. (2000). In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers, J. Gene Med. 2: 269-78.
76. Sebestyen, M. G., Ludtke, J. J., Bassik, M. C., Zhang, G., Budker, V., Lukhtanov, E. A., Hagstrom, J. E., and Wolff, J. A. (1998). DNA vector chemistry: the covalent attachment of signal peptides to plasmid DNA, Nat. Biotechnol. 16: 80-5.
77. Su, H., Raymond, L., Rockey, D. D., Fischer, E., Hackstadt, T., and Caldwell, H. D. (1996). A recombinant Chlamydia trachomatis major outer membrane protein binds to heparan sulfate receptors on epithelial cells, Proc. Natl. Acad. Sci. USA 93: 11143-8.
78. Suh, W., Han, S. O., Yu, L., and Kim, S. W. (2002). An angiogenic, endothelial-cell-targeted polymeric gene carrier, Mol Ther 6: 664-72.
79. Summerford, C., and Samulski, R. J. (1998). Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions, J. Virol. 72: 1438-45.
80. Vernejoul, F., Faure, P., Benali, N., Calise, D., Tiraby, G., Pradayrol, L., Susini, C., and Buscail, L. (2002). Antitumor effect of in vivo somatostatin receptor subtype 2 gene transfer in primary and metastatic pancreatic cancer models, Cancer Res. 62: 6124-31.
81. Vinogradlov, S., Batrakova, E., Li, S., and Kabanov, A. (1999). Polyion complex micelles with protein-modified corona for receptor-mediated delivery of oligonucleotides into cells, Bioconjug. Chem. 10: 851-60.
82. Wang, S., Ma, N., Gao, S. J., Yu, H., and Leong, K. W. (2001). Transgene expression in the brain stem effected by intramuscular injection of polyethylenimine/DNA complexes, Mol. Ther. 3: 658-64.
83. Wightman, L., Kircheis, R., Rossler, V., Carotta, S., Ruzicka, R., Kursa, M., and Wagner, E. (2001). Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo, J. Gene Med. 3: 362-72.
84. Wolfert, M. A., Schacht, E. H., Toncheva, V., Ulbrich, K., Nazarova, O., and Seymour, L. W. (1996). Characterization of vectors for gene therapy formed by self-assembly of DNA with synthetic block co-polymers, Hum. Gene Ther. 7: 2123-33.
85. Woods, A., and Couchman, J. R. (1994). Syndecan 4 heparan sulfate proteoglycan is a selectively enriched and widespread focal adhesion component, Mol. Biol. Cell 5: 183-92.
86. Zabner, J., Fasbender, A. J., Moninger, T., Poellinger, K. A., and Welsh, M. J. (1995). Cellular and molecular barriers to gene transfer by a cationic lipid, J. Biol. Chem. 270: 18997-9007.
87. Zanta, M. A., Belguise-Valladier, P., and Behr, J. P. (1999). Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus, Proc. Natl. Acad. Sci. USA 96 91-6.
88. Zelphati, O., and Szoka, F. C., Jr. (1996). Mechanism of oligonucleotide release from cationic liposomes, Proc. Natl. Acad. Sci. USA 93: 11493-8.
89. Zou, S. M., Erbacher, P., Remy, J. S., and Behr, J. P. (2000). Systemic linear polyethylenimine (L-PEI)-mediated gene delivery in the mouse, J. Gene Med. 2: 128-34.
90. Zuber, G., Zammut-Italiano, L., Dauty, E., and Behr, J. P. (2003). Targeted Gene Delivery to Cancer Cells: Directed Assembly of Nanometric DNA Particles Coated with Folic Acid, Angew. Chem. Int. Ed. Engl. 42: 2666-2669.