Nonviral vectors for in vivo gene delivery: Physicochemical and pharmacokinetic considerations

Critical Reviews in Therapeutic Drug Carrier Systems

Volumn 14, Issue 2, 1997, Pages 133-172

  Mahato, Ram I ^a   Takakura, Yoshinobu ^a   Hashida, Mitsuru ^a  

^a KYOTO UNIVERSITY   (Japan)

Author keywords

gene delivery; liposomes; pharmacokinetics; plasmid DNA; polycation ligand conjugate

Indexed keywords

DRUG; LIPOSOME; POLYCATION;

DRUG ABSORPTION; DRUG DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; PARTICLE SIZE; REVIEW; TARGET CELL;

EID: 0030890713     PISSN: 07434863     EISSN: None     Source Type: Journal    
DOI: 10.1615/critrevtherdrugcarriersyst.v14.i2.20     Document Type: Review

References (177)

1. Wolf, J. A., Ed., Gene Therapeutics: Methods and Applications of Directed Gene Transfer, Birkhauser, Boston, 1994.
2. Lever, A. M. L. and Goodfellow, P., Eds., Br. Med. Bull.: Gene Therapy, Churchill Livingstone, London, 1995.
3. Berg, P. and Singer, M., Dealing with Genes: The Language of Heredity, University Science Books, Mill Valley, California, 1992.
4. Culver, K. W., Gene Therapy: A Handbook for Physicians, Mary Ann Liebert, New York, 1994.
5. Huber, B. E. and Lazo, J. S., Eds., Gene Therapy for Neoplastic Diseases, Ann. NY Acad. Sci., 716, 1, 1994.
6. Miller, N. and Vile, R., Review: Targeted vectors for gene therapy, FASEB J., 9, 190, 1995.
7. Uckert, W. and Walther, W., Retrovirus-mediated gene transfer in cancer therapy, Pharmacol. Ther., 63, 323, 1994.
8. Felgner, P. L. and Ringold, G. M., Cationic liposome-mediated transfection, Nature, 337, 387, 1989.
9. Felgner, P. L., Particulate systems and polymers for in vitro and in vivo delivery of polynucleotides, Adv. Drug Deliv. Rev., 5, 163, 1990.
10. Muller, A. D., Human gene therapy comes of age, Nature, 357, 455, 1992.
11. Rosenfeld, M. A., Yoshimura, K., Trapnell, B. C., Yoneyama, K., Rosenthal, E. R., Dalemans, W., Fukuyama, M., Bargon, J., Stier, L. E., Stratford-Perricaudet, L., In vivo gene transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium, Cell, 68, 143, 1992.
12. Soriano, P., Dijkstra, J., Legrand, A., Spanjer, H., Londos-Gagliardi, D., Roerdink, F., Scherhof, G., and Nicolau, C., Targeted and nontargeted liposomes for in vivo transfer to rat liver cells of a plasmid containing the preproinsulin I gene, Proc. Natl. Acad. Sci. USA, 80, 7128, 1983.
13. Nicolau, C., Legrand, A., and Grosse, E., Liposomes as carriers for in vivo gene transfer and expression, Meth. Enzymol., 149, 157, 1987.
14. Zhu, N., Liggitt, D., Liu, Y., and Debs, R., Systemic gene expression after intravenous DNA delivery into adult mice, Science, 261, 209, 1993.
15. Liu, Y., Liggitt, D., Zhong, W., Tu, G., Gaensler, K., and Debs, R., Cationic liposome-mediated intravenous gene therapy, J. Biol. Chem., 270, 24864, 1995.
16. Thierry, A. R., Lunardi-Iskandar, Y., Bryant, J. L., Rabinovich, P., Gallo, R. C., and Mahan, L. C., Systemic gene therapy: Biodistribution and long-term expression of transgene mice, Proc. Natl. Acad. Sci. USA, 92, 9742, 1995.
17. Brigham, K. L., Meyrick, B., Christman, B., Magnuson, M. A., King, G., and Berry, I. C., Rapid communications: In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle, Am. J. Med. Sci., 298, 278, 1989.
18. Brigham, K. L. and Schreier, H., Cationic liposomes and DNA delivery, J. Liposome Res., 3, 31, 1993.
19. Brigham, K. L., Meyrick, B., Christman, B., Conary, J. T., King, G., Berry, Jr., L. C., and Magnuson, M. A., Expression of human growth hormone fusion genes in cultured lung endothelial cells and in the lungs of mice, Am. J. Respir. Cell Mol. Biol., 8, 209, 1993.
20. Canonico, A. E., Conrary, J. T., Meyrick, B., and Brigham, K. L., Aerosol and intravenous transfection of human α1-antitrypsin gene to lungs of rabbits, Am. J. Resp. Cell Mol. Biol., 10, 24, 1994.
21. Hashida, M. and Takakura, Y., Pharmacokinetics in design of drug delivery systems, J. Control. Rel., 31, 163, 1994.
22. Takakura, Y., Fujita, T., Furitsu, H., Nishikawa, M., Sezaki, H., and Hashida, M., Pharmacokinetics of succinylated proteins and dextran sulfate in mice: Implications for hepatic targeting of protein drugs by direct succinylation via scavenger receptors, Int. J. Pharm., 105, 19, 1994.
23. Nishikawa, M., Ohtsubo, Y., Ohno, J., Fujita, T., Koyama, Y., Yamashita, F., Hashida, M., and Sezaki, H., Pharmacokinetics of receptor-mediated hepatic uptake of glycosylated albumin in mice, Int. J. Pharm., 85, 75, 1992.
24. Takino, T., Nakajima, C., Takakura, Y., Sezaki, H., and Hashida, M., Controlled bio-distribution of highly lipophilic drugs with various parenteral formulations, J. Drug Target., 1, 117, 1993.
25. Miyao, T., Takakura, Y., Akiyama, T., Yoneda, F., Sezaki, H., and Hashida, M., Stability and pharmacokinetics of oligonucleotides modified at terminal linkages in mice, Antisense Res. Develop., 5, 115, 1995.
26. Miyao, T., Takakura, Y., and Hashida, M., Stability and in vivo disposition characteristics of oligonucleotides and oligonucleotide conjugate with macromolecules, Proceedings of the International Symposium on Controlled Release of Bioactive Material, 20, 492, 1993.
27. Takakura, Y., Miyao, T., Kawabata, K., and Hashida, M., Control of in vivo characteristics of gene and antisense DNA disposition, Jpn. J. Cancer Chemother., 21, 320, 1994.
28. Nishida, K. M., Mihara, K., Takino, T., Nakane, S., Takakura, Y., Hashida, M., and Sezaki, H., Hepatic disposition characteristics of electrically charged macromolecules in rat in vivo and in the perfused liver, Pharm. Res., 8, 237, 1991.
29. Kawabata, K., Takakura, Y., and Hashida, M., The fate of plasmid DNA after intravenous injection in mice: Involvement of scavenger receptors in its hepatic uptake, Pharm. Res., 12, 825, 1995.
30. Yoshida, M., Mahato, R. I., Kawabata, K., Takakura, Y., and Hashida, M., Disposition characteristics of plasmid DNA in the single-pass rat liver perfusion. Pharm. Res. 13, 597, 1996.
31. Mahato, R. I., Kawabata, K., Takakura, Y., and Hashida, M., In vivo disposition characteristics of plasmid DNA completed with cationic liposomes, J. Drug Target., 3, 149, 1995.
32. Mahato, R.I., Kawabata, K., Nomura, T., Takakura, Y., and Hashida, M., Physico-chemical and pharmacokinetic characteristics of plasmid DNA completed with cationic liposomes, J. Pharm. Sci., 84, 1267, 1995.
33. Nicolau, C., Pape, A. L., Soriano, P., Fagette, F., and Juhel, M.-F., In vivo expression of rat insulin after intravenous administration of the liposome-entrapped gene for rat insulin I, Proc. Natl. Acad. Sci. USA, 80, 1068, 1983.
34. Wu, G. Y. and Wu, C. H., Receptor-mediated gene delivery and expression in vivo, J. Biol. Chem., 263, 14621, 1988.
35. Wilson, J. M., Grossman, M., Wu, C. H., Chowdhury, N. R., Wu, G. Y., and Chowdhury, J. R., Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesteromia in low density lipoprotein receptor-deficient rabbits, J. Biol. Chem., 267, 963, 1992.
36. Stankovics, J., Crane, A. N., Andrews, E., Wu, C. H., Wu, G. Y., and Ledley, F. D., Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes, Hum. Gene Ther., 5, 1095, 1994.
37. Ohkouchi, K., Imoto, H., Takakura, Y., Hashida, M., and Sezaki, H., Disposition of anticancer drugs after bolus arterial administration in a tissue-isolated tumor perfusion system, Cancer Res., 50, 1640, 1990.
38. Imoto, H., Sakurama, Y., Ohkouchi, K., Atsumi, R., Takakura, Y., Sezaki, H., and Hashida, M., Disposition characteristics of macromolecules in the perfused tissue-isolated tumor preparation, Cancer Res., 52, 4396, 1992.
39. Nomura, T., Yamashita, F., Takakura, Y., and Hashida, M., Pharmacokinetic analysis of various lipid carrier systems after intratumoral injection in tissue-isolated tumors, Proceedings of the International Symposium on Controlled Release of Bioactive Material, 22, 420, 1995.
40. Nomura, T., Yamashita, F., Takakura, Y., and Hashida, M., Effect of cationic liposomes on intratumoral behavior of gene medicines after local injection, 54th Annual Meeting of Japanese Cancer Association, 695, 1995.
41. Ledley, F. D., Pharmacokinetic considerations in the use of genes as pharmaceuticals, in Wolf, J. A., Ed., Gene Therapeutics: Methods and Applications of Directed Gene Transfer, Birkhauser, Boston, 235, 1994.
42. Ledley, T. S. and Ledley, F. D, Multicompartment, numerical model of cellular events in the pharmacokinetics of gene therapies, Hum. Gene Ther., 5, 679, 1994.
43. Capecchi, M. R., High efficiency transformation by direct microinjection of DNA into cultured mammalian cells, Cell, 22, 479, 1980.
44. Machy, P., Lewis, F., McMillan, L., and Jonak, Z. L., Gene transfer from targeted liposomes to specific lymphoid cells by electroporation, Proc. Natl. Acad. Sci. USA, 85, 8027, 1988.
45. Keating, A. and Toneguzzo, F., Gene transfer by electroporation: A model for gene therapy, Prog. Clin. Biol. Res., 333, 491, 1990.
46. Yang, N.-S., Burkholder, J., Roberts, B., Martinell, B., and McCabe, D., In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment, Proc. Natl. Acad. Sci. USA, 87, 9568, 1990.
47. Chen, C. A. and Okayama, H., High-efficiency transformation of mammalian cells by plasmid DNA, Mol. Cell Biol., 7, 2745, 1987.
48. Chen, C. A. and Okayama, H., Calcium phosphate mediated gene transfer: A highly efficient transfection system for stable transforming cells with plasmid, DNA, 6, 632, 1988.
49. Lapata, M. L., Cleveland, D. W., and Sollner-Webbs, B., High level transient expression of a chloramphenicol acetyltransferase gene by a DEAE-dextran mediated DNA transfection coupled with a dimethyl sulfoxide or glycerol shock treatment, Nucleic Acids Res., 12, 5707, 1984.
50. Takai, T. and Ohmori, H., DNA transfection of mouse lymphoid cells by the combination of DEAE-dextran mediated DNA uptake and osmotic shock procedure, Biochim. Biophys. Acta., 1048, 205, 1990.
51. Rupprecht, A. P. and Coleman, D. L., Transfection of adherent murine peritoneal macrophages with a reporter gene using DEAE-dextran, J. Immunol. Meth., 144, 157, 1991.
52. Hodgson, C. P, The vector void in gene therapy, Biotechnol., 13, 222, 1995.
53. Felgner, P. L., Gadek, T. R., Holm, M., Roman, R., Chan, H. W., Wenz, M., Northrop, J. P., Ringold, G. M., and Danielsen, M., Lipofectin: A highly efficient, lipid-mediated DNA-transfection procedure, Proc. Natl. Acad. Sci. USA, 84, 7413, 1987.
54. Wu, G. Y. and Wu, C. H., Receptor-mediated in vitro gene transformation by a soluble DNA carrier system, J. Biol. Chem., 262, 4429, 1987.
55. Wu, G. Y. and Wu, C. H., Evidence for targeted gene delivery to Hep G2 hepatoma cells in vitro, Biochemistry, 27, 887, 1988.
56. Wu, G. Y. and Wu, C. H., Liver-directed gene delivery, Adv. Drug Deliv. Rev., 12, 159, 1993.
57. Szoka, Jr., F., and Papahadjopoulos, D., Procedures for preparation of liposomes with large internal aqueous space and high capture by reverse-phase evaporation, Proc. Natl. Acad. Sci. USA, 75, 4194, 1978.
58. Fraley, R., Subramani, S., Berg, P., and Papahadjopoulos, D., Introduction of liposome-encapsulated SV40 DNA into cells, J. Biol. Chem., 255, 10431, 1980.
59. Mannino, R. J. and Gould-Fogerite, S., Liposome mediated gene transfer, Biotechniques, 6, 682, 1988.
60. Smith, J. G., Walzern, R. L., and German, J. B., Liposomes as agents of DNA transfer, Biochim. Biophys. Acta, 1154, 327, 1993.
61. Huang, A., Huang, L., and Kennel, S. J., Monoclonal antibody covalently coupled with fatty acid, J. Biol Chem, 255, 8015, 1980.
62. Litzinger, D. C. and Huang, L., Phosphatidylethanolamine liposomes: Drug delivery, gene transfer and immunodiagnostic applications, Biochim. Biophys. Acta, 1113, 201, 1992.
63. Wang, C. Y. and Huang, L., pH-sensitive immunoliposome mediated target cell-specific delivery and controlled expression of a foreign gene in mouse, Proc. Natl. Acad. Sci. USA, 84, 7851, 1987.
64. Zhou, X., Klibanov, A. L., and Huang, L., Improved encapsulation of DNA in pH-sensitive liposomes for transfection, J. Liposome Res., 2, 125, 1992.
65. Legendre, J. Y. and Szoka, Jr., F. C., Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: Comparison with cationic liposomes, Pharm. Res., 9, 1235, 1992.
66. Yagi, K., Noda, H., Kurono, M., and Ohishi, N., Efficient gene transfer with less cytotoxicity by means of cationic multilamellar liposomes, Biochem. Biophys. Res. Commun., 196, 1042, 1993.
67. Kojima, H., Ohishi, N., Takamori, M., and Yagi, K., Cationic multilamellar liposome-mediated gene transfer into primary myoblasts, Biochem. Biophys. Res. Commun., 207, 8, 1995.
68. Mizuno, M., Yoshida, J., Sugita, K., Inoue, I., Seo, H., Hayashi, Y., Koshizaka, T., and Yagi, K., Growth inhibition of glioma cells transfected with the human β-interferon gene by liposomes coupled with a monoclonal antibody, Cancer Res., 50, 7826, 1990.
69. Mizuno, M., Yoshida, J., Takaoka, T., and Sugita, K., Liposomal transfection of human γinterferon gene into human glioma cells and adoptive immunotherapy using lymphokine-activated killer cells, Neurosurgery, 80, 510, 1994.
70. Rose, J. K., Buonocore, L., and Whitt, M. A., A new cationic liposome reagent mediating nearly quantitative transfection of animal cells, Biotechniques, 10, 520, 1991.
71. Behr, J., Demeneix, B., Loeffler, J., Perez-Mutul, J., Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA, Proc. Natl. Acad. Sci. USA, 86, 6982, 1989.
72. Hawley-Nelson, P., Ciccarone, V., Gebeyehu, G., Jessee, J., and Feigner, P. L., Lipofectamine reagent: A new, higher efficiency polycationic liposome transfection reagent, Focus, 15, 73, 1993.
73. Leventis, R. and Silvius, J. R., Interactions of mammalian cells with dispersions containing novel metabolizable cationic amphiphiles, Biochim. Biophys. Acta, 1023, 124, 1990.
74. Gao, X. and Huang, L., A novel cationic liposome reagent for efficient transfection of mammalian cells, Biochem. Biophys. Res. Commun., 179, 280, 1991.
75. Felgner, J. H., Kumar, R., Sridhar, C. N., Wheeler, C. J., Tsai, Y. J., Border, R., Ramsey, P., Martin, M., and Feigner, P. L., Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations, J. Biol. Chem., 269, 2550, 1994.
76. Hug, P., and Sleight, R. G., Liposomes for the transformation of eukaryotic cells, Biochim. Biophys. Acta, 1097, 1, 1991.
77. Brigham, K. L., Meyrick, B., Christman, B., Berry, L. C., and King, G., Expression of a prokaryotic gene in cultured lung endothelial cells after lipofection with a plasmid vector, Am. J. Resp. Cell Mol. Biol., 1, 95, 1989.
78. Brigham, K. L., Meyrick, B., Christman, B., Conrary, J. T., King, G., Berry, I. C., and Magnuson, M. A., Expression of human growth hormone fusion genes in culture lung endothelial cells and in mice, Am. J. Resp. Cell Mol. Biol., 8, 209, 1993.
79. Jarnagin, W. R., Debs, R. J., Wang, S.-S., and Bissell, D., M., Cationic lipid-mediated transfection of liver cells in primary cultures, Nucleic Acids Res., 20, 4205, 1992.
80. Watanabe, Y., Nomoto, H., Takezawa, R., Miyoshi, N., and Akaiki, T., Highly efficient transfection into primary cultured mouse hepatocytes by use of cation-liposomes: An application for immunization, J. Biochem., 116, 1220, 1994.
81. Gao, X. and Huang, L., Cationic liposomes and polymers for gene transfer, J. Liposome Res., 3, 17, 1993.
82. Caplen, N. J., Kinrade, E., Sorgi, F., Gao, X., Gruenert, D., Geddes, D., Couttelle, C., Huang, L., Alton, E. W. F. W., and Williamson, R., In vitro liposome-mediated DNA transfection of epithelial cell lines using the cationic liposome DC-Chol/DOPE, Gene Ther., 2, 603, 1995.
83. Loeffler, J.-P. and Behr, J.-P., Gene transfer into primary and established mammalian cell lines with lipopolyamine-coated DNA, Methods Enzymol., 217, 599, 1993.
84. Barthel, F., Remy, J.-S., Loeffler, J.-P., and Behr, J.-P., Gene transfer optimization with lipospermine-coated DNA, DNA Cell Biol., 12, 553, 1993.
85. Demeneix, B. A., Abdel-Taweb, H., Benoist, C., Seugnet, I., and Behr, J.-P., Temporal and spatial expression of lipospermine-compacted genes transferred into chick embryos in vivo, Biotechniques, 16, 496, 1994.
86. Remy, J.-S., Sirlin, C., Vierling, P., and Behr, J.-P., Gene transfer with a series of lipophilic DNA-binding molecules, Bioconjugate Chem., 5, 647, 1994.
87. Remy, J.-S., Kichler, A., Mordvinov, V., Schuber, F., and Behr, J.-P., 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, 1995.
88. Kichler, A., Remy, J.-S., Boussif, O., Frisch, B., Boeckler, C., Behr, J.-P., and Schuber, F., Efficient gene delivery with neutral complexes of lipospermine and thiol-reactive phopholipids, Biochem. Biophys. Res. Commun., 209, 444, 1995.
89. Findeis, M. A., Wu, C. H., and Wu, G. Y., Ligand-based carrier systems for delivery of DNA to hepatocytes, Methods Enzymol., 247, 341, 1994.
90. Chen, J., Stickles, R. J., and Daichendt, K. A., Galactosylated histone-mediated gene transfer and expression, Hum. Gene Ther., 5, 429, 1994.
91. Wagner, E., Zenke, M., Cotten, M., Beug, H., and Birnsteil, M. L., Transferrin-polycation conjugates as carriers for DNA uptake into cells, Proc. Natl. Acad. Sci. USA, 87, 3410, 1990.
92. Cotten, M., Laengle-Ranault, F., Kirlappos, H., Wagner, E., Mechtler, K., Zenke, M., Berg, H., and Birnsteil, M. L., Transferrin-polycation mediated introduction of DNA into human leukemic cells: Stimulation agents that affect survival of transfected DNA or modulate transferrin receptor levels, Proc. Natl. Acad. Sci. USA, 87, 4033, 1991.
93. Cotten, M. and Wagner, E., Nonviral approaches to gene therapy, Curr. Opin. Biotechnol., 4, 705, 1993.
94. Wagner, E., Gotten, M., Mechtler, K., Kirlappos, H., and Birnstiel, M. L., DNA-binding transferrin conjugates as functional gene delivery agents: Synthesis by linkage of polylysine or ethidium homodimer to the transferrin carbohydrate moiety, Bioconjugate Chem., 2, 76, 1991.
95. Huckett, B., Ariatti, M., and Hawtrey, A. O., Evidence for targeted gene transfer by receptor-mediated endocytosis: Stable expression following insulin-directed entry of NEO in HepG2 cells, Biochem. Pharmacol., 40, 253, 1990.
96. Rosenkranz, A. A., Yachmenev, S. V., Jans, D. A., Serebryakova, N. V., Muravev, V. I., Peters, R., and Sobolev, A. S., Receptor-mediated endocytosis and nuclear transport of a transfecting DNA construct, Exp. Cell Res., 199, 323, 1992.
97. Trubetskoy, V. S., Torchilin, V. P., Kennel, S., and Huang, L., Use of N-terminal modified poly-L-lysine -antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells, Bioconjugate Chem., 3, 323, 1992.
98. Chen, J., Gamou, S., Takayanagi, A., and Shimizu, N., A novel gene delivery system using EGF receptor-mediated endocytosis, FEBS Letters, 338, 167, 1994.
99. Cheng, P.-W. and Yin, W., Lectin conjugate-directed gene transfer to airway epithelial cells, Biochem. Biophys. Res. Commun., 205, 826, 1994.
100. Perales, J. C., Ferkol, T., Molas, M., and Hanson, R. W., An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes, Eur. J. Biochem., 226, 255, 1994.
101. McKee, T. D., DeRome, M. E., Wu, G. Y., and Findeis, M. A., Preparation of asia-loorosomucoid-polylysine conjugates, Bioconjugate Chem., 5, 306, 1994.
102. Ferkol, T., Lindberg, G. L., Chen, J., Perales, J. C., Crawford, D. R., Ratnoflf, O. D., and Hanson, R. W., Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer, FASEB J., 7, 1081, 1993.
103. Midoux, P., Mendes, C., Legrand, A., Raimond, J., Mayer, R., Monsigny, M., and Roche, A. C., Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells, Nucleic Acids Res., 21, 871, 1993.
104. Breeuwer, M. and Goldfarb, D., Facilitated nuclear transport of histone H1 and other small nucleophilic proteins, Cell, 60, 999, 1990.
105. Plank, C., Wagner, E., Cotten, M., Mechtler, K., and Wagner, E., Gene transfer into hepatocytes using asialoglycoprotein receptor-mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand, Bioconjugate Chem., 3, 533, 1992.
106. Wagner, E., Cotten, M., Foisner, R., and Birnstiel, M. L., Transferrin-polycation-DNA complexes: The effect of polycations on the structure of the complex and DNA delivery to cells, Proc. Natl. Acad. Sci. USA, 88, 4255, 1991.
107. Strydom, S., Jaarsveld, P. V., Helden, E. V., Ariatti, M., and Hawtrey, A., Studies on the transfer of DNA into cells through use of avidin-polylysine conjugates complexed to biotinylated transferrin and DNA, J. Drug Target., 1, 165, 1993.
108. Shoeman, R., Joubert, D., Ariatti, M., and Hawtrey, A., Further studies on targeted DNA transfer to cells using a highly efficient delivery system of biotinylated transferrin and biotinylated polylysine complexed to streptavidin, J. Drug Target., 2, 509, 1995.
109. Trabetskoy, V. S., Torchilin, V. P., Kennel, S., and Huang, L., Cationic liposomes enhance targeted delivery and expression of exogenous DNA mediated by N-terminal modified poly-L-lysine -antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells, Biochem. Biophys. Acta, 1131, 311, 1992.
110. Behzadian, M. A. and Shimizu, N., Monoclonal antibody that immunoreacts with a sub-class of human receptors for epidermal growth factor, Cell Struct. Funct., 10, 219, 1985.
111. Ozawa, S., Ueda, M., Ando, N., Abe, O., Minoshima, S., and Shimizu, N., Selective killing of squamous carcinoma cells by an immunotoxin that recognizes the EGF receptor, Int. J. Cancer, 43, 152, 1989.
112. Hirota, N., Ueda, M., Ozawa, S., Abe, O., and Shimizu, N., Suppression of an epidermal growth factor receptor-hyperproducing tumor by an immunotoxin conjugate of gelonin and a monoclonal anti-epidermal growth factor receptor antibody, Cancer Res., 49, 7106, 1989.
113. Buschle, M., Cotten, M., Kirlappos, H., Mechtler, K., Schaffner, G., Zauner, W., Birnstiel, M. L., and Wagner, E., Receptor-mediated gene transfer into human T lymphocytes via binding of DNA/CD3 antibody particles to the CD3 T cell receptor complex, Hum. Gene Ther., 6, 753, 1995.
114. Ferkol, T., Kaetzel, C. S., and Davis, P. B., Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor, J. Clin. Invest., 92, 2394, 1993.
115. Sharon, N. and Lis, H., Lectins as cell recognizing molecules, Science, 246, 227, 1989.
116. Cerami, C., Frevert, U., Sinnis, P., Takacs, B., and Nussenzweig, V., Rapid clearance of malaria circumsporozoite protein (CS) by hepatocytes, J. Exp. Med., 179, 695, 1994.
117. Ding, Z.-M., Cristiano, R. J., Roth, J. A., Takacs, B., and Kuo, M. T., Malarial circum-sporozoite protein is a novel gene delivery vehicle to primary hepatocyte cultures and cultured cells, J. Biol Chem., 270, 3667, 1995.
118. Zhou, X., Klibanov, A. L., and Huang, L., Lipophilic polylysines mediate efficient transfection of mammalian cells, Biochim. Biophys. Acta, 1065, 8, 1991.
119. Zhou, X. and Huang, L., DNA transfection mediated by cationic liposomes containing lipopolylysine: Characterization and mechanism of action, Biochim. Biophys. Acta., 1189, 195, 1992.
120. Ledley, F. D., Nonviral gene therapy: The promise of genes as pharmaceutical products, Hum. Gene Ther., 6, 1129, 1995.
121. Nabel, E. G., Gordon, D., Yang, Z. Y., Xu, L., San, H., Plautz, G. E., Wu, B. Y., Gao, X., Huang, L., and Nabel, E. G., Gene transfer in vivo with DNA-liposome complexes: Lack of autoimmunity and gonadal localization, Hum. Gene Ther., 3, 649, 1992.
122. Stewart, M. J., Plautz, G. E., Buono, L. D., Yang, Z. Y., Xu, L., Gao, X., Huang L., Nabel, E. G., and Nabel, G. J., Gene transfer in vivo wth DNA-liposome complexes: Safety and acute toxicity in mice, Hum. Gene Ther., 3, 267, 1992.
123. San, H., Yang, Z. Y., Pompili, V. J., Jaffe, M. L., Plautz, G. E., Xu, L., Feigner, J. H., Wheeler, C. J., Feigner, P. L., Gao, X., and Nabel, G. J., Safety and short-term toxicity of a novel cationic lipid formulation for human gene therapy, Hum. Gene Ther., 4, 781, 1993.
124. Canonico, A. E., Plitman, J. D., Conary, J. T., Meyrick, B. O., and Brigham, K. L., No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes, J. Appl. Physiol, 77, 415, 1994.
125. Wu, G. Y., Wilson, J. M., Shalaby, F., Grossman, M., Shafritz, D. A., and Wu, C. H., Receptor-mediated gene delivery in vivo: Partial correlation of genetic analbuminemia in nagase rats, J. Biol. Chem., 266, 14338, 1991.
126. Wu, C. H., Wilson, J. M., and Wu, G. Y., Targeting genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo, J. Biol. Chem., 264, 16985, 1989.
127. Chowdhury, N. R., Wu, C. H., Wu, G. Y., Yerneni, P. C., Bommineni, V. R., and Chowdhury, J. R., Fate of DNA targeted to the liver by asialoglycoprotein receptor-mediated endocytosis in vivo: Prolonged persistence in cytoplasmic vesicles after partial hepatectomy, J. Biol. Chem., 268, 11265, 1993.
128. Frese, Jr., J., Wu, C. H., and Wu, G. Y., Targeting of genes to the liver with glycoprotein carriers, Adv. Drug Del. Rev., 14, 137, 1994.
129. Perales, J. C., Ferkol, T., Beegen, H., Ratnoff, O. D., and Hanson, R. W., 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, 1994.
130. Ferkol, T., Perales, J. C., Eckman, E., Kaetzel, C. S., Hanson, R. W., and Davis, P. B., Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor, J. Clin. Invest., 95, 493, 1995.
131. Kaneda, Y., Iwai, K., and Uchida, T., Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science, 243, 375, 1989.
132. Kaneda, Y., Iwai, K., and Uchida, T., Introduction and expression of the human insulin gene in adult rat liver, J. Biol. Chem., 264, 12126, 1989.
133. Kato, K., Nakanishi, M., Kaneda, Y., Uchida, T., and Okada, Y., Expression of hepatitis B virus surface antigen in adult rat liver, J. Biol. Chem., 266, 3361, 1991.
134. Kato, K., Kaneda, Y., Sakurai, M., Nakanishi, M., and Okada, Y., Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats, J. Biol. Chem., 266, 22071, 1991.
135. Tomita, N., Higaki, J., Morishita, R., Kato, K., Mikami, H., Kaneda, Y., and Ogihara, T., Direct in vivo gene introduction into rat kidney, Biochem. Biophys. Commun., 186, 129, 1992.
136. Leyen, H. E. V. D., Gibbons, G. H., Morishita, R., Lewis, N. P., Zhang, L., Nakajima, M., Kaneda. Y., Cooke, J. P., and Dzau, V. J., Gene therapy inhibiting neointimal vascular lesion: In vivo transfer of endothelial cell nitric oxide synthase gene. Proc. Natl. Acad. Sci. USA, 92, 1137, 1995.
137. Schofield, J. P., and Caskey, C. T., Non-viral approaches to gene therapy, Br. Med. Bull., 51, 56, 1995.
138. Kaneda, Y., Kato, K., Nakanishi, M., and Uchida, T., Introduction of plasmid DNA and nuclear protein into cells by using erythrocyte ghosts, liposomes, and Sendai virus, Methods Enzymol., 221, 317, 1993.
139. Nabel, E. G., Shum, L., Pompili, V. J., Yang, Z.-Y., San, H., Shu, H. B., Liptay, S., Gold, L., Gordon, D., Derynck, R., and Nabel, G. J., Direct transfer of transforming growth factor β1 gene into arteries stimulates fibrocellular hyperplasia, Proc. Natl. Acad. Sci. USA, 90, 10759, 1993.
140. Nabel, E. G., Yang, Z., Liptay, S., San, H., Gordon, D., Haudenschild, C. C., and Nabel, G. J., Recombinant platelet-derived growth factor B gene expression in porcine arteries induce intimal hyperplasia in vivo, J. Clin. Invest., 91, 1822, 1993.
141. Nabel, E. G., Yang, Z., Plautz, G., Forough, R., Zhan, X., Haudenschild, C. C., Mciag, T., and Nabel, G. J., Recombinant fibroblast growth factor-1 promoters intimal hyperplasia and angiogenesis in arteries in vivo, Nature, 362, 844, 1993.
142. Plautz, G. E., Nabel, E. G., and Nabel, G. J., Liposome mediated gene transfer into vascular cells, J. Liposome Res., 3, 179, 1993.
143. Wolf, J. A., Malone, R. W., Williams, P., Chong, W., Acsadi, G., Jani, A., and Feigner, P. L., Direct gene transfer into mouse muscle in vivo, Science, 247, 1465, 1990.
144. Acsadi, G., Jiao, S., Jani, A., Duke, D., Williams, P., Chong, W., and Wolf, J., Direct gene transfer and expression into rat heart in vivo, New Biol., 3, 71, 1991.
145. Wolf, J. A., Ludtke, J. J., Acsadi, G., Williams, P., and Jani, A., Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle, Hum. Mol. Genet., 1, 363, 1992.
146. Danko, I., Fritz, J. D., Jiao, S., Hogan, K., Latendresse, J. S., and Wolff, J. A., Pharmacological enhancement of in vivo gene expression in muscle, Gene Ther., 1, 114, 1994.
147. Buttrick, P. M., Kass, A., Kitsis, R. N., Kaplan, M. L., and Leinwand, L. A., Behavior of gene directly injected into the rat heart in vivo, Circ. Res., 70, 193, 1992.
148. Davis, H. L., Michel, M. L., Mancini, M., Schleef, M., and Whale, R. G., Direct gene transfer in skeletal muscle: Plasmid DNA-based immunization against the hepatitis B virus surface antigen, Vaccine, 12, 1503, 1994.
149. Wells, D. J. and Goldspink, G., Age and sex influence expression of plasmid DNA directly injected into mouse skeletal muscle, FEBS Lett., 306, 203, 1992.
150. Nakanishi, M., Gene introduction into animal tissues, Crit. Rev. Ther. Drug Carrier Syst., 12, 263, 1995.
151. Sikes, M., O'Malley, Jr., B. W., Finegold, M. J., and Ledley, F. D., In vivo gene transfer into rabbit thyroid follicular cells by direct DNA injection, Hum. Gene Ther., 5, 837, 1994.
152. Yovandich, J., O'Malley, Jr., B. W., Sikes, M., and Ledley, F. D., Gene transfer to synovial cells by intraarticular administration of plasmid DNA, Hum. Gene Ther., 6, 603, 1995.
153. Hickman, M. A., Malon, R. W., Lehmann-Bruinsoma, K., Sih, T. R., Knoell, D., Szoka, F. C., Walzern, R., Carlson, D. M., and Powell, J. S., Gene expression following direct injection of DNA into liver, Hum. Gene Ther., 5, 1477, 1994.
154. Davis, H. L., Whalen, R. G., and Demeneix, B. A., Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression, Hum. Gene Ther., 4, 151, 1993.
155. Nabel, G. J., Nabel, E. G., Yang, Z.-Y., Fox, B. A., Gao, X., Huang, L., Shu, S., Gordon, D., and Chang, A. E., Direct gene transfer with DNA/liposome complexes in melanoma: Expression, biologic activity, and lack of toxicity in humans, Proc. Natl. Acad. Sci. USA, 90, 11307, 1993.
156. Plautz, G. E., Yang, Z.-Y., Wu, B. Y., Gao, X., Huang, L., and Nabel, G. J., Immunotherapy of malignancy by in vivo gene transfer into tumors, Proc. Natl. Acad. Sci. USA, 90, 4645, 1993.
157. Vile, R. G. and Hart, I. R., In vitro and in vivo targeting of gene expression to melanoma cells, Cancer Res., 53, 962, 1993.
158. Kawabata, K., Takakura, Y., Watanabe, Y., and Hashida, M., IFN-γ gene transfer to mouse tumor cells with various cationic carriers for in vivo gene therapy, 54th Annual Meeting of the Japanese Cancer Association, 695, 1995.
159. Nomura, T., Takakura, Y., and Hashida, M., In vivo gene expression following direct injection of naked plasmid DNA into tumor, Proceedings of the International Symposium on Controlled Release of Bioactive Material, 23, 1996.
160. Yoshimura, K., Rosenfeld, M. A., Nakamura, H., Scherer, E. M., Pavirani, A., Lecocoq, J. P., and Crystal, R. G., Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer, Nucleic Acids Res., 20, 3233, 1992.
161. Meyer, K. B., Thompson, M. M., Levy, M. Y., and Szoka, Jr., F. C., Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics, Gene Ther., 2, 450, 1995.
162. Tsan, M.-F., White, J. E., and Shepard, B., Lung-specific direct in vivo gene transfer with recombinant plasmid DNA, Am. J. Physiol., 268, 12, L1052, 1995.
163. Stribling R., Brunette, E., Liggitt, D., Gaensler, K., and Debs, R., Aerosol gene delivery in vivo, Proc. Natl. Acad. Sci. USA, 89, 11277, 1992.
164. Philip, R., Liggitt, D., Philip, M., Dazin, P., and Debs, R., In vivo gene delivery: Efficient transfection of T lymphocytes in adult mice, J. Biol. Chem., 268, 16087, 1993.
165. Hofland, H. and Huang, L., Inhibition of human ovarian carcinoma cell proliferation by liposome-plasmid DNA complex. Biochem. Biophys. Res. Commun., 207, 492, 1995.
166. Zabner, J., Fasbender, A. J., Maninger, T., Poellinger, K. A., and Welsh, M. J., Cellular and molecular barriers to gene transfer by a cationic lipid, J. Biol. Chem., 270, 18997, 1995.
167. Olins, D. E., Olins, A. L., and Von Hippel, P. H., Model nucleoprotein complexes: Studies on the interaction of cationic homopolypeptides with DNA,J. Mol. Biol., 24, 157, 1967.
168. Petrak, K., Design and properties of particulate carriers for intravascular administration, in Rolland, A., Ed., Pharmaceutical Particulate Carriers: Therapeutic Applications, Marcel Dekker, New York, 1993, 225.
169. Takakura, Y., and Hashida, M., Delivery of plasmid DNA to endothelial cells, Eur. J. Pharm. Biopharm., 42, 269, 1996.
170. Nicolau, C. and Cudd, A., Liposomes as carriers of DNA, Crit. Rev. Ther. Drug Carrier Syst., 6, 239, 1989.
171. Schlepper-Schäfer, J., Hülsmann, D., Djovkar, A., Meyer, H. E., Herbertz, L., Kolb, H., and Kolb-Bachofen, V., Endocytosis via galactose receptors in vivo: Ligand size directs uptake by hepatocytes and/or liver macrophages, Exp. Cell Res., 165, 494, 1986.
172. Curiel, D. T., Agrawal, S., Wagner, E., and Cotten, M., Adenovirus enhancement of transferrin-polylysine-mediated gene delivery, Proc. Natl. Acad. Sci. USA, 88, 8850, 1991.
173. Cotten, M., Wagner, E., Zatloukal, K., Phillips, S., Curiel, D. T., and Birnstiel, M. L., High-efficiency receptor mediated delivery of small and large (48 kb) gene constructs using the endosome distruption activity of defective or chemically inactivated adenovirus particles,Proc. Natl. Acad. Sci. USA, 89, 6094, 1992.
174. Wagner, E., Zatloukal, F., Cotten, M., Kirlappos, H., Mechtler, K., Curiel, D. T., and Birnstiel, M. L., Coupling of adenovirus to transferrinpolylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes, Proc. Natl. Acad. Sci. USA, 89, 6099, 1992.
175. Cristiano, R. J., Smith, L. C., Kay, M. A., Brinkley, B. R., and Woo, S. L. C., Hepatic gene therapy: Efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex, Proc. Natl. Acad. Sci. USA, 90, 11548, 1993.
176. Curiel, D. T., High-efficiency gene transfer employing adenovirus-polylysine-DNA complex, Nat. Immunol., 13, 141, 1994.
177. Wagner, E., Plank, C., Zafloukal, K., Cotten, M., and Birnstiel, M. L., Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptide augment gene transfer by transferrin-polylysine-DNA complexes: Toward a synthetic virus-like gene transfer vehicle, Proc. Natl. Acad. Sci. USA, 89, 7934, 1992.