Advertisement

Nonviral Methods for Gene Transfer

  • A. Djeha
  • L. S. Lashford
Part of the Blood Cell Biochemistry book series (BLBI, volume 8)

Abstract

Nucleic acids have acquired considerable significance as clinical reagents in genetic diseases. In genetic approaches to human disease, genes are introduced into cells to synthesize therapeutically active products which may in turn influence the clinical progression of the disease. Successful gene therapy requires identifying an appropriate therapeutic gene and also developing delivery systems through which genes are efficiently transferred to the desired cell type. One approach is to use viruses which, through evolutionary pressures, have been selected to introduce genes successfully into mammalian cells. This is manifested by the development of sophisticated and highly specific mechanisms which facilitate viral cell binding to target cells, cellular entry, and survival, followed by rapid intracellular liberation of the viral contents and subsequent replication of the virus genome. Rapid progress in understanding the mechanisms of viral infection of cells has inspired the development of gene delivery systems that emulate many functions of viruses. However, the safety of any therapy is of paramount importance. The ideal agents for gene therapy are those which are unlikely to infect cells outside the target tissue and are also nontoxic and unable to induce a host immune response. Many viral vectors in clinical use have been chosen because they are efficient and relatively stable in gene transfer. However, there are significant concerns regarding the safety of virally mediated gene transfer. The report of a breakout of a replication-competent retrovirus in a producer cell line that is widely considered among the safest used in clinical trials has sent a strong warning signal (Chong and Vile, 1996).

Keywords

Gene Therapy Gene Transfer Gene Delivery Particle Bombardment Cationic Lipid 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  1. Acsadi, G., Dickson, G., Love, D. R., Jani, A., Walsh, F. S., Gurusinghe, A., Wolff, J. A., and Davies, K. E., 1991a, Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs, Nature 352:815–818.PubMedGoogle Scholar
  2. Acsadi, G., Jiao, S. S., Jani, A., Duke, D., Williams, P., Chong, W., and Wolff, J. A., 1991b, Direct gene transfer and expression into rat heart in vivo, New Biol. 3:71–81.PubMedGoogle Scholar
  3. Aliño, S. F., Bobadilla, M., Garcia Sanz, M., Lejarreta, M., Unda, F., and Hilario, E., 1993, In vivo delivery of human alpha 1-antitrypsin gene to mouse hepatocytes by liposomes, Biochem. Biophys. Res. Commun. 192:174–181.PubMedGoogle Scholar
  4. Aliño, S. F., Crespo, J., Bobadilla, M., Lejarreta, M., Blaya, C., and Crespo, A., 1994, Expression of human alpha 1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes, Biochem. Biophys. Res. Commun. 204:1023–1030.PubMedGoogle Scholar
  5. Alino, S. F., Mobadilla, M., Crespo, J., and Lejarreta, M., 1996, Human 1 antitrypsin gene transfer to in vivo mouse hepatocytes, Hum. Gene Ther. 7:531–536.PubMedGoogle Scholar
  6. Alton, E. W., Middleton, P. G., Caplen, N. J., Smith, S. N., Steel, D. M., Munkonge, F. M., Jeffery, P. K., Geddes, D. M., Hart, S. L., Williamson, R., et al., 1993, Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice, Nat. Genet. 5:135–142.PubMedGoogle Scholar
  7. Andreason, G. L., and Evans, G. A., 1989, Optimization of electroporation for transfection of mammalian cell lines, Anal. Biochem. 180:269–275.PubMedGoogle Scholar
  8. Baatz, J. E., Bruno, M. D., Ciraolo, P. J., Glasser, S. W., Stripp, B. R., Smyth, K. L., and Korfhagen, T. R., 1994, Utilization of modified surfactant-associated protein B for delivery of DNA to airway cells in culture, Proc. Natl. Acad. Sci. USA 91:2547–2551.PubMedGoogle Scholar
  9. Baeza, I., Gariglio, P., Rangel, L. M., Chavez, P., Cervantes, L., Arguello, C., Wong, C., and Montanez, C., 1987, Electron microscopy and biochemical properties of polyamine-compacted DNA, Biochemistry 26:6387–6392.PubMedGoogle Scholar
  10. Baru, M., Axelrod, J. H., and Nur, I., 1995, Liposome-encapsulated DNA-mediated gene transfer and synthesis of human factor IX in mice, Gene 161:143–150.PubMedGoogle Scholar
  11. 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 86:6982–6986.PubMedGoogle Scholar
  12. Benvenisty, N., and Reshef, L., 1986, Direct introduction of genes into rats and expression of the genes, Proc. Natl. Acad. Sci. USA 83:9551–9555.PubMedGoogle Scholar
  13. Böttger, M., Vogel, F., Platzer, M., Kiessling, U., Grade, K., and Strauss, M., 1988, Condensation of vector DNA by the chromosomal protein HMG1 results in efficient transfection, Biochim. Biophys. Acta 950:221–228.PubMedGoogle Scholar
  14. Boussif, O., Lezoulc’h, F., Zanta, M. A., Schermann, 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 92:7297–7301.PubMedGoogle Scholar
  15. Brigham, K. L., Meyrick, B., Christman, B., Magnuson, M., King, G., and Berry, L. C., Jr., 1989, In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle, Am. J. Med. Sci. 298:278–281.PubMedGoogle Scholar
  16. Budker, V., Zhang, G., Knechtle, S., and Wolff, J. A., 1996, Naked DNA delivered intraportally expresses efficiently in hepatocytes, Gene Ther. 3:593–598.PubMedGoogle Scholar
  17. Buschle, M., Cotten, M., Kirlappos, H., Mechtler, K., Schaffner, G., Zauner, W., Birnstiel, M. L., and Wagner, E., 1995, 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. 6:753–761.PubMedGoogle Scholar
  18. Canonico, A. E., Plitman, J. D., Conary, J. T., Meyrick, B. O., and Brigham, K. L., 1994, No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes, J. Appl. Physiol. 77:415–419.PubMedGoogle Scholar
  19. Cao, L., Zheng, Z. C., Zhao, Y. C., Jiang, Z. H., Liu, Z. G., Chen, S. D., Zhou, C. F., and Liu, X. Y., 1995, Gene therapy of Parkinson disease model rat by direct injection of plasmid DNA-lipofectin complex, Hum. Gene Ther. 6:1497–1501.PubMedGoogle Scholar
  20. Caplen, N. J., Alton, E. W., Middleton, P. G., Dorin, J. R., Stevenson, B. J., Gao, X., Durham, S. R., Jeffery, P. K., Hodson, M. E., Coutelle, C., et. al., 1995, Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis, Nat. Med. 1:39–46.PubMedGoogle Scholar
  21. Chen, J., Stickles, R. J., and Daichendt, K. A., 1994, Galactosylated histone-mediated gene transfer and expression, Hum. Gene Ther. 5:429–435.PubMedGoogle Scholar
  22. Cheng, S., Merlino, G. T., and Pastan, I. H., 1983, A versatile method for the coupling of protein to DNA: Synthesis of alpha 2-macroglobulin-DNA conjugates, Nucleic Acids Res. 11:659–669.PubMedGoogle Scholar
  23. Cheng, L., Ziegelhoffer, P. R., and Yang, N. S., 1993, In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment, Proc. Natl. Acad. Sci. USA 90:4455–4459.PubMedGoogle Scholar
  24. Chiou, H. C., Tangco, M. V., Levine, S. M., Robertson, D., Kormis, K., Wu, C. H., and Wu, G. Y., 1994, Enhanced resistance to nuclease degradation of nucleic acids complexed to asialoglycoprotein-polylysine carriers, Nucleic. Acids. Res. 22:5439–5446.PubMedGoogle Scholar
  25. Chong, H., and Vile, R. G., 1996, Replication-competent retrovirus produced by a’ split-function’ third generation amphotropic packaging cell line, Gene Ther. 3:624–629.PubMedGoogle Scholar
  26. Chowdhury, N. R., Wu, C. H., Wu, G. Y., Yemeni, P. C., Bommineni, V. R., and Chowdhury, J. R., 1993, 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. 268:11265–11271.PubMedGoogle Scholar
  27. Chowdhury, N. R., Hays, R. M., Bommineni, V. R., Franki, N., Chowdhury, J. R., Wu, C. H., and Wu, G. Y., 1996, Microtubular disruption prolongs the expression of human bilirubinuridinediphospho-glucuronate-glucuronosyltransferase-1 gene transferred into Gunn rat livers, J. Biol. Chem. 271:2341–2346.PubMedGoogle Scholar
  28. Christou, P., 1995, Particle bombardment, Methods Cell Biol. 50:375–382.PubMedGoogle Scholar
  29. Chu, G., Hayakawa, H., and Berg, P., 1987, Electroporation for the efficient transfection of mammalian cells with DNA, Nucleic Acids Res. 15:1311–1326.PubMedGoogle Scholar
  30. Cook, D. R., Maxwell, I. H., Glode, L. M., Maxwell, F., Stevens, J. O., Purner, M. B., Wagner, E., Curiel, D. T., and Curiel, T. J., 1994, Gene therapy for B-cell lymphoma in a SCID mouse model using an immunoglobulin-regulated diphtheria toxin gene delivered by a novel adenovirus-polylysine conjugate, Cancer Biother. 9:131–141.PubMedGoogle Scholar
  31. Cooper, M. J., 1996, Noninfectious gene transfer and expression systems for cancer gene therapy, Semin. Oncol. 23:172–187.PubMedGoogle Scholar
  32. Cotten, M., Langle Rouault, F., 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. USA 87:4033–4037.PubMedGoogle Scholar
  33. Cotten, M., Wagner, E., Zatloukal, K., Phillips, S., Curiel, D. T., and Birnstiel, M. L., 1992, High-efficiency receptor-mediated delivery of small and large (48 kilobase gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles, Proc. Natl. Acad. Sci. USA 89:6094–6098.PubMedGoogle Scholar
  34. Cotten, M., Wagner, E., and Birnstiel, M. L., 1993a, Receptor-mediated transport of DNA into eukaryotic cells, Methods Enzymol. 217:618–644.PubMedGoogle Scholar
  35. Cotten, M., Wagner, E., Zatloukal, K., and Birnstiel, M. L., 1993b, Chicken adenovirus (CELO virus) particles augment receptor-mediated DNA delivery to mammalian cells and yield exceptional levels of stable transformants, J. Virol. 67:3777–3785.PubMedGoogle Scholar
  36. Cotten, M., Baker, A., Saltik, M., Wagner, E., and Buschle, M., 1994a, Lipopolysaccharide is a frequent contaminant of plasmid DNA preparations and can be toxic to primary human cells in the presence of adenovirus, Gene Ther. 1:239–246.PubMedGoogle Scholar
  37. Cotten, M., Saltik, M., Kursa, M., Wagner, E., Maass, G., and Birnstiel, M. L., 1994b, Psoralen treatment of adenovirus particles eliminates virus replication and transcription while maintaining the endosomolytic activity of the virus capsid, Virology 205:254–261.PubMedGoogle Scholar
  38. Cox, G. J., Zamb, T. J., and Babiuk, L. A., 1993, Bovine herpesvirus 1: Immune responses in mice and cattle injected with plasmid DNA, J. Virol. 67:5664–5667.PubMedGoogle Scholar
  39. Cristiano, R. J., Smith, L. C., Kay, M. A., Brinkley, B. R., and Woo, S. L., 1993, Hepatic gene therapy: Efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex, Proc. Natl. Acad. Sci. USA 90:11548–11552.PubMedGoogle Scholar
  40. Curiel, D. T., Agarwal, S., Wagner, E., and Cotten, M., 1991, Adenovirus enhancement of transferrin-polylysine-mediated gene delivery, Proc. Natl. Acad. Sci. USA 88:8850–8854.PubMedGoogle Scholar
  41. Curiel, D. T., Wagner, E., Cotten, M., Birnstiel, M. L., Agarwal, S., Li, C. M., Loechel, S., and Hu, P. C., 1992, High-efficiency gene transfer mediated by adenovirus coupled to DNA-polylysine complexes, Hum. Gene Ther. 3:147–154.PubMedGoogle Scholar
  42. Curiel, D. T., 1994, High-efficiency gene transfer mediated by adenovirus-polylysine-DNA complexes, Ann. NY Acad. Sci. 716:36–56; discussion 56-38.PubMedGoogle Scholar
  43. Curiel, T. J., Cook, D. R., Bogedain, C., Jilg, W., Harrison, G. S., Cotten, M., Curiel, D. T., and Wagner, E., 1994, Efficient foreign gene expression in Epstein-Barr virus-transformed human B-cells, Virology 198:577–585.PubMedGoogle Scholar
  44. Danko, I., Fritz, J. D., Latendresse, J. S., Herweijer, H., Schultz, E., and Wolff, J. A., 1993, Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection, Hum. Mol. Genet. 2:2055–2061.PubMedGoogle Scholar
  45. Danko, I., Fritz, J. D., Jiao, S., Hogan, K., Latendresse, J. S., and Wolff, J. A., 1994, Pharmacological enhancement of in vivo foreign gene expression in muscle, Gene Ther. 1:114–121.PubMedGoogle Scholar
  46. Davis, H. L., and Jasmin, B. J., 1993, Direct gene transfer into mouse diaphragm, FEBS Lett. 333:146–150.PubMedGoogle Scholar
  47. Davis, H. L., Demeneix, B. A., Quantin, B., Coulombe, J., and Whalen, R. G., 1993a, Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle, Hum. Gene Ther. 4:733–740.PubMedGoogle Scholar
  48. Davis, H. L., Michel, M. L., and Whalen, R. G., 1993b, DNA-based immunization induces continuous secretion of hepatitis B surface antigen and high levels of circulating antibody, Hum. Mol. Genet. 2:1847–1851.PubMedGoogle Scholar
  49. Davis, H. L., Whalen, R. G., and Demeneix, B. A., 1993c, Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression, Hum. Gene Ther. 4:151–159.PubMedGoogle Scholar
  50. Davis, H. L., Michel, M. L., Mancini, M., Schleef, M., and Whalen, R. G., 1994, Direct gene transfer in skeletal muscle: Plasmid DNA-based immunization against the hepatitis B virus surface antigen, Vaccine 12:1503–1509.PubMedGoogle Scholar
  51. Davis, H. L., Schirmbeck, R., Reimann, J., and Whalen, R. G., 1995, DNA-mediated immunization in mice induces a potent MHC class I-restricted cytotoxic T lymphocyte response to the hepatitis B envelope protein, Hum. Gene Ther. 6:1447–1456.PubMedGoogle Scholar
  52. Ding, Z. M., Cristiano, R. J., Roth, J. A., Takacs, B., and Kuo, M. T., 1995, Malarial circumsporozoite protein is a novel gene delivery vehicle to primary hepatocyte cultures and cultured cells, J. Biol. Chem. 270:3667–3676.PubMedGoogle Scholar
  53. Dubensky, T. W., Campbell, B. A., and Villarreal, L. P., 1984, Direct transfection of viral and plasmid DNA into the liver or spleen of mice, Proc. Natl. Acad. Sci. USA 81:7529–7533.PubMedGoogle Scholar
  54. Dzau, V. J., Morishita, R., and Gibbons, G. H., 1993, Gene therapy for cardiovascular disease, Trends Biotechnol. 11:205–210.PubMedGoogle Scholar
  55. Egilmez, N. K., Cuenca, R., Yokota, S. J., Sorgi, F., and Bankert, R. B., 1996, In vivo cytokine gene therapy of human tumor xenogrates in SCID mice by liposome-mediated DNA delivery, Gene Ther. 3:607–614.PubMedGoogle Scholar
  56. Eisenbraun, M. D., Fuller, D. H., and Haynes, J. R., 1993, Examination of parameters affecting the elicitation of humoral immune responses by particle bombardment-mediated genetic immunization, DNA Cell. Biol. 12:791–797.PubMedGoogle Scholar
  57. 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.PubMedGoogle Scholar
  58. Farhood, H., Bottega, R., Epand, R. M., and Huang, L., 1992, Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity, Biochim. Biophys. Acta. 1111:239–246.PubMedGoogle Scholar
  59. Farhood, H., Gao, X., Son, K., Yang, Y. Y., Lazo, J. S., Huang, L., Barsoum, J., Bottega, R., and Epand, R. M., 1994, Cationic liposomes for direct gene transfer in therapy of cancer and other diseases, Ann. NY Acad. Sci. 716:23–34.PubMedGoogle Scholar
  60. Fasbender, A. J., Zabner, J., and Welsh, M. J., 1995, Optimization of cationic lipid-mediated gene transfer to airway epithelia, Am. J. Physiol. 269:L45–51.PubMedGoogle Scholar
  61. Fazio, V. M., Fazio, S., Rinaldi, M., Catani, M. V., Zotti, S., Ciafre, S. A., Seripa, D., Ricci, G., and Farace, M. G., 1994, Accumulation of human apolipoprotein-E in rat plasma after in vivo intramuscular injection of naked DNA, Biochem. Biophys. Res. Commun. 200:298–305.PubMedGoogle Scholar
  62. Felgner, P. L., Gadek, T. R., Holm, M., Roman, R., Chan, H. W., Wenz, M., Northrop, J. P., Ringold, G. M., and Danielsen, M., 1987, Lipofection: A highly efficient, lipid-mediated DNA-transfection procedure, Proc. Natl. Acad. Sci. USA 84:7413–7417.PubMedGoogle Scholar
  63. Feigner, J. H., Kumar, R., Sridhar, C. N., Wheeler, C. J., Tsai, Y. J., Border, R., Ramsey, P., Martin, M., and Feigner, P. L., 1994, Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations, J. Biol. Chem. 269:2550–2561.Google Scholar
  64. Feigner, P. L., Tsai, Y. J., Sukhu, L., Wheeler, C. J., Manthorpe, M., Marshall, J., and Cheng, S. H., 1995, Improved cationic lipid formulations for in vivo gene therapy, Ann. NY Acad. Sci. 772:126–139.Google Scholar
  65. Ferkol, T., Kaetzel, C. S., and Davis, P. B., 1993, Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor, J. Clin. Invest. 92:2394–2400.PubMedGoogle Scholar
  66. Ferkol, T., Perales, J. C., Eckman, E., Kaetzel, C. S., Hanson, R. W., and Davis, P. B., 1995, Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor, J. Clin. Invest. 95:493–502.PubMedGoogle Scholar
  67. Ferkol, T., Pellicena-Palle, A., Eckman, E., Perales, J. C., Trzaska, T., Tosi, M., Redline, R., and Davis, P. B., 1996a, Immunologie responses to gene transfer into mice via the polymeric immunoglobulin receptor, Gene Ther. 3:669–678.PubMedGoogle Scholar
  68. Ferkol, T., Perales, J. C., Mularo, F., and Hanson, R. W., 1996b, Receptor-mediated gene transfer into macrophages, Proc. Natl. Acad. Sci. USA 93:101–105.PubMedGoogle Scholar
  69. de Fiebre, C. M., Bryant, S. O., Notabartolo, D., Wu, P., and Meyer, E. M., 1993, Fusogenic properties of Sendai virosome envelopes in rat brain preparations, Neurochem. Res. 18:1089–1094.PubMedGoogle Scholar
  70. Findeis, M. A., Wu, C. H., and Wu, G. Y., 1994, Ligand-based carrier systems for delivery of DNA to hepatocytes, Methods Enzymol. 247:341–351.PubMedGoogle Scholar
  71. Fisher, K. J., and Wilson, J. M., 1994, Biochemical and functional analysis of an adenovirus-based ligand complex for gene transfer, Biochem. J. 299:49–58.PubMedGoogle Scholar
  72. Forstová, J., Krauzewicz, N., Sandig, V., Elliot, J., Plaková, Z., Strauss, M., and Griffin, B. E., 1995, Polyoma virus as efficient carriers of heterologous DNA into mammalian cells, Hum. Gene Ther. 6:297–306.PubMedGoogle Scholar
  73. Fraley, R., Straubinger, R. M., Rule, G., Springer, E. L., and Papahadjopoulos, D., 1981, Liposomemediated delivery of deoxyribonucleic acid to cells: Enhanced efficiency of delivery related to lipid composition and incubation conditions, Biochemistry 20:6978–6987.PubMedGoogle Scholar
  74. Furth, P. A., Shamay, A., Wall, R. J., and Hennighausen, L., 1992, Gene transfer into somatic tissues by jet injection, Anal. Biochem. 205:365–368.PubMedGoogle Scholar
  75. Furth, P. A., Kerr, D., and Wall, R., 1995, Gene transfer by jet injection into differentiated tissues of living animals and in organ culture, Mol. Biotechnol. 4:121–127.PubMedGoogle Scholar
  76. Gao, L., Wagner, E., Cotten, M., Agarwal, S., Harris, C., Romer, M., Miller, L., Hu, P. C., and Curiel, D., 1993, Direct in vivo gene transfer to airway epithelium employing adenovirus-polylysine-DNA complexes, Hum. Gene Ther. 4:17–24.PubMedGoogle Scholar
  77. Gao, X., and Huang, L., 1991, A novel cationic liposome reagent for efficient transfection of mammalian cells, Biochem. Biophys. Res. Commun. 179:280–285.PubMedGoogle Scholar
  78. Gao, X., and Huang, L., 1996, Potentiation of cationic liposome-mediated gene delivery by polycations, Biochemistry 35:1027–1036.PubMedGoogle Scholar
  79. Geisert, E. E., Jr., Del Mar, N. A., Owens, J. L., and Holmberg, E. G., 1995, Transfecting neurons and glia in the rat using pH-sensitive immunoliposomes, Neurosci. Lett. 184:40–43.PubMedGoogle Scholar
  80. Gershon, H., Ghirlando, R., Guttman, S. B., and Minsky, A., 1993, Mode of formation and structural features of DNA-cationic liposome complexes used for transfection, Biochemistry 32:7143–7151.PubMedGoogle Scholar
  81. Ghirlando, R., Wachtel, E. J., Arad, T., and Minsky, A., 1992, DNA packaging induced by micellar aggregates: A novel in vitro DNA condensation system, Biochemistry 31:7110–7119.PubMedGoogle Scholar
  82. Giladi, E., Raz, E., Karmeli, F., Okon, E., and Rachmilewitz, D., 1995, Transforming growth factor-beta gene therapy ameliorates experimental colitis in rats, Eur. J. Gastroenterol. Hepatol. 7:341–347.PubMedGoogle Scholar
  83. Gottschalk, S., Cristiano, R. J., Smith, L. C., and Woo, S. L., 1994, Folate receptor mediated DNA delivery into tumor cells: Potosomal disruption results in enhanced gene expression, Gene Ther. 1:185–191.PubMedGoogle Scholar
  84. Gottschalk, S., Tweten, R. K., Smith, L. C., and Woo, S. L., 1995, Efficient gene delivery and expression in mammalian cells using DNA coupled with perfringolysin O, Gene Ther. 2:498–503.PubMedGoogle Scholar
  85. Grasso, A. W., and Wu, G. Y., 1994, Therapeutic implications of delivery and expression of foreign genes in hepatocytes, Adv. Pharmacol. 28:169–192.PubMedGoogle Scholar
  86. Haensler, J., and Szoka, F. C., Jr., 1993a, Polyamidoamine cascade polymers mediate efficient transfection of cells in culture, Bioconjugate Chem. 4:372–379.Google Scholar
  87. Haensler, J., and Szoka, F. C., Jr., 1993b, Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes, Bioconjugate Chem. 4:85–93.Google Scholar
  88. Hara, T., Aramaki, Y., Takada, S., Koike, K., and Tsuchiya, S., 1995, Receptor-mediated transfer of pSV2CAT DNA to a human hepatoblastoma cell line HepG2 using asialofetuin-labeled cationic liposomes, Gene 159:167–174.PubMedGoogle Scholar
  89. Hart, S. L., Harbottle, R. P., Cooper, R., Miller, A., Williamson, R., and Coutelle, C., 1995a, Gene delivery and expression mediated by an integrin-binding peptide, Gene Ther. 2:552–554.PubMedGoogle Scholar
  90. Hart, S. L., Mayall, E., Stern, M., Munkonge, F. M., Frost, A., Huang, L., Vasilliou, M., Williamson, R., Alton, E. W., and Coutelle, C., 1995b, The introduction of two silent mutations into a CFTR cDNA construct allows improved detection of exogenous mRNA in gene transfer experiments, Hum. Mol. Genet. 4:1597–1602.PubMedGoogle Scholar
  91. Hartikka, J., Sawdey, M., Cornefert-Jensen, F., Margalith, M., Barnhart, K., Nolasco, M., Vahlsing, H. L., Meek, J., Marquet, M., Hobart, P., Norman, J., and Manthorpe, M., 1996, An improved plasmid DNA expression vector for direct injection into skeletal muscle, Hum. Gene Ther. 7:1205–1217.PubMedGoogle Scholar
  92. Heiser, W. C., 1994, Gene transfer into mammalian cells by particle bombardment, Anal. Biochem. 217:185–196.PubMedGoogle Scholar
  93. Hengge, U. R., Chan, E. F., Foster, R. A., Walker, P. S., and Vogel, J. C., 1995, Cytokine gene expression in epidermis with biological effects following injection of naked DNA, Nat. Genet. 10:161–166.PubMedGoogle Scholar
  94. Hickman, M. A., Malone, R. W., Lehmann Bruinsma, K., Sih, T. R., Knoell, D., Szoka, F. C., Walzern, R., Carlson, D. M., and Powell, J. S., 1994, Gene expression following direct injection of DNA into liver, Hum. Gene Ther. 5:1477–1483.PubMedGoogle Scholar
  95. Holmberg, E. G., Reuer, Q. R., Geisert, E. E., and Owens, J. L., 1994, Delivery of plasmid DNA to glial cells using pH-sensitive immunoliposomes, Biochem. Biophys. Res. Commun. 201:888–893.PubMedGoogle Scholar
  96. Huckett, B., Ariatti, M., and Hawtrey, A. O., 1990, Evidence for targeted gene transfer by receptormediated endocytosis. Stable expression following insulin-directed entry of NEO into HepG2 cells, Biochem. Pharmacol. 40:253–263.PubMedGoogle Scholar
  97. Hui, K. M., Sabapathy, T. K., Oei, A. A., and Chia, T. F., 1994, Generation of allo-reactive cytotoxic T lymphocytes by particle bombardment-mediated gene transfer, J. Immunol. Methods 171:147–155.PubMedGoogle Scholar
  98. Huxley, C., 1994, Mammalian artificial chromosomes: A new tool for gene therapy, Gene Ther. 1:7–12.PubMedGoogle Scholar
  99. Hyde, S. C., Gill, D. R., Higgins, C. F., Trezise, A. E., MacVinish, L. J., Cuthbert, A. W., Ratcliff, R., Evans, M. J., and Colledge, W. H., 1993, Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy, Nature 362:250–255.PubMedGoogle Scholar
  100. Jarnagin, W. R., Debs, R. J., Wang, S. S., and Bissell, D. M., 1992, Cationic lipid-mediated transfection of liver cells in primary culture, Nucleic Acids Res. 20:4205–4211.PubMedGoogle Scholar
  101. Jelinek, J., Kleibl, K., Dexter, T. M., and Margison, G. P., 1988, Transfection of murine multi-potent haemopoietic stem cells with an E. coli DNA alkyltransferase gene confers resistance to the toxic effects of alkylating agents, Carcinogenesis 9:81–87.PubMedGoogle Scholar
  102. Jiao, S., Williams, P., Berg, R. K., Hodgeman, B. A., Liu, L., Repetto, G., and Wolff, J. A., 1992, Direct gene transfer into nonhuman primate myofibers in vivo, Hum. Gene Ther. 3:21–33.PubMedGoogle Scholar
  103. Jones, S. E., McHugh, J. D., Jomary, C., Shallal, A., and Neal, M. J., 1994, Assessment of liposomal transfection of ocular tissues in vivo, Gene Ther. 1(Suppl 1):S61.PubMedGoogle Scholar
  104. Kaneda, Y., Iwai, K., and Uchida, T., 1989a, Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science 243:375–378.PubMedGoogle Scholar
  105. Kaneda, Y., Iwai, K., and Uchida, T., 1989b, Introduction and expression of the human insulin gene in adult rat liver, J. Biol. Chem. 264:12126–12129.PubMedGoogle Scholar
  106. Kato, K., Kaneda, Y., Sakurai, M., Nakanishi, M., and Okada, Y., 1991a, Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats, J. Biol. Chem. 266:22071–22074.PubMedGoogle Scholar
  107. Kato, K., Nakanishi, M., Kaneda, Y., Uchida, T., and Okada, Y., 1991b, Expression of hepatitis B virus surface antigen in adult rat liver. Co-introduction of DNA and nuclear protein by a simplified liposome method, J. Biol. Chem. 266:3361–3364.PubMedGoogle Scholar
  108. Keating, A., Horsfall, W., Hawley, R. G., and Toneguzzo, F., 1990, Effect of different promoters on expression of genes introduced into hematopoietic and marrow stromal cells by electroporation, Exp. Hematol. 18:99–102.PubMedGoogle Scholar
  109. Keating, A., and Toneguzzo, F., 1990, Gene transfer by electroporation: A model for gene therapy, Prog. Clin. Biol Res. 333:491–498.PubMedGoogle Scholar
  110. Kitsis, R. N., Buttrick, P. M., McNally, E. M., Kaplan, M. L., and Leinwand,, L. A., 1991, Hormonal modulation of gene injected into rat heart in vivo, Proc. Natl. Acad. Sci. USA 88:4138–4142.PubMedGoogle Scholar
  111. Klein, T. M., Arentzen, R., Lewis, P. A., and Fitzpatrick McElligott, S., 1992, Transformation of microbes, plants and animals by particle bombardment, Biotechnol. NY 10:286–291.Google Scholar
  112. Kupfer, J. M., Ruan, X. M., Liu, G., Matloff, J., Forrester, J., and Choux, A., 1994, High efficiency gene transfer to autologous rabbit jugular vein grafts using adenovirus-transferrin/polylysine-DNA complexes, Hum. Gene Ther. 5:1437–1443.PubMedGoogle Scholar
  113. Lai, W. C., Bennett, M., Johnston, S. A., Barry, M. A., and Pakes, S. P., 1995, Protection against Mycoplasma pulmonis infection by genetic vaccination, DNA Cell Biol. 14:643–651.PubMedGoogle Scholar
  114. Legendre, J. Y., and Szoka, F. C., Jr., 1992, Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: Comparison with cationic liposomes, Pharm. Res. 9:1235–1242.PubMedGoogle Scholar
  115. Legendre, J. Y., and Supersaxo, A., 1995, Short-chain phospholipids enhance amphipathic peptidemediated gene transfer, Biochem. Biophys. Res. Commun. 217:179–185.PubMedGoogle Scholar
  116. Leibiger, I., Leibiger, B., Sarrach, D., Walther, R., and Zuhlke, H., 1990, Genetic manipulation of rat hepatocytes in vivo. Implications for a therapy model of type-1 diabetes, Biomed. Biochim. Acta 49:1193–1200.PubMedGoogle Scholar
  117. Lesoon Wood, L. A., Kim, W. H., Kleinman, H. K., Weintraub, B. D., and Mixson, A. J., 1995, Systemic gene therapy with p53 reduces growth and metastases of a malignant human breast cancer in nude mice, Hum. Gene Ther. 6:395–405.PubMedGoogle Scholar
  118. Levy, M. Y., Barron, L. G., Meyer, K. B., and Szoka, F. C., 1996, Characterization of plasmid DNA transfer into mouse skeletal muscle: Evaluation of uptake mechanism, expression and secretion of gene products into blood, Gene Ther. 3:201–211.PubMedGoogle Scholar
  119. Li, L., and Hoffman, R. M., 1995, The feasibility of targeted selective gene therapy of the hair follicle, Nat. Med. 1:705–706.PubMedGoogle Scholar
  120. Lin, H., Parmacek, M. S., Morle, G., Boiling, S., and Leiden, J. M., 1990, Expression of recombinant genes in myocardium in vivo after direct injection of DNA, Circulation 82:2217–2221.PubMedGoogle Scholar
  121. Liu, Y., Liggitt, D., Zhong, W., Tu, G., Gaensler, K., and Debs, R., 1995, Cationic liposome-mediated intravenous gene delivery, J. Biol. Chem. 270:24864–24870.PubMedGoogle Scholar
  122. Logan, J. J., Bebok, Z., Walker, L. C., Peng, S., Feigner, P. L., Siegal, G. P., Frizzell, R. A., Dong, J., Howard, M., Matalon, A., et al., 1995, Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium, Gene Ther. 2:38–49.PubMedGoogle Scholar
  123. Maass, G., Schweighoffer, T., Berger, M., Schmidt, W., Herbst, E., Zatloukal, K., Buschle, M., and Birnstiel, M. L., 1995a, Tumor vaccines: Effects and fate of IL-2 transfected murine melanoma cells in vivo, Int. J. Immunopharmacol. 17:65–73.PubMedGoogle Scholar
  124. Maass, G., Schmidt, W., Berger, M., Schilcher, F., Koszik, F., Schneeberger, A., Stingl, G., Birnstiel, M. L., and Schweighoffer, T., 1995b, Priming of tumor-specific T cells in the draining lymph nodes after immunization with interleukin 2-secreting tumor cells: Three consecutive stages may be required for successful tumor vaccination, Proc. Natl. Acad. Sci. USA 92:5540–5544.PubMedGoogle Scholar
  125. Mahato, R. I., Kawabata, K., Nomura, T., Takakura, Y., and Hashida, M., 1995, Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes, J. Pharm. Sci. 84:1267–1271.PubMedGoogle Scholar
  126. Major, M. E., Vitvitski, L., Mink, M. A., Schleef, M., Whalen, R. G., Trepo, C., and Inchauspe, G., 1995, DNA-based immunization with chimeric vectors for the induction of immune responses against the hepatitis C virus nucleocapsid, J. Virol. 69:5798–5805.PubMedGoogle Scholar
  127. Malone, R. W., Hickman, M. A., Lehmann Bruinsma, K., Sih, T. R., Walzern, R., Carlson, D. M., and Powell, J. S., 1994, Dexamethasone enhancement of gene expression after direct hepatic DNA injection, J. Biol. Chem. 269:29903–29907.PubMedGoogle Scholar
  128. Manthorpe, M., Cornefert Jensen, F., Hartikka, J., Feigner, J., Rundell, A., Margalith, M., and Dwarki, V., 1993, Gene therapy by intramuscular injection of plasmid DNA: Studies on firefly luciferase gene expression in mice, Hum. Gene Ther. 4:419–431.PubMedGoogle Scholar
  129. Martinez Fong, D., Mullersman, 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.PubMedGoogle Scholar
  130. McKee, T. D., DeRome, M. E., Wu, G. Y., and Findeis, M. A., 1994, Preparation of asialoorosomucoid-polylysine conjugates, Bioconjugate Chem. 5:306–311.Google Scholar
  131. Merwin, J. R., Noell, G. S., Thomas, W. L., Chiou, H. C., DeRome, M. E., McKee, T. D., Spitalny, G. L., and Findeis, M. A., 1994, Targeted delivery of DNA using YEE(GalNAcAH)3, a synthetic glycopeptide ligand for the asialoglycoprotein receptor, Bioconjugate Chem. 5:612–620.Google Scholar
  132. Merwin, J. R., Carmichael, E. P., Noell, G. S., DeRome, M. E., Thomas, W. L., Robert, N., Spitalny, G., and Chiou, H. C., 1995, CD5-mediated specific delivery of DNA to T lymphocytes: Compartmentalization augmented by adenovirus, J. Immunol. Methods 186:257–266.PubMedGoogle Scholar
  133. Meyer, K. B., Thompson, M. M., Levy, M. Y., Barron, L. G., and Szoka, F. C., Jr., 1995, Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics, Gene Ther. 2:450–460.PubMedGoogle Scholar
  134. Michael, S. I., Hong, J. S., Curiel, D. T., and Engler, J. A., 1995, Addition of a short peptide ligand to the adenovirus fiber protein, Gene Ther. 2:660–668.PubMedGoogle Scholar
  135. Michel, M. L., Davis, H. L., Schleef, M., Mancini, M., Tiollais, P., and Whalen, R. G., 1995, DNA-mediated immunization to the hepatitis B surface antigen in mice: Aspects of the humoral response mimic hepatitis B viral infection in humans, Proc. Natl. Acad. Sci. USA 92:5307–5311.PubMedGoogle Scholar
  136. Midoux, P., Mendes, C., Legrand, A., Raimond, J., Mayer, R., Monsigny, M., and Roche, A. C., 1993, Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells, Nucleic Acids Res. 21:871–878.PubMedGoogle Scholar
  137. Missol, E., Sochanik, A., and Szala, S., 1995, Introduction of murine Il-4 gene into B16(F10) melanoma tumors by direct gene transfer with DNA-liposome complexes, Cancer Lett. 97:189–193.PubMedGoogle Scholar
  138. Mizuguchi, H., Nakagawa, T., Nakanishi, M., Imazu, S., Nakagawa, S., and Mayumi, T., 1996, Efficient gene transfer into mammalian cells using fusogenic liposome, Biochem. Biophys. Res. Commun. 218:402–407.PubMedGoogle Scholar
  139. Morishita, R., Gibbons, G. H., Kaneda, Y., Ogihara, T., and Dzau, V. J., 1993, Novel in vitro gene transfer method for study of local modulators in vascular smooth muscle cells, Hypertension 21:894–899.PubMedGoogle Scholar
  140. Nabel, E. G., Plautz, G., and Nabel, G. J., 1990, Site-specific gene expression in vivo by direct gene transfer into the arterial wall, Science 249:1285–1288.PubMedGoogle Scholar
  141. Nabel, G. J., Nabel, E. G., Yang, Z. Y., Fox, B. A., Plautz, G. E., Gao, X., Huang, L., Shu, S., Gordon, D., and Chang, A. E., 1993, Direct gene transfer with DNA-liposome complexes in melanoma: Expression, biologie activity, and lack of toxicity in humans, Proc. Natl. Acad. Sci. USA 90:11307–13011.PubMedGoogle Scholar
  142. Nabel, G. J., Chang, A. E., Nabel, E. G., Plautz, G. E., Ensminger, W., Fox, B. A., Feigner, P., Shu, S., and Cho, K., 1994, Immunotherapy for cancer by direct gene transfer into tumors, Hum. Gene Ther. 5:57–77.PubMedGoogle Scholar
  143. Nakamura, N., Horibe, S., Matsumoto, N., Tomita, T., Natsuume, T., Kaneda, Y., Shino, K., and Ochi, T., 1996, Transient introduction of a foreign gene into healing rat patellar ligament, J. Clin. Invest. 97:226–231.PubMedGoogle Scholar
  144. Narayanan, R., Jastreboff, M. M., Chiu, C. F., and Bertino, J. R., 1986, In vivo expression of a nonselected gene transferred into murine hematopoietic stem cells by electroporation, Biochem. Biophys. Res. Commun. 141:1018–1024.PubMedGoogle Scholar
  145. Narayanan, R., Tare, N. S., Benjamin, W. R., and Gubler, U., 1989, A sensitive technique to monitor gene transfer and expression in bone marrow stem cells, Exp. Hematol. 17:832–835.PubMedGoogle Scholar
  146. Neil, G. A., and Zimmermann, U., 1993, Electroinjection, Methods Enzymol. 221:339–361.PubMedGoogle Scholar
  147. Nicolau, C., Le Pape, A., Soriano, P., Fargette, F., and Juhel, M. F., 1983, In vivo expression of rat insulin after intravenous administration of the liposome-entrapped gene for rat insulin I, Proc. Natl. Acad. Sci. USA 80:1068–1072.PubMedGoogle Scholar
  148. Nicolet, C. M., Burkholder, J. K., Gan, J., Culp, J., Kashmiri, S. V., Schlom, J., Yang, N. S., and Sondel, P. M., 1995, Expression of a tumor-reactive antibody-interleukin 2 fusion protein after in vivo particle-mediated gene delivery, Cancer Gene Ther. 2:161–170.PubMedGoogle Scholar
  149. Ono, T., Fujino, Y., Tsuchiya, T., and Tsuda, M., 1990, Plasmid DNAs directly injected into mouse brain with lipofectin can be incorporated and expressed by brain cells, Neurosci. Lett. 117:259–263.PubMedGoogle Scholar
  150. Perales, J. C., Ferkol, T., Beegen, H., Ratnoff, O. D., and Hanson, R. W., 1994a, 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.PubMedGoogle Scholar
  151. Perales, J. C., Ferkol, T., Molas, M., and Hanson, R. W., 1994b, An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes, Eur. J. Biochem. 226:255–266.PubMedGoogle Scholar
  152. Philip, R., Liggitt, D., Philip, M., Dazin, P., and Debs, R., 1993, In vivo gene delivery. Efficient transfection of T lymphocytes in adult mice, J. Biol. Chem. 268:16087–16090.PubMedGoogle Scholar
  153. Philip, R., Brunette, E., Kilinski, L., Murugesh, D., McNally, M. A., Ucar, K., Rosenblatt, J., Okarma, T. B., and Lebkowski, J. S., 1994, Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes, Mol. Cell. Biol. 14:2411–2418.PubMedGoogle Scholar
  154. Plank, C., Oberhauser, B., Mechtler, K., Koch, C., and Wagner, E., 1994, The influence of endosomedisruptive peptides on gene transfer using synthetic virus-like gene transfer systems, J. Biol. Chem. 269:12918–12924.PubMedGoogle Scholar
  155. Plank, C., Mechtler, K., Szoka, F. C., Jr., and Wagner, E., 1996, Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery, Hum. Gene Ther. 7:1437–1446.PubMedGoogle Scholar
  156. Plautz, G. E., Yang, Z. Y., Wu, B. Y., Gao, X., Huang, L., and Nabel, G. J., 1993, Immunotherapy of malignancy by in vivo gene transfer into tumors, Proc. Natl. Acad. Sci. USA 90:4645–4659.PubMedGoogle Scholar
  157. Poncet, P., Panczak, A., Goupy, C., Gustafsson, K., Blanpied, C., Chavanel, G, Hirsch, R., and Hirsch, F, 1996, Antifection: An antibody-mediated method to introduce genes into lymphoid cells in vitro and in vivo, Gene Ther. 3:731–738.PubMedGoogle Scholar
  158. Potter, H., 1988, Electroporation in biology: Methods, applications, and instrumentation, Anal. Biochem. 174:361–373.PubMedGoogle Scholar
  159. Potter, C. G., Tan, C. C., and Ratcliffe, P. J., 1991, Quantification of 32P-labeled samples in gel fragments using the flat-bed liquid scintillation counter, Anal. Biochem. 197:121–124.PubMedGoogle Scholar
  160. Puchalski, R. B., and Fahl, W. E., 1992, Gene transfer by electroporation, lipofection, and DEAE-dextran transfection: Compatibility with cell-sorting by flow cytometry, Cytometry 13:23–30.PubMedGoogle Scholar
  161. Raja-Walia, R., Webber, J., Naftilan, J., Chapman, G. D., and Naftilan, A. J., 1995, Enhancement of liposome-mediated gene transfer into vascular tissue by replication-deficient adenovirus, Gene Ther. 2:521–530.PubMedGoogle Scholar
  162. Raz, E., Carson, D. A., Parker, S. E., Parr, T. B., Abai, A. M., Aichinger, G., Gromkowski, S. H., Singh, M., Lew, D., Yankauckas, M. A., et al., 1994, Intradermal gene immunization: The possible role of DNA uptake in the induction of cellular immunity to viruses, Proc. Natl Acad. Sci. USA 91:9519–9523.PubMedGoogle Scholar
  163. Reimer, D. L., Zhang, Y., Kong, S., Wheeler, J. J., Graham, R. W., and Bally, M. B., 1995, Formation of novel hydrophobic complexes between cationic lipids and plasmid DNA, Biochemistry 34:12877–12883.PubMedGoogle Scholar
  164. Remy, J. S., Sirlin, C., Vierling, P., and Behr, J. P., 1994, Gene transfer with a series of lipophilic DNA-binding molecules, Bioconjugate Chem. 5:647–654.Google Scholar
  165. 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 92:1744–1748.PubMedGoogle Scholar
  166. Riessen, R., Rahimizadeh, H., Blessing, E., Takeshita, S., Barry, J. J., and Isner, J. M., 1993, Arterial gene transfer using pure DNA applied directly to a hydrogel-coated angioplasty balloon, Hum. Gene Ther. 4:749–758.PubMedGoogle Scholar
  167. Robinson, H. L., Hunt, L. A., and Webster, R. G., 1993, Protection against a lethal influenza virus challenge by immunization with a haemagglutinin-expressing plasmid DNA, Vaccine 11:957–960.PubMedGoogle Scholar
  168. Roessler, B. J., and Davidson, B. L., 1994, Direct plasmid mediated transfection of adult murine brain cells in vivo using cationic liposomes, Neurosci. Lett. 167:5–10.PubMedGoogle Scholar
  169. Rojanasakul, Y., Wang, L. Y., Malanga, C. J., Ma, J. K., and Liaw, J., 1994, Targeted gene delivery to alveolar macrophages via Fc receptor-mediated endocytosis, Pharm. Res. 11:1731–1736.PubMedGoogle Scholar
  170. Ross, G. F., Morris, R. E., Ciraolo, G., Huelsman, K., Bruno, M., Whitsett, J. A., Baatz, J. E., and Korfhagen, T. R., 1995, Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture, Hum. Gene Ther. 6:31–40.PubMedGoogle Scholar
  171. Rubin, J., Charboneau, J. W., Reading, C., and Kovach, J. S., 1994, Phase I study of immunotherapy of hepatic metastases of colorectal carcinoma by direct gene transfer, Hum. Gene Ther. 5:1385–1399.PubMedGoogle Scholar
  172. 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., et al., 1993, Safety and short-term toxicity of a novel cationic lipid formulation for human gene therapy, Hum. Gene Ther. 4:781–788.PubMedGoogle Scholar
  173. Sawa, Y., Suzuki, K., Bai, H. Z., Shirakura, R., Morishita, R., Kaneda, Y., and Matsuda, H., 1995, Efficiency of in vivo gene transfection into transplanted rat heart by coronary infusion of HVJ liposome, Circulation 92:II479–482.PubMedGoogle Scholar
  174. Schmidt, W., Schweighoffer, T., Herbst, E., Maass, G., Berger, M., Schilcher, F., Schaffner, G., and Birnstiel, M. L., 1995a, Cancer vaccines: The interleukin-2 dosage effect, Proc. Natl. Acad. Sci. USA 92:4711–4714.PubMedGoogle Scholar
  175. Schmidt Wolf, G. D., and Schmidt Wolf, I. G., 1995b, Cytokines and gene therapy, Immunol. Today 16:173–175.PubMedGoogle Scholar
  176. Schoeman, R., Joubert, D., Ariatti, M., and Hawtrey, A. O., 1995, 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. 2:509–516.PubMedGoogle Scholar
  177. Schwarz, L. A., Johnson, J. L., Black, M., Cheng, S. H., Hogan, M. E., and Waldrep, J. C., 1996, Delivery of DNA-cationic liposome complexes by small-particle aerosol, Hum. Gene Ther. 7:731–741.PubMedGoogle Scholar
  178. Schwartz, P., Benoist, C., Abdallah, B., Scherman, D., Behr, J-P., and Demeneix, B. A., 1995, Lipospermine-based gene transfer into the newborn mouse brain is optimized by a low lipospermine DNA charge ratio, Hum. Gene Ther. 6:1515–1524.PubMedGoogle Scholar
  179. Schwartz, P., Benoist, C., Abdallah, B., Ragara, R., Hassan, J-P., Scherman, D., and Demeneix, B. A., 1996, Gene transfer by naked DNA into adult mouse brain, Gene Ther. 3:405–411.PubMedGoogle Scholar
  180. Schwarzenberger, P., Spence, S. E., Gooya, J. M., Michiel, D., Curiel, D. T., Ruscetti, F. W., and Keller, J. R., 1996, Targeted gene transfer to human hematopoietic progenitor cell lines through the c-kit receptor, Blood 87:472–478.PubMedGoogle Scholar
  181. Sedegah, M., Hedstrom, R., Hobart, P., and Hoffman, S. L., 1994, Protection against malaria by immunization with plasmid DNA encoding circumsporozoite protein, Proc. Natl. Acad. Sci. USA 91:9866–9870.PubMedGoogle Scholar
  182. Seth, P., Rosenfeld, M., Higginbotham, J., and Crystal, R. G., 1994, Mechanism of enhancement of DNA expression consequent to cointernalization of a replication-deficient adenovirus and unmodified plasmid DNA, J. Virol. 68:933–940.PubMedGoogle Scholar
  183. Shi, Y., Fard, A., Vermani, P., and Zalewski, A., 1994, Transgene expression in the coronary circulation: Transcatheter gene delivery, Gene Ther. 1:408–414.PubMedGoogle Scholar
  184. Sikes, M. L., O’Malley B. W., Jr., Finegold, M. J., and Ledley, F. D., 1994, In vivo gene transfer into rabbit thyroid follicular cells by direct DNA injection, Hum. Gene Ther. 5:837–844.PubMedGoogle Scholar
  185. Smith, J. G., Walzern, R. L., and German, J. B., 1993, Liposomes as agents of DNA transfer, Biochim. Biophys. Acta 1154:327–340.PubMedGoogle Scholar
  186. Smith, M. J., Rousculp, M. D., Goldsmith, K. T., Curiel, D. T., and Garver, R. I., Jr., 1994, Surfactant protein A-directed toxin gene kills lung cancer cells in vitro, Hum. Gene Ther. 5:29–35.PubMedGoogle Scholar
  187. Son, K., and Huang, L., 1994, Exposure of human ovarian carcinoma to cisplatin transiently sensitizes the tumor cells for liposome-mediated gene transfer, Proc. Natl. Acad. Sci. USA 91:12669–12672.PubMedGoogle Scholar
  188. Son, K., and Huang, L., 1996, Factors influencing the drug sensitlization of human tumor cells for in situ. lipofection, Gene Ther. 3:630–634.PubMedGoogle Scholar
  189. Soriano, P., Dijkstra, J., Legrand, A., Spanjer, H., Londos Gagliardi, D., Roerdink, F., Scherphof, G., and Nicolau, C., 1983, 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 80:7128–7131.PubMedGoogle Scholar
  190. Stankovics, J., Crane, A. M., Andrews, E., Wu, C. H., Wu, G. Y., and Ledley, F. D., 1994, Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/ polylysine/DNA complexes, Hum. Gene Ther. 5:1095–1104.PubMedGoogle Scholar
  191. Straubinger, R. M., Hong, K., Friend, D. S., and Papahadjopoulos, D., 1983, Endocytosis of liposomes and intracellular fate of encapsulated molecules: Encounter with a low pH compartment after internalization in coated vesicles, Cell 32:1069–1079.PubMedGoogle Scholar
  192. Takahashi, M., Furukawa, T., Tanaka, I., Nikkuni, K., Aoki, A., Kishi, K., Koike, T., Moriyama, Y., and Shibata, A., 1992, Gene introduction into granulocyte-macrophage progenitor cells by electroporation: The relationship between introduction efficiency and the proportion of cells in S-phase, Leukemia Res. 16:761–767.Google Scholar
  193. Takehara, T., Hayashi, N., Yamamoto, M., Miyamoto, Y., Fusamoto, H., and Kameda, T., 1996, In vivo gene transfer and expression in rat stomach by submucosal injection of plasmid DNA, Hum. Gene Ther. 7:589–593.PubMedGoogle Scholar
  194. Tekle, E., Astumian, R. D., and Chock, P. B., 1991, Electroporation by using bipolar oscillating electric field: An improved method for DNA transfection of NIH 3T3 cells, Proc. Natl. Acad. Sci. USA 88:4230–4234.PubMedGoogle Scholar
  195. Thierry, A. R., Lunardi Iskandar, Y., Bryant, J. L., Rabinovich, P., Gallo, R. C., and Mahan, L. C., 1995, Systemic gene therapy: Biodistribution and long-term expression of a transgene in mice, Proc. Natl. Acad. Sci. USA 92:9742–9746.PubMedGoogle Scholar
  196. Thurnher, M., Wagner, E., Clausen, H., Mechtler, K., Rusconi, S., Dinter, A., Birnstiel, M. L., Berger, E. G., and Cotten, M., 1994, Carbohydrate receptor-mediated gene transfer to human T leukaemic cells, Glycobiology 4:429–435.PubMedGoogle Scholar
  197. Tikchonenko, T. I., Glushakova, S. E., Kislina, O. S., Grodnitskaya, N. A., Manykin, A. A., and Naroditsky, B. S., 1988, Transfer of condensed viral DNA into eukaryotic cells using proteoliposomes, Gene 63:321–330.PubMedGoogle Scholar
  198. Tomita, N., Higaki, J., Morishita, R., Kato, K., Mikami, H., Kaneda, Y., and Ogihara, T., 1992, Direct in vivo gene introduction into rat kidney, Biochem. Biophys. Res. Commun. 186:129–134.PubMedGoogle Scholar
  199. Toneguzzo, F., and Keating, A., 1986, Stable expression of selectable genes introduced into human hematopoietic stem cells by electric field-mediated DNA transfer, Proc. Natl. Acad. Sci. USA 83:3496–3499.PubMedGoogle Scholar
  200. Trubetskoy, V. S., Torchilin, V. P., Kennel, S., and Huang, L., 1992a, Cationic liposomes enhance targeted delivery and expression of exogenous DNA mediated by N-terminal modified poly(L-lysine)-antibody conjugate in mouse lung endothelial cells, Biochim. Biophys. Acta 1131:311–313.PubMedGoogle Scholar
  201. Trubetskoy, V. S., Torchilin, V. P., Kennel, S. J., and Huang, L., 1992b, Use of N-terminal modified poly(L-lysine)-antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells, Bioconjugate Chem. 3:323–327.Google Scholar
  202. Tsan, M. F., White, J. E., and Shepard, B., 1995, Lung-specific direct in vivo gene transfer with recombinant plasmid DNA, Am. J. Physiol. 268:L1052–1056.PubMedGoogle Scholar
  203. Tsukamoto, M., Ochiya, T., Yoshida, S., Sugimura, T., and Terada, M., 1995, Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice, Nat. Genet. 9:243–248.PubMedGoogle Scholar
  204. Ulmer, J. B., Donnelly, J. J., Parker, S. E., Rhodes, G. H., Feigner, P. L., Dwarki, V. J., Gromkowski, S. H., Deck, R. R., DeWitt, C. M., Friedman, A., et al., 1993, Heterologous protection against influenza by injection of DNA encoding a viral protein, Science 259:1745–1749.PubMedGoogle Scholar
  205. Vile, R. G., and Hart, I. R., 1993, In vitro and in vivo targeting of gene expression to melanoma cells, Cancer Res. 53:962–967.PubMedGoogle Scholar
  206. Vile, R. G., and Hart, I. R., 1994, Targeting of cytokine gene expression to malignant melanoma cells using tissue specific promoter sequences, Ann. Oncol. 5(Suppl. 4):59–65.PubMedGoogle Scholar
  207. Vitadello, M., Schiaffino, M. V., Picard, A., Scarpa, M., and Schiaffino, S., 1994, Gene transfer in regenerating muscle, Hum. Gene Ther. 5:11–18.PubMedGoogle Scholar
  208. Vitiello, L., Chonn, A., Wasserman, J. D., Duff, C., and Worton, R. G., 1996, Condensation of plasmid DNA with polylysine improves liposome-mediated gene transfer into established and primary muscle cells, Gene Ther. 3:396–404.PubMedGoogle Scholar
  209. Vogelzang, N. J., Lestingi, T. M., Sudakoff, G., and Kradjian, S. A., 1994, Phase I study of immuno-therapy of metastatic renal cell carcinoma by direct gene transfer into metastatic lesions, Hum. Gene Ther. 5:1357–1370.PubMedGoogle Scholar
  210. Wagner, E., Zenke, M., Cotten, M., Beug, H., and Birnstiel, M. L., 1990, Transferrin-polycation conjugates as carriers for DNA uptake into cells, Proc. Natl. Acad. Sci. USA 87:3410–3414.PubMedGoogle Scholar
  211. Wagner, E., Cotten, M., Foisner, R., and Birnstiel, M. L., 1991a, Transferrin-polycation-DNA complexes: The effect of polycations on the structure of the complex and DNA delivery to cells, Proc. Natl. Acad. Sci. USA 88:4255–4259.PubMedGoogle Scholar
  212. Wagner, E., Cotten, M., Mechtler, K., Kirlappos, H., and Birnstiel, M. L., 1991b, DNA-binding transferrin conjugates as functional gene-delivery agents: Synthesis by linkage of polylysine or ethidium homodimer to the transferrin carbohydrate moiety, Bioconjugate Chem. 2:226–231.Google Scholar
  213. Wagner, E., Plank, C., Zatloukal, K., Cotten, M., and Birnstiel, M. L., 1992a, Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: Toward a synthetic virus-like gene-transfer vehicle, Proc. Natl. Acad. Sci. USA 89:7934–7938.PubMedGoogle Scholar
  214. Wagner, E., Zatloukal, K., Cotten, M., Kirlappos, H., Mechtler, K., Curiel, D. T., and Birnstiel, M. L., 1992b, Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptormediated gene delivery and expression of transfected genes, Proc. Natl. Acad. Sci. USA 89:6099–6103.PubMedGoogle Scholar
  215. 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. 14:113–135.Google Scholar
  216. Walther, R., Leibiger, I., Kiessling, U., Sarrach, D., and Zuhlke, H., 1988, Transfer of a human preproinsulin gene containing plasmid into non-pancreatic mammalian cells, Biomed. Biochim. Acta 47:343–348.PubMedGoogle Scholar
  217. Wang, C. Y., and Huang, L., 1987a, pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse, Proc. Natl. Acad. Sci. USA 84:7851–7855.PubMedGoogle Scholar
  218. Wang, C. Y., and Huang, L., 1987b, Plasmid DNA adsorbed to pH-sensitive liposomes efficiently transforms the target cells, Biochem. Biophys. Res. Commun. 147:980–985.PubMedGoogle Scholar
  219. Wang, C. Y., and Huang, L., 1989, Highly efficient DNA delivery mediated by pH-sensitive immunoliposomes, Biochemistry 28:9508–9514.PubMedGoogle Scholar
  220. Wang, B., Ugen, K. E., Srikantan, V., Agadjanyan, M. G., Dang, K., Refaeli, Y., Sato, A. I., Boyer, J., Williams, W. V., and Weiner, D. B., 1993, Gene inoculation generates immune responses against human immunodeficiency virus type 1, Proc. Natl. Acad. Sci. USA 90:4156–4160.PubMedGoogle Scholar
  221. Wang, C., Chao, L., and Chao, J., 1995, Direct gene delivery of human tissue kallikrein reduces blood pressure in spontaneously hypertensive rats, J. Clin. Invest. 95:1710–1716.PubMedGoogle Scholar
  222. Wattiaux, R., Jadot, M., Dubois, F., Misquith, S., and Wattiaux De Coninck, S., 1995, Uptake of exogenous DNA by rat liver: Effect of cationic lipids, Biochem. Biophys. Res. Commun. 213:81–87.PubMedGoogle Scholar
  223. Wells, D. J., and Goldspink, G., 1992, Age and sex influence expression of plasmid DNA directly injected into mouse skeletal muscle, FEBS Lett. 306:203–205.PubMedGoogle Scholar
  224. Westbrook, C. A., Chmura, S. J., Arenas, R. B., Kim, S. Y., and Otto, G., 1994, Human APC gene expression in rodent colonie epithelium in vivo using liposomal gene delivery, Hum. Mol. Genet. 3:2005–2010.PubMedGoogle Scholar
  225. Williams, R. S., Johnston, S. A., Riedy, M., DeVit, M. J., McElligott, S. G., and Sanford, J. C., 1991, Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles, Proc. Natl. Acad. Sci. USA 88:2726–2730.PubMedGoogle Scholar
  226. Wilson, J. M., Grossman, M., Wu, C. H., Chowdhury, N. R., Wu, G. Y., and Chowdhury, J. R., 1992, Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits, J. Biol Chem. 267:963–967.PubMedGoogle Scholar
  227. Wolfert, M. A., and Seymour, I. W., 1996, Atomic force microscopic analysis of the influence of poly(L)lysine on the size of polylelectrolyte complexes formed with DNA, Gene Ther. 3:269–273.PubMedGoogle Scholar
  228. Wolff, J. A., Malone, R. W., Williams, P., Chong, W., Acsadi, G., Jani, A., and Feigner, P. L., 1990, Direct gene transfer into mouse muscle in vivo, Science 247:1465–1468.PubMedGoogle Scholar
  229. Wolff, J. A., Williams, P., Williams, P., Acsadi, G., Jiao, S., Jani, A., and Chong, W., 1991, Conditions affecting direct gene transfer into rodent muscle in vivo, Biotechniques 11:474–485.PubMedGoogle Scholar
  230. Wolff, J. A., Dowty, M. E., Jiao, S., Repetto, G., Berg, R. K., Ludtke, J. J., Williams, P., and Slautterback, D. B., 1992, Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle, J. Cell Sci. 103:1249–1259.PubMedGoogle Scholar
  231. van der Woude, I., Visser, H. W., ter Beest, M. B., Wagenaar, A., Ruiters, M. H., Engberts, J. B., and Hoekstra, D., 1995, Parameters influencing the introduction of plasmid DNA into cells by the use of synthetic amphiphiles as a carrier system, Biochim. Biophys. Acta 1240:34–40.PubMedGoogle Scholar
  232. Wu, G. Y., and Wu, C. H., 1987, Receptor-mediated in vitro gene transformation by a soluble DNA carrier system, J. Biol. Chem. 262:4429–4432.PubMedGoogle Scholar
  233. Wu, G. Y., and Wu, C. H., 1988a, Evidence for targeted gene delivery to Hep G2 hepatoma cells in vitro, Biochemistry 27:887–892.PubMedGoogle Scholar
  234. Wu, G. Y., and Wu, C. H., 1988b, Receptor-mediated gene delivery and expression in vivo, J. Biol Chem. 263:14621–14624.PubMedGoogle Scholar
  235. Wu, C. H., Wilson, J. M., and Wu, G. Y., 1989, Targeting genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo, J. Biol. Chem. 264:16985–16987.PubMedGoogle Scholar
  236. Wu, G. Y., Wilson, J. M., Shalaby, F., Grossman, M., Shafritz, D. A., and Wu, C. H., 1991, Receptormediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats, J. Biol. Chem. 266:14338–14342.PubMedGoogle Scholar
  237. Wu, G. Y., Zhan, P., Sze, L. L., Rosenberg, A. R., and Wu, C. H., 1994, Incorporation of adenovirus into a ligand-based DNA carrier system results in retention of original receptor specificity and enhances targeted gene expression, J. Biol Chem. 269:11542–11546.PubMedGoogle Scholar
  238. Wu, P., de Fiebre, C. M., Millard, W. J., Elmstrom, K., Gao, Y., and Meyer, E. M., 1995, Sendai virosomal infusion of an adeno-associated virus-derived construct containing neuropeptide Y into primary rat brain cultures, Neurosci. Lett. 190:73–76.PubMedGoogle Scholar
  239. Wu, P., de Fiebre, C. M., Millard, W. J., King, M. A., Wang, S., Bryant, S. O., Gao, Y. P., Martin, E. J., and Meyer, E. M., 1996, An AAV promoter-driven neuropeptide Y gene delivery system using Sendai virosomes for neurons and rat brain, Gene Ther. 3:246–253.PubMedGoogle Scholar
  240. Xiang, Z. Q., Spitalnik, S., Tran, M., Wunner, W. H., Cheng, J., and Ertl, H. C., 1994, Vaccination with a plasmid vector carrying the rabies virus glycoprotein gene induces protective immunity against rabies virus, Virology 199:132–140.PubMedGoogle Scholar
  241. Yang, N. S., Burkholder, J., Roberts, B., Martinell, B., and McCabe, D., 1990, In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment, Proc. Natl. Acad. Sci. USA 87:9568–9572.PubMedGoogle Scholar
  242. Yang, T. A., Heiser, W. C., and Sedivy, J. M., 1995, Efficient in situ electroporation of mammalian cells grown on microporous membranes, Nucleic Acids Res. 23:2803–2810.PubMedGoogle Scholar
  243. Yin, W., and Cheng, P. W., 1994, Lectin conjugate-directed gene transfer to airway epithelial cells, Biochem. Biophys. Res. Commun. 205:826–833.PubMedGoogle Scholar
  244. Yovandich, J., O’Malley, B. W. Jr., Sikes, M., and Ledley, F. D., 1995, Gene transfer to synovial cells by intra-articular administration of plasmid DNA, Hum. Gene Ther. 6:603–610.PubMedGoogle Scholar
  245. 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. 270:18997–19007.PubMedGoogle Scholar
  246. Zarozinski, C. C., Fynan, E. F., Selin, L. K., Robinson, H. L., and Welsh, R. M., 1995, Protective CTL-dependent immunity and enhanced immunopathology in mice immunized by particle bombardment with DNA encoding an internal virion protein, J. Immunol. 154:4010–4017.PubMedGoogle Scholar
  247. Zauner, W., Blaas, D., Kuechler, E., and Wagner, E., 1995, Rhinovirus-mediated endosomal release of transfection complexes, J. Virol. 69:1085–1092.PubMedGoogle Scholar
  248. Zenke, M., Steinlein, P., Wagner, E., Cotten, M., Beug, H., and Birnstiel, M. L., 1990, Receptor-mediated endocytosis of transferrin-polycation conjugates: An efficient way to introduce DNA into hematopoietic cells, Proc. Natl Acad. Sci. USA 87:3655–3659.PubMedGoogle Scholar
  249. Zhou, X. H., Klibanov, A. L., and Huang, L., 1991, Lipophilic polylysines mediate efficient DNA transfection in mammalian cells, Biochim. Biophys. Acta 1065:8–14.PubMedGoogle Scholar
  250. Zhou, X., and Huang, L., 1994, DNA transfection mediated by cationic liposomes containing lipopolylysine: Characterization and mechanism of action, Biochim. Biophys. Acta 1189:195–203.PubMedGoogle Scholar
  251. Zhou, H., Zeng, G., Zhu, X., Tang, J., Chen, G., Huang, Q., Peng, T., and Hu, B., 1995, Enhanced adenoassociated virus vector expression by adenovirus protein-cationic liposome complex. A novel and high efficient way to introduce foreign DNA into endothelial cells, Chin. Med. J. Engl. 108:332–337.PubMedGoogle Scholar
  252. Zhu, N., Liggitt, D., Liu, Y., and Debs, R., 1993, Systemic gene expression after intravenous DNA delivery into adult mice, Science 261:209–211.PubMedGoogle Scholar
  253. Zeigler, S. T., Kerby, J. D., Curiel, D. T., Diethelm, A. G., and Thompson, J. A., 1996, Molecular conjugate-mediated gene transfer into isolated human kidneys, Transplantation 61:812–817.PubMedGoogle Scholar

Copyright information

© Springer Science+Business Media New York 1999

Authors and Affiliations

  • A. Djeha
    • 1
  • L. S. Lashford
    • 2
  1. 1.CRC Department of Experimental HaematologyPaterson Institute for Cancer ResearchManchesterUK
  2. 2.Paterson Institute for Cancer ResearchChristie Hospital (NHS) TrustManchesterUK

Personalised recommendations