Nonviral delivery systems for gene therapy

Understanding Gene Therapy

Volumn , Issue , 2023, Pages 43-69

  Miller, Andrew D ^a  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85161101030     PISSN: None     EISSN: None     Source Type: Book    
DOI: None     Document Type: Chapter

References (145)

1. Felgner, P.L., Barenholz,Y., Behr, J.-P., Cheng, S.H., Cullis, P., Huang, L., Jessee, J.A., Seymour, L., Szoka, F., Thierry, A.R., Wagner, E. and Wu, G. (1997) Nomenclature for synthetic gene delivery systems. Hum. Gene Ther., 8, 511-512.
2. Miller, A.D. (1998) Cationic liposomes for gene therapy. Angew. Chem. Intl. Ed., 37, 1768-1785.
3. Miller, A.D. (1998) Cationic liposome systems in gene therapy. Curr. Res. Mol. Ther., 1, 494-503.
4. 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.
5. Leventis, R. and Silvius, J.R. (1990) Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles. Biochim. Biophys. Acta, 1023, 124-132.
6. Gebeyehu, G., Jessee, J.A., Valentina, C. and Hawley-Nelson, P. (1993) Cationic lipids. US-05334761, GIBCO/BRL.
7. Nantz, M.H., Bennett, M.J. and Malone, R.W. (1996) Cationic transport reagents. US-05527928, Promega.
8. Behr, J.-P., Demeneix, B., Loeffler, J.-P. and Perez-Mutui, J. (1989) Efficient gene transfer into mammalian primary endocrine cells with lipopolyaminecoated DNA. Proc. Natl Acad. Sci. USA, 86, 6982-6986.
9. Ruysschaert, J.-M., El Ouahabi A., Willeaume, V., Huez, G., Fuks, R., Vandenbranden, M. and Di Stefano, P. (1994) A novel cationic amphiphile for transfection of mammalian cells. Biochem. Biophys. Res. Commun., 203, 1622-1628.
10. Defrise-Quertain, F., Duquenoy, P., Brasseur, R., Brak, P., Caillaux, B., Fuks, R. and Ruysschaert, J.-M. (1986) Vesicle formation by double long-chain amidines. J. Chem. Soc. Chem. Commun., 1060-1062.
11. Kunitake, T., Okahata, Y., Tamaki, K., Kumamaru, F. and Takayanagi, M. (1977) Formation of the bilayer membrane from a series of quaternary ammonium salts. Chem. Lett., 387-390.
12. Moradpour, D., Schauer, J.I., Zurawski, V.R. Jr., Wands, J.R. and Boutin, R.H. (1996) Efficient gene transfer into mammalian cells with cholesterolspermidine. Biochem. Biophys. Res. Commun., 221, 82-88.
13. Gao, X. and Huang, L. (1991) A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun., 179,280-285.
14. Felgner, 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.
15. Wheeler, C.J., Feigner, P.L., Tsai Y.J., Marshall, J., Sukhu, L., Doh, S.G., Hartikka, J., Nietupski, J., Manthorpe, M., Nichols, M., Plewe, M., Liang, X., Norman, J., Smith, A. and Cheng, S. H. (1996) A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc. Natl Acad. Sci. USA, 93, 11454-11459.
16. Balasubramaniam, R.P., Bennett, M.J., Aberle, A.M., Malone, J.G., Nantz, M.H. and Malone, R.W. (1996) Structural and functional analysis of cationic transfection lipids: the hydrophobic domain. Gene Ther., 3, 163-172.
17. Akao, T., Nakayama, T., Takeshia, K. and Ito, A. (1994) Design of a new cationic amphiphile with efficient DNA-transfection ability. Biochem. Mol. Biol. Intl., 34, 915-920.
18. Kunitake, T., Nakashima, N., Shimomura, M., Okahata, Y., Kano, K. and Ogawa, T. (1980) Unique properties of chromophore-containing bilayer aggregates: enhanced chirality and photo-chemically induced morphological change. J. Am. Chem. Soc, 102, 6642-6644.
19. Lewis, J.G., Lin, K.-Y., Kothavale, A., Flanagan, W.M., Matteucci, M.D., DePrince, B., Mook, R.A. Jr., Hendren R.W. and Wagner R.W. (1996) A serum-resistant cytofectin for cellular delivery of antisense oligodeoxynucleotides and plasmid DNA. Proc. Natl Acad. Sci. USA, 93, 3176-3181.
20. Pitard, B., Aguerre, O., Airiau, M., Lachages, A.-M., Boukhnikachvili, T., Byk, G., Dubertret, C, Herviou, C, Scherman, D., Mayaux, J.-F. and Crouzet, J. (1997) Virus-sized self assembling lamellar complexes between plasmid DNA and cationic micelles promote gene transfer. Proc. Natl Acad. Sci. USA, 94, 14412-14417.
21. Byk, G., Dubertret, C, Escriou, V., Frederic, M., Jaslin, G., Rangara, R., Pitard, B., Crouzet, J., Wils, P., Schwartz, B. and Scherman, D. (1998) Synthesis, activity and structure-activity relationship studies of novel cationic lipids for DNA transfer. J. Med. Chem., 41, 224-235.
22. Thierry, A.R., Rabinovich, P., Peng, B., Mahan, L.C., Bryant, J.L. and Gallo, R.C. (1997) Characterization of liposome-mediated gene delivery: expression, stability and pharmacokinetics of plasmid DNA. Gene Ther., 4, 226-237.
23. Walker, S., Sofia, M.J., Kakarla, R., Kogan N.A., Wierichs, L., Longley, C.B., Bruker, K., Axelrod, H.R., Midha, S., Babu, S. and Kahne, D. (1996) Cationic facial amphiphiles: a promising class of transfection agents. Proc. Natl Acad. Sci. USA, 93, 1585-1590.
24. Lee, E.R., Marshall, J., Siegel, C.S., Jiang, C, Yew, N.S., Nichols M.R., Nietupski, J.B., Ziegler, R.J., Lane, M.B., Wang, K.X., Wan, N.C., Scheule, R.K., Harris, D.J., Smith, A.E. and Cheng, S.H. (1996) Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum. Gene Ther., 7, 1701-1717.
25. Cooper, R.G., Etheridge, C.J., Stewart, L., Marshall, J., Rudginsky, S., Cheng, S.H. and Miller, A.D. (1998) Polyamine analogues of 3B-[N-(N',N'-dimethylaminoethane)carbamoyl]cholesterol (DC-Choi) as agents for gene delivery. Chem. Eur. J., 4, 137-152.
26. Vigneron, J.P., Oudrhiri, N., Fauquet, M., Vergely, L., Bradley, J.-C, Basseville, M., Lehn, P. and Lehn, J.-M. (1996) Guanidinium-cholesterol cationic lipids: efficient vectors for the transfection of eukaryotic cells. Proc. Natl Acad. Sci. USA, 93, 9682-9686.
27. Puyal, C, Milhaud, P., Bienvenüe, A. and Philippot, J.R. (1995) A new cationic liposome encapsulating genetic material. A potential delivery system for polynucleotides. Eur. J. Biochem., 228, 697-703.
28. Brunette, E., Stribling, R. and Debs, R. (1992) Lipofection does not require the removal of serum. Nucleic Acids Res., 20, 1151.
29. Gorman, C.M., Aikawa, M., Fox, B., Fox, E., Lapuz, C., Michaud, B., Nguyen, H., Roche, E., Sawa, T. and Wiener-Kronish, J.P. (1997) Efficient in vivo delivery of DNA to pulmonary cells using the novel lipid EDMPC. Gene Ther., 4, 983-992.
30. Ito, A., Miyazoe, R., Mitoma, J.-Y., Akao, T., Osaki, T. and Kunitake, T. (1990) Synthetic cationic amphiphiles for liposome-mediated DNA transfection. Biochem. Intl., 22, 235-241.
31. Solodin, I., Brown, C.S., Bruno, M.S., Chow, C.-Y., Jang, E.-H, Debs, R.J. and Heath, T.D. (1995) A novel series of amphiphilic imidazolinium componds for in vitro and in vivo gene delivery. Biochemistry, 34, 13537-13544.
32. van der Woude, I., Wagenaar, A., Meekel, A.A.P., TerBeest, M.B.A., Ruiters, M.H.J., Engberts, J.B.F.N. and Hoekstra, D. (1997) Novel pyridinium surfactants for efficient, nontoxic in vitro gene delivery. Proc. Natl Acad. Sci. USA, 94, 1160-1165.
33. Dodds, E., Dunckley, M.G., Naujoks, K, Michaelis, U. and Dickson, G. (1998) Lipofection of cultured mouse muscle cells: a direct comparison of Lipofectamine and DOSPER. Gene Ther., 5, 542-551.
34. Vitiello, L., Bockhold, K., Joshi, P.B. and Worton, R.G. (1998) Transfection of cultured myoblasts in high serum concentration with DOD AC: DOPE liposomes. Gene Ther., 5, 1306-1313.
35. Schwartz, B., 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.
36. Liu, F., Qi, H., Huang, L. and Liu, D. (1997) Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration. Gene Ther., 4, 517-523.
37. Yang, J.-P. and Huang, L. (1997) Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther., 4, 950-960.
38. 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.
39. Alton, E.W.F.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., Fasold, K.I., Miller, A.D., Dickinson, P., Stevenson, B.J., McLachlan, G., Dorin, J.R. and Porteous, D.J. (1993) Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat. Genet., 5, 135-142.
40. McQuillin, A., Murray, K.D., Etheridge, C.J., Stewart, L., Cooper, R.G., Brett, P.M., Miller, A.D. and Gurling, H.M.D. (1997) Optimization of liposome mediated transfection of a neuronal cell line. NeuroReport, 8,1481-1484.
41. Fife, K., Bower, M., Cooper, R.G., Stewart, L., Etheridge, C.J., Coombes, R.C., Buluwela, L. and Miller, A.D. (1998) Endothelial cell transfection with cationic liposomes and herpes simplex-thymidine kinase mediated killing. Gene Ther., 5, 614-620.
42. Labat-Moleur, F., Steffan, A.-M., Brisson, C, Perron, H., Feugeas, O., Furstenberger, P., Oberling, F., Brambilla, E. and Behr, J.-P. (1996) An electron microscopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther., 3, 1010-1017.
43. 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.
44. Rädler, J.O., Koltover, L, Salditt, T. and Safinya, C.R. (1997) Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science, 275, 810-814.
45. Gustafsson, J., Arvidson, G., Karlsson, G. and Almgren, M. (1995) Complexes between cationic liposomes and DNA visualized by cryo-TEM. Biochim. Biophys. Acta, 1235, 305-312.
46. 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.
47. Eastman, S.J., Siegel, C, Tousignant, J., Smith, A.E., Cheng, S.H. and Scheule, R.K. (1997) Biophysical characterisation of cationic lipid:DNA complexes. Biochim. Biophys Acta, 1325, 41-62.
48. Sternberg, B., Sorgi, F.L. and Huang, L. (1994) New structures in complex formation between DNA and cationic liposomes visualised by freeze-fracture electron microscopy. FEBS Lett., 356, 361-366.
49. Stribling, R., Brunette, E., Liggitt, D., Gaensler, K. and Debs, R. (1992) Aerosol gene delivery in vivo. Proc. Natl Acad. Sci. USA, 89, 11277-11281.
50. Caplen, N.J., Alton, E.W.F.W., Middleton, P.G., Dorin, J.R., Stevenson, B.J., Gao, X., Durham, S.R., Jeffery, P.K., Hodson, M.E., Coutelle, C, Huang, L., Porteous, D.J., Williamson, R. and Geddes, D.M. (1995) Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat. Med., 1, 39-46.
51. Alton, E.W.F.W., Stern, M., Farley, R. et al. (1999) Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet, 353, 947-954.
52. Zhu, N., Liggitt, D., Liu, Y. and Debs, R. (1993) Systemic gene-expression after intravenous DNA delivery into adult mice. Science, 261, 209-211.
53. Rogy, M.A., Auffenberg, T., Espat, N.J., Philip, R., Remick, D., Wollenberg, G.K., Copeland III, E.M. and Moldawer, L.L. (1995) Human tumor necrosis factor receptor (p55) and interleukin 10 gene transfer in the mouse reduces mortality to lethal endotoxemia and also attenuates local inflammatory responses. J. Exp. Med., 181, 2289-2293.
54. Liu, Y., Liggitt, D., Zhong, W., Tu, G., Gaensler, K. and Debs, R. (1995) Cationic liposome-mediated intravenous gene delivery. J. Biol. Chem., 270, 2486-24870.
55. Thierry, A.R., Lunardiiskandar, 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.
56. Murray, K.D., McQuillin, A., Stewart, L., Etheridge, C.J., Cooper, R.G., Miller, A.D. and Gurling, H.M.D. (1999) Cationic liposome-mediated DNA transfection in organotypic expiant cultures of the ventral mesencephalon. Gene Ther., 6, 190-197.
57. Stephan, D.J., Yang, Z.-Y., San, H., Simari, R.D., Wheeler, C.J., Feigner, P.L., Gordon, D., Nabel, G.J. and Nabel, E.G. (1996) A new cationic liposome DNA complex enhances the efficiency of arterial gene transfer in vivo. Hum. Gene Ther., 7, 1803-1812.
58. Seung, L.P., Mauceri, H.J., Beckett, M.A., Hallahan, D.E., Hellman, S. and Weichselbaum, R.R. (1995) Genetic radiotherapy overcomes tumor resistance to cytotoxic agents. Cancer Res., 55, 5561-5565.
59. Zhu, J., Zhang, L., Hanisch, U.K., Feigner, P.L. and Reszka, R. (1996) A continuous intracerebral gene delivery system for in vivo liposome-mediated gene therapy. Gene Ther., 3, 472-476.
60. Wright, M.J., Rosenthal, E., Stewart, L., Wightman, L.M.L., Miller A.D., Latchman, D.S. and Marber, M.S. (1998) B-Galactosidase staining following intracoronary infusion of cationic liposomes in the in vivo rabbit heart is produced by microinfarction rather than effective gene transfer: a cautionary tale. Gene Ther., 5, 301-308.
61. Hong, K., Zheng, W., Baker, A. and Papahadjopoulos, D. (1997) Stabilization of cationic liposome-plasmid DNA complexes by polyamines and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery. FEBS Lett., 400, 233-237.
62. Hofland, H.E.J., Shephard, L. and Sullivan, S.M. (1996) Formation of stable cationic lipid/DNA complexes for gene transfer. Proc. Natl Acad. Sci. USA, 93, 7305-7309.
63. Litzinger, D.C., Brown, J.M., Wala, I., Kaufman, S.A., Van, G.Y., Farrell, C.L. and Collins, D. (1996) Fate of cationic liposomes and their complex with oligonucleotide in vivo. Biochim. Biophys. Acta, 1281, 139-149.
64. 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.
65. Yang, J.-P. and Huang, L. (1998) Time-dependent maturation of cationic liposome-DNA complex for serum resistance. Gene Ther., 5, 380-387.
66. Liu, Y., Mounkes, L.C., Liggitt, H.D., Brown, C.S., Solodin, I., Heath, T.D. and Debs, R.J. (1997) Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat. Biotech., 15, 167-173.
67. Templeton, N.S., Lasic, D.D., Frederik, P.M., Strey, H.H., Roberts, D.D. and Pavlakis, G.N. (1997) Improved DNA:liposome complexes for increased systemic delivery and gene expression. Nat. Biotech., 15, 647-652.
68. Spragg, D.D., Alford, D.R., Greferath, R., Larsen, C.E., Lee, K.-D., Gurtner, G.C., Cybulsky, M.I., Tosi, P.F., Nicolau, C. and Gimbrone, M.A. Jr. (1997) Immunotargeting of liposomes to activated vascular endothelial cells: a strategy for site-directed delivery in the cardiovascular system. Proc. Natl Acad. Sci. USA, 94, 8795-8800.
69. Simeõs, S., Slepushkin, V., Gaspar, R., Pedroso de Lima, M.C. and Düzgünes, N. (1998) Gene delivery by negatively charged ternary complexes of DNA, cationic liposomes and transferrin or fusigenic peptides. Gene Ther., 5, 955-964.
70. Kamata, H., Yagisawa, H., Takahashi, S. and Hirata, H. (1994) Amphiphilic peptides enhance the efficiency of liposome-mediated DNA transfection. Nucleic Acids Res., 22, 536-537.
71. Budker, V., Gurevich, V., Hagstrom, J.E., Bortzov, F. and Wolff, J.A. (1996) pH-sensitive, cationic liposomes: a new synthetic virus-like vector. Nat. Biotech., 14, 760-764.
72. Legendre, J.Y. and Szoka, F.C. (1992) Delivery of plasmid DNA into mammalian-cell lines using pH-sensitive liposomes: comparison with cationic liposomes. Pharmaceut. Res., 9, 1235-1242.
73. Behr, J.-P. (1996) The proton sponge, a means to enter cells viruses never thought of. M/S-Med. Sci., 12, 56-58.
74. Li, S. and Huang, L. (1997) In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther., 4, 891-900.
75. Li, S., Rizzo, M.A., Bhattacharya, S. and Huang, L. (1998) Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther., 5, 930-937.
76. Yonemitsu, Y., Alton, E.W.F.W., Komori, K., Yoshizumi, T., Sugimachi, K. and Kaneda, Y. (1998) HVJ (Sendai virus) liposome-mediated gene transfer: current status and future perspectives. Int. J. Oncol., 12, 1277-1285.
77. Kaneda, Y., Iwai, K. and Uchida, T. (1989) Increased expression of DNA cointroduced with nuclear-protein in adult-rat liver. Science, 243, 375-378.
78. Saeki, Y., Matsumoto, N., Nakano, Y., Mori, M., Awai, K. and Kaneda, Y. (1997) Development and characterization of cationic liposomes conjugated with HVJ (Sendai virus): reciprocal effect of cationic lipid for in vitro and in vivo gene transfer. Hum. Gene Ther., 8, 2133-2141.
79. Yonemitsu, Y., Kaneda, Y., Muraishi, A., Yoshizumi, T., Sugimachi, K. and Sueishi, K. (1997) HVJ (Sendai virus)-cationic liposomes: a novel and potentially effective liposome-mediated technique for gene transfer to the airway epithelium. Gene Ther., 4, 631-638.
80. Wolfert, M.A. and Seymour, L.W. (1996) Atomic force microscopic analysis of the influence of the molecular weight of poly(L)lysine on the size of polyelectrolyte complexes formed with DNA. Gene Ther., 3, 269-273.
81. Xu, B., Wiehle, S., Roth, J.A. and Cristiano, R.J. (1998) The contribution of poly-L-lysine, epidermal growth factor and streptavidin to EGF/PLL/DNA polyplex formation. Gene Ther., 5, 1235-1243.
82. Tang, M.X. and Szoka, F.C. (1997) The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther., 4, 823-832.
83. Wolfert, M.A., Schacht, E.H., Toncheva, V., Ulbrich, K., Nazarova, O. and Seymour, L.W. (1996) Characterization of vectors for gene therapy formed by self-assembly of DNA with synthetic block co-polymers. Hum. Gene Ther., 7, 2123-2133.
84. Wu, G.Y. and Wu, C. H. (1988) Evidence for targeted gene delivery to HepG2 hepatoma-cells in vitro. Biochemistry, 27, 887-892.
85. Michael, S.I. and Curiel, D.T. (1994) Strategies to achieve targeted gene delivery via the receptor-mediated endocytosis pathway. Gene Ther., 1, 223-232.
86. Findeis, M.A., Wu, C.H. and Wu, G.Y. (1994) Ligand-based carrier systems for delivery of DNA to hepatocytes. Meth. Enzymol, 247, 341-351.
87. Kollen, W.J.W., Midoux, P., Erbacher, P., Yip, A., Roche, A.C., Monsigny, M., Glick, M.C. and Scanlin, T.F. (1996) Gluconoylated and glycosylated polylysines as vectors for gene transfer into cystic fibrosis airway epithelial cells. Hum. Gene Ther., 7, 1577-1586.
88. 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.
89. Cotten, M., Langlerouault, 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.
90. Plank, C, Zatloukal, K., Cotten, M., Mechtler, K. and Wagner, E. (1992) Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconjugate Chem., 3, 533-539.
91. 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.
92. Erbacher, P., Roche, A.C., Monsigny, M. and Midoux, P. (1996) Putative role of chloroquine in gene transfer into a human hepatoma cell line by DNA lactosylated polylysine complexes. Exp. Cell Res., 225, 186-194.
93. Plank, C., Oberhauser, B., Mechtler, K., Koch, C. and Wagner, E. (1994) The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene transfer systems. J. Biol. Chem., 17, 12918-12924.
94. 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.
95. Wagner, E., Zatloukal, K., Cotten, M., Kirlappos, H., Mechtler, K., Curiel, D.T. and Birnstiel, M.L. (1992) Coupling of adenovirus to transferrinpolylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc. Natl Acad. Sci. USA, 89, 6099-6103.
96. Wu, G.Y., Wilson, J.M., Shalaby, F., Grossman, M., Shafritz, D.A. and Wu, C.H. (1991) Receptor-mediated gene delivery in vivo-partial correction of genetic analbuminemia in nagase rats. J. Biol. Chem., 266, 14338-14342.
97. Perales, J.C., Ferkol, T., Molas, M. and Hanson, R.W. (1994) An evaluation of receptor-mediated gene-transfer using synthetic DNA-ligand complexes. Eur. J. Biochem., 226, 255-266.
98. 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.
99. Hart, S.L., Harbottle, R.P., Cooper, R., Miller, A., Williamson, R. and Coutelle, C (1995) Gene delivery and expression mediated by an integrin-binding peptide. Gene Ther., 2, 552-554.
100. Harbottle, R.P., Cooper, R.G., Hart, S.L., Ladhoff, A., McKay, T., Knight, A.M., Wagner, E., Miller, A.D. and Coutelle, C (1998) An RGD-oligolysine peptide: a prototype construct for integrin-mediated gene delivery. Hum. Gene Ther., 9, 1037-1047.
101. Cooper, R.G., Harbottle, R.P., Schneider, H., Coutelle, C and Miller, A.D. (1999) Peptide mini-vectors for gene delivery. Angew. Chem. Intl. Ed., 38, 1949-1952.
102. Wickham, T.J., Mathias, P., Cheresh, D.A. and Nemerow, G.R. (1993) Integrins AvB3 and AvB5 promote adenovirus internalization but not virus attachment. Cell, 73, 309-319.
103. Bergelson, J.M., Shepley, M.P., Chan, B.M.C, Hemler, M.E. and Finberg, R.W. (1992) Identification of the integrin VLA-2 as a receptor for echovirus-1. Science, 255, 1718-1720.
104. Logan, D., Abughazaleh, R., Blakemore, W., Curry, S., Jackson, T., King, A., Lea, S., Lewis, R., Newman, J., Parry, N., Rowlands, D., Stuart, D. and Fry, E. (1993) Structure of a major immunogenic site on foot-and-mouth disease virus. Nature, 362, 566-568.
105. Hynes, R.O. (1992) Integrins: versatility, modulation, and signaling in cell adhesion. Cell, 69, 11-25.
106. Pierschbacher, M.D. and Ruoslahti, E. (1984) Cell attachment activity of fibronectin can be duplicated by small synthetic fragments of the molecule. Nature, 309, 30-33.
107. Haensler, J. and Szoka, F. C. Jr. (1993) Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconjugate Chem., 4, 372-379.
108. Tang, M.X., Redemann, C.T. and Szoka, F.C. Jr. (1996) In vitro gene delivery by degraded polyamidoamine dendrimers. Bioconjugate Chem., 7, 703-714.
109. Boussif, O., Lezoualch, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B. and Behr, J.-P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl Acad. Sci. USA, 92, 7297-7301.
110. Boussif, O., Zanta, M.A. and Behr, J.-P. (1996) Optimized galenics improve in vitro gene transfer with cationic molecules up to 1000-fold. Gene Ther., 3, 1074-1080.
111. Zanta, M.A., Boussif, O., Adib, A. and Behr, J.-P. (1997) In vitro gene delivery to hepatocytes with galactosylated polyethylenimine. Bioconjugate Chem., 8, 839-844.
112. Kircheis, R., Kichler, A., Wallner, G., Kursa, M., Ogris, M., Felzmann, T., Buchberger, M. and Wagner, E. (1997) Coupling of cell-binding ligands to polyethylenimine for targeted gene delivery. Gene Ther., 4, 409-418.
113. Erbacher, P., Remy, J.-S. and Behr, J.-P. (1999) Gene transfer with synthetic virus-like particles via the integrin-mediated endocytosis pathway. Gene Ther., 6, 138-145.
114. Abdallah, B., Hassan, A., Benoist, C, Goula, D., Behr, J.-P. and Demeneix, B.A. (1996) A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine. Hum. Gene Ther., 7, 1947-1954.
115. Goula, D., Remy, J.-S., Erbacher, P., Wasowicz, M., Levi, G, Abdallah, B. and Demeneix, B.A. (1998) Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervous system. Gene Ther., 5, 712-717.
116. Ferrari, S., Moro, E., Pettenazzo, A., Behr, J.-P., Zacchello, F. and Scarpa, M. (1997) ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther., 4, 1100-1106.
117. Goula, D., Benoist, C, Maniero, S., Merlo, G, Levi, G. and Demeneix, B.A. (1998) Polyethylenimine-based intravenous delivery of transgenes to mouse lung. Gene Ther., 5, 1291-1295.
118. Kren, B.T., Bandyopadhyay, P. and Steer, C.J. (1998) In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat. Med., 4, 285-290.
119. Goldman, C.K., Soroceanu, L., Smith, N., Gillespie, G.Y., Shaw, W., Burgess, S., Bilbao, G. and Curiel, D.T. (1997) In vitro and in vivo gene delivery mediated by a synthetic polycationic amino polymer. Nat. Biotech., 15, 462-466.
120. Kabanov, A.V., Astafieva, I.V., Maksimova, I.V., Lukanidin, E.M., Georgiev, G.P. and Kabanov, V.A. (1993) Efficient transformation of mammalian-cells using DNA interpolyelectrolyte complexes with carbon-chain polycations. Bioconjugate Chem., 4, 448-454.
121. Astafieva, I., Maksimova, I., Lukanidin, E., Alakhov, V. and Kabanov, A. (1996) Enhancement of the polycation-mediated DNA uptake and cell transfection with pluronic P85 block copolymer. FEBS Lett., 389, 278-280.
122. Cherng, J.Y., van de Wetering, P., Talsma, H., Crommelin, D.J.A. and Hennink, W.E. (1996) Effect of size and serum proteins on transfection efficiency of poly((2-dimethylamino)ethyl methacrylate)-plasmid nanoparticles. Pharmaceut. Res., 13, 1038-1042.
123. van de Wetering, P., Cherng, J.Y., Talsma, H. and Hennink, W.E. (1997) Relation between transfection efficiency and cytotoxicity of poly((2-dimethylamino)ethyl methacrylate)/plasmid complexes. J. Control. Rel., 49, 59-69.
124. van de Wetering, P., Cherng, J.Y., Talsma, H., Crommelin, D.J.A. and Hennink, W.E. (1998) 2-(Dimethylamino)ethyl methacrylate based (copolymers as gene transfer agents. J. Control. Rel, 53, 145-153.
125. Fritz, J.D., Herweijer, H., Zhang, G and Wolff, J.A. (1996) Gene transfer into mammalian cells using histone-condensed plasmid DNA. Hum. Gene Ther., 7, 1395-1404.
126. Mistry, A.R., Falciola, L., Monaco, L., Tagliabue, R., Acerbis, G., Knight, A., Harbottle, R.P., Soria, M., Bianchi, M.E., Coutelle, C. and Hart, S.L. (1997) Recombinant HMG1 protein produced in Pichia pastoris: a nonviral gene delivery agent. BioTechniques, 22, 718-729.
127. Gottschalk, S., Sparrow, J.T., Hauer, J., Mims, M.P., Leiand, F.E., Woo, S.L.C, and Smith, L.C. (1996) A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. Gene Ther., 3, 448-457.
128. Wadhwa, M.S., Collard, W.T., Adami, R.C., McKenzie, D.L. and Rice, K.G. (1997) Peptide-mediated gene delivery: Influence of peptide structure on gene expression. Bioconjugate Chem., 8, 81-88.
129. Legendre, J.Y., Trzeciak, A., Bohrmann, B., Deuschle, U., Kitas, E. and Supersaxo, A. (1997) Dioleoylmelittin as a novel serum-insensitive reagent for efficient transfection of mammalian cells. Bioconjugate Chem., 8, 57-63.
130. Wyman, T.B., Nicol, F., Zelphati, O., Scaria, P.V., Plank, C. and Szoka, F.C. Jr. (1997) Design, synthesis and characterization of a cationic peptide that binds to nucleic acids and permeabilizes bilayers. Biochemistry, 36, 3008-3017.
131. Murphy, J.E., Uno, T., Hamer, J.D., Cohen, F.E., Dwarki, V. and Zuckermann, R.N. (1998) A combinatorial approach to the discovery of efficient cationic peptoid reagents for gene delivery. Proc. Natl Acad. Sci. USA, 95, 1517-1522.
132. Zuckermann, R.N., Kerr, J.M., Kent, S.B.H. and Moos, W.H. (1992) Efficient method for the preparation of peptoids [oligo(N-substituted glycines)] by submonomer solid-phase synthesis. J. Am. Chem. Soc, 114, 10646-10647.
133. Trubetskoy, V.S, Budker, V.G. Hanson, L.J, Slattum, P.M., Wolff, J.A. and Hagstrom, J.E. (1998) Self-assembly of DNA-polymer complexes using template polymerization. Nucleic Acids Res., 26, 4178-4185.
134. Uherek, C, Fominaya, J. and Wels, W. (1998) A modular DNA carrier protein based on the structure of diphtheria toxin mediates target cell-specific gene delivery. J. Biol. Chem., 273, 8835-8841.
135. Fominaya, J, Uherek, C. and Wels, W. (1998) A chimeric fusion protein containing transforming growth factor-oc mediates gene transfer via binding to the EGF receptor. Gene Ther., 5, 521-530.
136. Sebestyén, M.G., Ludtke, J.J, Bassik, M.C, Zhang, G, Budker, V, Lukhtanov, E.A, Hagstrom, J.E. and Wolff, J.A. (1998) DNA vector chemistry: the covalent attachment of signal peptides to plasmid DNA. Nat. Biotech., 16, 80-85.
137. Zanta, M.A, Belguise-Valladier, P. and Behr, J.-P. (1999) Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl Acad. Sci. USA, 96, 91-96.
138. Sanford, J.C, Klein, T.M, Wolf, E.D. and Allen, N. (1987) Delivery of substances into cells and tissues using a particle bombardment process. Part. Sci. Technol, 5, 27-37.
139. 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.
140. Shark, K.B., Smith, F.D., Harpending, P.R, Rasmussen, J.L, and Sanford, J.C. (1991) Biolistic transformation of a prokaryote, Bacillus megaterium. Appl. Environ. Microbiol., 57, 480-485.
141. Johnston, S.A., Anziano, P.Q., Shark, K, Sanford, J.C. and Butow, R.A. (1988) Mitochondrial transformation in yeast by bombardment with microprojectiles. Science, 240, 1538-1541.
142. Daniell, H, Krishnan, M. and McFadden, B.F. (1991) Transient expression of beta-glucuronidase in different cellular compartments following Biolistic delivery of foreign DNA into wheat leaves and calli. Plant Cell Rep., 9, 615-619.
143. Mathiesen, I. (1999) Electropermeabilization of skeletal muscle enhances gene transfer in vivo. Gene Ther., 6, 508-514.
144. Rizzuto, G, Cappelletti, M, Maione, D. et al. (1999) Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation. Proc. Natl Acad. Sci. USA, 96, 6417-6422.
145. Potter, H. (1988) Electroporation in biology: methods, applications and instrumentation. Anal. Biochem., 174, 361-373.