Non-viral peptide-based approaches to gene delivery

Journal of Drug Targeting

Volumn 7, Issue 4, 1999, Pages 249-268

  Mahato, Ram I ^a  

^a UNIVERSITY OF UTAH   (United States)

Author keywords

Compacted DNA; Gene therapy; Intracellular trafficking; Peptide; Polylysine; Stability

Indexed keywords

DNA; NUCLEASE;

CELL NUCLEUS; CYTOPLASM; ENDOSOME; GENE TARGETING; GENE TRANSFER; HYDROPHILICITY; INTRACELLULAR TRANSPORT; PLASMID; PRIORITY JOURNAL; REVIEW;

EID: 0033376405     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.3109/10611869909085509     Document Type: Review

References (135)

1. Aronsohn, A.I. and Hughes, J.A. (1998) Nuclear localization signal peptides enhance cationic liposome-mediated gene therapy. J. Drug Target. 5, 163-169.
2. Arscott, P.G., Li, A.Z. and Bloomfield, V.A. (1990) Condensation of DNA by trivalent cations. 1. Effects of DNA length and topology on the size and shape of condensed particles. Biopolymers 30, 619-630.
3. Asayama, S., Nagawa, M., Takei, Y., Akaike, T. and Maruyama, A. (1998) Hyaluronic acid grafted comb-type polylysines; Synthesis of novel polyampholyte comb-type copolymers consisting of polylysine back bone and hyaluronic acid side chains for a DNA carrier. Bioconjugate Chem. 9, 476-481.
4. Baaz, J.E., Bruno, M.D., Ciraolo, P.J., Glasser, S.W., Stripp, B.R., Smyth, K.L. and Korfhagen, T.R. (1995) Utilization of modified surfactant-associated protein B for delivery of DNA to airway cells in culture. Proc. Natl. Acad. Sci. USA 91, 2547-2551.
5. Basak, A., Boudreault, A., Chen, A., Chretien, M., Seidah, N.G. and Lazure, C. (1995) Application of the multiple antigenic peptides (MAP) strategy to the production of prohormone convertases antibodies: synthesis, characterization and use of 8-branched immunogenic peptides. J. Peptide Sci. 1, 385-395.
6. Bijsterbosch, M.K., Ziere, G.J. and van Berkel, T.J. (1989) Lactosylated low density lipoprotein: a potential carrier for the site-specific delivery of drugs to Kupffer cells. Mol. Pharmacol. 36, 484-489.
7. Blessing, T., Remy, J.-S. and Behr, J.-P. (1998) Monomolecular collapse of plasmid DNA into stable virus-like particles. Proc. Natl. Acad. Sci. USA 95, 1427-1431.
8. Bloomfield, V.A. (1996) DNA condensation. Curr. Opin. Struct. Biol. 6, 334-341.
9. Bloomfield, V.A. (1998) DNA condensation by multivalent cations. Biopolymers 44, 269-282.
10. Boulikas, T. (1998) Nucleocytoplasmic trafficking: implications for the nuclear import of plasmid DNA during gene therapy. Gene Ther. Mol. Biol. 1, 713-740.
11. Boussif, O., Lezoualc'h, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B. and Behr, J.P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. USA 92, 7297-7301.
12. Brinster, R., Chen, H., Trumbauer, M., Yagle, M. and Palmiter, R. (1985) Factors affecting the efficiency of introducing foreign DNA into mice by microinjecting eggs. Proc. Natl. Acad. Sci. USA 82, 4432-4442.
13. Buschle, M., Cotton, 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.
14. Cantor, C.R. and Schimmel, P.R. (1980) Biophysical Chemistry, Part II, Freeman & Co., New York, p. 554.
15. Capecchi, M.R. (1980) High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cell 22, 479-488.
16. Chan, C.K., Hubner, S., Hu, W. and Jans, D.A. (1998) Mutual exclusivity of DNA binding and nuclear localization signal recognition by the yeast transcritpion factor GAL4: implications for non-viral DNA delivery. Gene Ther. 5, 1204-1212.
17. Cheng, P.-W. and Yin, W. (1994) Lectin conjugate-directed gene transfer to airway epithelial cells. Biochem. Biophys. Res. Commun. 205, 826-833.
18. Chen, J., Stickles, R.J. and Daichendi, K.A. (1994a) Galactosylated histone-mediated gene transfer and expression. Hum. Gene Ther. 5, 429-435.
19. Chen, J., Gamou, S., Takayanagi, A. and Shimizu, N. (1994b) A novel gene delivery system using EGF receptor-mediated endocytosis. FEBS Lett. 338, 167-169.
20. Chiu, M.H., Tamura, T., Wadhwa, M.S. and Rice, K.G. (1994) In vivo targeting function of N-linked oligosaccharides with terminating galactase and N-acetylgalactosamine residues. J. Biol. Sci. 269: 16195-16202.
21. Colin, M., Harbottle, R.P., Knight, A., Kornprobst, M., Cooper, R.G., Mike, A.D., Trugnan, G., Capeau, J., Coutelle, C. and Brahimi-Horn, M.C. (1998) Liposomes enhance delivery and expression of an RGD-oligolysine gene transfer vector in human tracheal cells. Gene Ther. 5, 1488-1498.
22. Collas, P., Husebye, H. and Alestrom, P. (1996) The nuclear localization sequence of the SV40 T antigen promotes transgene uptake and expression in zebrafish embryo nuclei. Transgenic Res. 5, 451-458.
23. Cooper, M.J. (1996) Non-infectious gene transfer and expression systems for cancer gene therapy. Sem. Oncol. 23, 172-187.
24. Dang, C.V. and Lee, W.M. (1988) Identification of the human c-myc protein nuclear translocation signal. Mol. Cell Biol. 8, 4048-4054.
25. Dash, P.R., Read, M.L., Barrett, L.B., Wolfert, M.A. and Seymour, L.W. (1999) Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery. Gene Ther. 6, 643-650.
26. 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, 3667-3670.
27. Duguid, J.G., Li, C., Shi, M., Logan, M.J., Alila, H., Rolland, A., Tomlinson, E., Sparrow, J.T. and Smith, L.C. (1998) A physicochemical approach for predicting the effectiveness of peptide-based gene delivery systems for use in plusmid-based gene therapy. Biophysical J. 74, 2802-2814.
28. Eickbush, T.H. and Moundrianakis, E.N. (1978) The compaction of DNA helices into either continuous supercoils or folded-fiber rods and toroids. Cell 13, 295-306.
29. Emi, N., Kidoaki, S., Yoshikawa, K. and Saito, H. (1997) Gene transfer mediated by polyarginine requires a formation of big carrier-complex of DNA aggregate. Biochem. Biophys. Res. Commun. 231, 421-424.
30. Epand, R.M., Shai, Y., Segrest, J.P. and Anantharamaih, G.M. (1995) Mechanisms for the modulation of membrane bilayer properties by amphipathic helical peptides. Biopolymers 37, 319-338.
31. Erbacher, P., Roche, A.C., Monsigny, M. and Midoux, P. (1995) Glycosylated polylysine DNA complexes: Gene transfer efficiency in relation with the size and the sugar substitution level of glycosylated polylysines and with the plasmid size. Bioconjugate Chem. 6, 401-410.
32. Erbacher, P., Roche, A.C., Monsigny, M. and Midoux, P. (1997) The reduction of the positive charge of polylysine by partial gluconoylation increases the transfection efficiency of polylysine/DNA complexes. Biophys. Biochim. Acta 1324, 27-36.
33. Fasbender, A.J., Zabner, J., Zeiher, B.G. and Welsh, M.J. (1997) A low rate of cell proliferation and reduced DNA uptake limit cationic lipid-mediated gene transfer to primary cultures of ciliated human airway epithelia. Gene Ther. 4, 1173-1180.
34. Fattal, E., Nir, S., Parente, R.A. and Szoka, F.C. (1994) Poreforming peptides induce rapid phospholipid flip-flop in membranes. Biochemistry 33, 6721-6731.
35. 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) Lipofectin: A highly efficient, lipi-mediated DNA transfection procedures. Proc. Natl. Acad. Sci. USA 84, 7413-7417.
36. Ferkol, T., Kartzel, 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.
37. Ferkol, T., Perales, J.C., Eckman, E., Kaetzel, C.S., Hanson, R.W. and Davis, P.B. (1995) Gene transfer into the respiratory epithelium of animals by targeting the polymeric immunoglobulin receptor. J. Clin. Invest. 95, 493-502.
38. Ferkol, T., Pellicena-Palle, A., Eckman, E., Perales, J.C., Trzaska, T., Tosi, M., Redline, R. and Davis, P.B. (1996) Immunologic responses to gene transfer into mice via the polymeric immunoglobulin receptor. Gene Ther. 3, 669-678.
39. Ferkol, T., Perales, J.C., Mularo, F. and Hansen, R.W. (1996) Receptor mediated gene transfer into macrophages. Proc. Natl., Acad. Sci. USA 93, 101-105.
40. Ferrari, M.E., Ngugen, C.M., Zelphati, O., Tsai, Y. and Felgner, P.L. (1998) Analytical methods for the characterization of cationic lipid-nucleic acid complexes. Hum. Gene Ther. 9, 341-351.
41. Fraley, R., Subramani, S., Berg, P. and Papahadjopoulos, D. (1980) Introduction of liposome-encapsulated SV40 DNA into cells. J. Biol. Chem. 255, 10 431-10 435.
42. Gharakhanian, E., Takahashi, J. and Kasamatsu, H. (1987) The carboxyl 35 amino acids of SV40 VP3 are essential for its nuclear accumulation. Virology 157, 440-448.
43. Görlich, D. and Mattaj, I.W. (1996) Nucleocytoplasmic transport. Science 271, 1513-1518.
44. Gottschalk, S., Christiano, 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.
45. Gottschalk, S., Sparrow, J.T., Hauer, J., Mims, M.P., Leland, 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.
46. Hagstrom, J.E., Sebestyen, M.G., Budker, V., Ludtke, J.J., Fritz, J.D. and Wolff, J.A. (1996) Complexes of non-cationic liposomes and histone H1 mediate efficient transfection of DNA without encapsulation. Biochim. Biophys. Acta 1284, 47-55.
47. Hall, M.N., Hereford, L. and Herskowitz, I. (1984) Targeting of E. coli β-galactosidase to the nucleus in yeast. Cell 36, 1057-1065.
48. Hanson, R.W., Perales, J.C. and Ferkol, T.W. (1999) Compacted nucleic acids and their delivery to cells. US Patent 5,877,302.
49. 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 protoype construct for integrin-mediated gene delivery. Hum. Gene Ther. 9, 1037-1047.
50. Hart, S.L., Harbottle, R.P., Cooper, R., Miller, A., Williamson, R. and Couttele, C. (1995) Gene delivery and expression mediated by an integrin-binding peptide. Gene Ther. 2, 552-554.
51. 3 antigonists. J. Am. Chem. 118, 7461-7472.
52. Huckett, B., Ariatti, M. and Hawtrey, A.O. (1990) Evidence for targeted gene transfer by receptor-mediated endocytosis: Stable expression following insulin-directed entry of NEO in HepG2 cells. Biochem. Pharmacol. 40, 253-263.
53. Hud, N.V., Allen, M.J., Downing, K.H., Lee, J. and Balhorn, R. (1993) Identification of the elemental packing unit of DNA in mammalian sperm cells by atomic force microscopy. Biochem. Biophy. Res. Commun. 193, 1347-1354.
54. Ibanez, M., Gariglio, P., Chavez, P., Santiago, R., Wong, C. and Baeza, I. (1996) Spermidine-condensed DNA and cone-shaped lipids improve delivery and expression of exogenous DNA transfer by liposomes. Biochem. Cell Biol. 74, 633-643.
55. Jiang, C., O'Connor, S.P., Fong, S.L., Wang, K.X., Marshall, J., Williams, J.L., Wilburn, B., Echeland, Y. and Cheng, S.H. (1998) Efficiency of cationic lipid-mediated transfection of polarized and differentiated airway epithelial cells in vitro and in vivo. Hum. Gene Ther. 9, 1531-1542.
56. Kalderon, D., Richardson, W.D., Markham, A.F. and Smith, A.E. (1984) Sequence requirements for nuclear location of simian virus 40 large T-antigen. Nature 311, 109-118.
57. Kawakami, S., Yamashita, F., Nishikawa, M., Tukakura, Y. and Hashida, M. (1998) Asialoglycoprotein receptor-mediated gene transfer using novel galactosylated cationic liposomes. Biochem. Biophys. Res. Commun. 252, 78-83.
58. Kleinschmidt, J.A. and Steiter, A. (1988) Identification of domains involved in nuclear uptake and histone binding of protein N1 of Xenopus laevis. EMBO J. 7, 1605-1614.
59. Kohn, W.D., Monera, O.D., Kay, C.M. and Hodges, R.S. (1995) The effects of interhelical electrostatic repulsion between glutamic acid residues in controlling the dimerization and stability of two-stranded α-helical coiled-coils. J. Biol. Chem. 270, 25 495-25 506.
60. Kwoh, D.Y., Coffin, C.C., Lollo, C.P., Jovernal, J., Banaszczyk, M.G., Mullen, P., Phillips, A., Amini, A., Fabrycki, J., Bartholomew, R.M., Brostoff, S.W. and Carlo, D.J. (1999) Stabilisation of poly-L-lysine/DNA polyplexes for in vivo gene delivery to the liver. Biochim. Biophys. Acta 1444, 171-190.
61. Kuntz Jr., I.D. and Kauzmann, W. (1974) Hydration of protein and polypeptides. In: Advances in Protein Chemistry (C.B. Anfinsen, J.T. Edsall and F.M. Richards, Eds.), Acadmic Press, New York, p. 244.
62. Ladokhin, A.S., Selsted, M.E. and White, S.H. (1997) Sizing membrane pores in lipid vesicles by leakage of co-encapsulated markers: pore formation by melittin. Biophys. J. 72, 1762-1766.
63. Lacmmli, U.K., Paulson, J.R. and Hitchins, V. (1974) Maturation of the head of bacteriophage T4. V. A possible DNA packaging mechanism: in vitro cleavage of the head proteins and the structures of the core of the polyhead. J. Supramol. Struct. 2, 276-301.
64. Lanford, R.E., Kanda, P. and Kennedy, R.C. (1986) Induction of nuclear transport with a synthetic peptide homologous to the SV40 T antigen transport signal. Cell 46, 575-582.
65. 2 terminus of influenza HA-2. J. Biol. Chem. 262, 6500-6505.
66. Lechardeur, D., Sohn, K.-J., Haardt, M., Joshi, P.B., Monck, M., Graham, R.W., Beatly, B., Squire, J., O'Brodovich, H. and Lukacs, G.L. (1999) Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer. Gene Ther. 6, 482-497.
67. Ledley, F.D. (1996) Pharmaceutical approaches to somatic gene therapy. Pharm. Res. 13, 1595-1614.
68. Legendre, J.Y. and Szoka Jr., F.C. (1993) Cyclic amphipathic peptide-DNA complexes mediate high efficiency transfection of adherent mammalian cells. Proc. Natl. Acad. Sci. USA 90, 893-897.
69. Legendre, J.-Y., Trzeciak, A., Bur, D., Deusche, U. and Supersaxo, A. (1997) N-acyl-(α,γ-diaminobutaric acid)n hydrazide as an efficient gene transfer vector in mammalian cells in culture. Pharm. Res. 14, 619-624.
70. Loewinger, L. and McKeon, F. (1988) Mutations in the nuclear lamin proteins resulting in their aberrant assembly in the cytoplasm. EMBO J. 7, 2301-2309.
71. Lyons, R.H., Ferguson, B.Q. and Rosenberg, M. (1987) Pentapeptide nuclear localization signal in adenovirus E1A. Mol. Cell Biol. 7, 2451-2456.
72. MacLaughlin, F.C., Mumper, R.J., Wang, J., Tagliaferri, F., Hnichliffe, M. and Rolland, A.P. (1998) Chitosan and depolymerized chitosan oligomers as a condensing carriers for in vivo plasmid delivery. J. Control. Rel. 56, 259-272.
73. Mahato, R.I., Takakura, Y. and Hashida, M. (1997) Non-viral vectors for in vivo gene delivery: Physicochemical and pharmacokinetic considerations. Crit. Rev. Ther. Drug. Carrier. Syst. 14, 133-172.
74. Mahato, R.I., Smith, L.C. and Rolland, A. (1999) Pharmaceutical perspectives of non-viral gene therapy. Adv. Genet. 41, 95-156.
75. Makkerh, J.P.S., Dingwall, C. and Faskey, R.A. (1996) Comparative mutagenesis of nuclear localization signals reveals the importance of neutral and acidic amino acids. Curr. Biol. 6, 1025-1027.
76. Malim, M., Tiley, L.S., McCarn, D.F., Rusche, J.R., Hauber, J. and Cullen, B.R. (1990) HIV-1 structural gene expression requires binding of the rev trans-activator to its RNA target. Cell 60, 675-683.
77. Middaugh, C.R., Evans, R.K., Montgomery, D.L. and Casimiro, D.L. (1998) Analysis of plasmid DNA from a pharmaceutical perspective. J. Pharm. Sci. 87, 130-146.
78. Midoux, P., Kichler, A., Boutin, V., Maurizot, J.-C. and Monsigny, M. (1998) Membrane permeabilization and efficient gene transfer by a peptide containing several histidines. Bioconj. Chem. 9, 260-267.
79. Mirzayans, R., Aubin, R. and Paterson, M. (1992) Differential expression and stability of foreign genes introduced into human fibroblasts by nuclear versus cytoplasmic microinjection. Mutat. Res. 281, 115-122.
80. Moradpour, D., Schauer, J.I., Zurawski Jr., V.R., Wands, J.R. and Bouten, R.H. (1996) Efficient gene transfer into mammalian cells with cholesteryl-spermidine. Biochem. Biophys. Res. Commun. 221, 82-88.
81. Moreland, R.B., Langevin, G.F., Singer, R.H., Garcea. R.L. and Hereford, L.M. (1987) Amino acid sequences that determine the nuclear localization of yeast histone 2B. Mol. Cell Biol. 7, 4048-4057.
82. Mortimer, I., Tam, P., MacLahan, I., Graham, R.W., Saravolac, E.G. and Joshi, P.B. (1999) Cationic lipid-mediated transfection of cells in culture requires mitotic activity. Gene Ther. 6, 403-411.
83. Murphy, J.E., Uno, T., Hames, 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.
84. Nead, M.A. and McCance, D.J. (1995) Poly-L-ornithine-mediated transfection of human keratinocytes. J. Invest. Dermatol. 105, 668-671.
85. Nigg, E.A. (1997) Nucleoctoplasmic transport: signals, mechanisms and regulation. Nature 386, 779-787.
86. Niidome, T., Ohmori, N., Ichinose, A., Wada, A., Mihara, H., Hirayama, T. and Aoyagi, H. (1997) Binding of cationic alphahelical peptides to plasmid DNA and their gene transfer abilities into cells. J. Biol. Chem. 272, 15 307-15 312.
87. Nishikawa, M., Takemura, S. Takakura, Y. and Hashida, M. (1998) Targeted delivery of plasmid DNA to hepatocytes in vivo: Optimization of the pharmacokinetics of plasmid DNA galactosylated poly(L-lysine) complexes by controlling then physicochemical properties. J. Pharmacol. Exp. Ther. 287, 408-415.
88. Parente, R.A., Nadasdi, L., Subbarao, N.K. and Szoka Jr., F.C. (1990) Association of a pH-sensitive peptide membrane vesicles, role of amino acid sequence. Biochemistry 29, 8713-8719.
89. v integrins as receptors for tumor targeting by circulating ligands. Nature Biotechnol. 15, 542-546.
90. Paul, R.W., Weisser, K.E., Loomis, A., Sloane, O.L., LaFoe, D., Atkinson, E.M. and Overell, R.W. (1997) Gene transfer using a novel fusion protein. Gal4 Invasin. Hum. Gene Ther. 8, 1253-1262.
91. Perales, J.C., Ferkol, T., Beegen, H., Ratnoff, O.D. and Hanson, R.W. (1994) 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.
92. Perales, J.C., Grossman, G.A., Molas, M., Liu, G., Ferkol, T., Harpst, J., Oda, H. and Hanson, R.W. (1997) Biochemical and functional characterization of DNA complexes capable of targeting genes to hepatocytes via the asialoglycoprotein receptor. J. Biol. Chem. 272, 7398-7407.
93. Picard, D. and Yamamoto, K.R. (1987) Two signals mediate hormone-dependent nuclear localization of the glucocorticoid receptor. EMBO J. 6, 3333-3340.
94. Plank, C., Oberhauser, B., Mechtler, K., Koch, C. and Wagner, E. (1996) Activation of the compliment system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum. Gene Ther. 7, 1437-1446.
95. Plank, C., Zauner, W. and Wagner, E. (1998) Application of membrane-active peptides for drug and gene delivery across cellular membrane. Adv. Drug Del. Rev. 34, 21-35.
96. Poulat, F., Girard, F., Chevran, M.P., Goze, C., Rebillard, X., Calas, B., Lamb, N. and Berta, P. (1995) Nuclear localization of the testis determining gene product SRY. J. Cell Biol. 128, 737-748.
97. Pouton, C.W. (1999) Biological barriers to gene transfer. In: Advanced Gene Delivery: From Concepts to Pharmaceutical Products (A. Rolland, Ed.), Harwood Press, pp. 65-102.
98. Pouton, C.W., Lucas, P., Thomas, B.J., Uduechi, A.N., Milroy, D.A. and Moss, S.H. (1998) Polycation-DNA complexes for gene-delivery: A comparison of the biopharmaceutical properties of cationic polypeptides and cationic lipids. J. Control. Rel. 5, 289-299.
99. Qin, L., Pahud, D.R., Ding, Y., Bielinska, A.U., Kukowska-Latallo, J.F., Baker Jr., J.R. and Bromberg, J.S. (1998) Efficient transfer of genes into murine cardiac grafts by Starburst polyamidoamine dendrimers. Hum Gene Ther. 9, 553-560.
100. 2 N-terminal peptides with phospholipid vesicles. Biochemistry 30, 10 211-10 220.
101. Robbins, J., Dilworth, S.M., Laskey, R.A. and Dingwall, C. (1991) Two interdependent basic domains in nucleoplasmin nuclear targeting sequence: identification of a class of bipartite nuclear targeting sequence. Cell 64, 615-623.
102. Rolland, A. (Ed.) (1999). Advanced Gene Delivery: From Concepts to Pharmaceutical Products. Harwood Press.
103. Ross, G.F. Morris, R.E., Ciraolo, G., Huelsman, K., Bruno, M., Whitsett, J.A., Baatz, J.A. and Korfthagen, T.R. (1995) Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture. Hum. Gene Ther. 6, 31-40.
104. Schneider, H., Huse, K., Birkenmeier, G., Otto, A. and Scholz, G.H. (1996) Gene transfer mediated by α2-macroglobulin. Nucleic Acids Res. 24, 3873-3874.
105. Schwarzenberger, P., Spence, S.E., Gooya, J.M., Michiel, D., Curriel, 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.
106. Sebestyen, M.G., Ludtke. J.J., Bassik, M.C., Zhang, G., Budker, V., Lukhtanov, E.A., Hagstrom, J.E. and Wolff, J.A. (1998) DNA vector chemistry: The covalent attachment of signal peptides to plasmid DNA Nature Biotechnol. 16, 80-85.
107. Smith, L.C., Sparrow, J.T. and Woo, S.L. (1996) Nucleic acid transporters for delivery of nucleic acids into cells. PCT WO 96 40958.
108. Sola, B., Staedel, C., Remy. J.S., Behr, A. and Behr, J.P. (1997) Lipospermine-mediated gene transfer techniques in murine cultured cortical cells. J. Neurocsi. Methods 71, 183-186.
109. Sorgi, F.L., Bhattacharya, S. and Huang, L. (1997) Protamine sulfate enhances lipid-mediated gene transfer. Gene Ther. 4, 961-968.
110. Strydom, S., Van Jaarsveld, P., Van Helden, E., Ariatti, M. and Hawtrey, A. (1993) Studies on the transfer of DNA into cells through use of avidin-polylysine conjugates complexed to biotinylated transferrin and DNA. J. Drug Target. 1, 165-74.
111. Subbarao, N.K., Parente, R.A., Szoka Jr., F.C., Nadasdi, L. and Pongracz, K. (1987) pH-dependent bilayer destabilization by an amphipathic peptide. Biochemistry 26, 2964-2972.
112. Tang, M.X. and Szoka, F.C. (1997a) The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther. 4, 823-832.
113. Tang, M.X. and Szoka Jr., F.C. (1997b) Biophysical and colloidal aspects of DNA compaction. In: Self-Assembling Complexes for Gene Delivery: From Chemistry to Clinical Trial (A.V. Kabanov, L.W. Seymour and P.L. Felgner, Eds.), John Wiley & Sons, NY.
114. Thurnher, M., Wagner, E., Clausen, H., Mechtler, K., Rusconi, S., Dinter, A., Birnstiel, M.L., Berger, E.G. and Cotton, M. (1994) Carbohydrate receptor-mediated gene transfer to human T leukaemic cells. Glycobiology 4, 429-435.
115. Tomlinson, E. (1998) Site-specific gene therapy: design and use of plasmid-based gene medicines to control in vivo production, delivery and effect of therapeutic proteins. Peptide and Protein Drug Delivery: Alfred Brenzon Symposium 43, 462-475.
116. Toncheva, V., Wolfert, M.A., Dash, P.R., Oupicky, D., Ulbrich, K., Seymour, L.W. and Schacht, E.H. (1998) Novel vectors for gene delivery formed by self-assembly of DNA with poly(L-lysine) grafted with hydrophilic polymers. Biochem. Biophys. Acta 1380, 354-368.
117. Trubetskoy, V.S., Torchilin, V.S., Kennerl, S.J. and Huang, L. (1992) Use of N-terminal modified poly(L-lysine)-antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells. Bioconj. Chem. 3, 323-327.
118. Wadhwa, M.S., Knoell, D.L., Young, A.P. and Rice, K.G. (1995) Targeted gene delivery with a low molecular weight glycopeptide carrier. Bioconj. Chem. 6, 283-291.
119. Wadhwa, M.S., Collard, W.T., Adami, R.C., McKenzie, D.L. and Rice, K.G. (1997) Peptide-mediated gene delivery: Influence of peptide structures on gene expression. Bioconj. Chem. 8, 81-88.
120. Wagner, E., Zenke, M., Cotton, 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.
121. Wagner, E., Plank, C., Zatloukal, K., Cotton, M. and Birnstiel, M.L. (1992) Influenza virus hemagglutinin HA-2N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: towards a synthetic virus-like gene transfer vehicle. Proc. Natl. Acad. Sci. USA 89, 7934-7938.
122. Wilke, M., Fortunati, E., van den Broek, M., Hoogeveen, A.T. and Scholte, B.J. (1996) Efficacy of a peptide-based gene delivery system depends on mitotic activity. Gene Ther. 3, 1133-1142.
123. Wolfert, M.A. and Seymour, L.W. (1998) Chloroquine and amphipathic peptide helices show synergistic transfection in vitro. Gene Ther. 5, 409-414.
124. 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 copolymers. Hum. Gene Ther. 7, 2123-2133.
125. Wu, G.Y. and Wu, C.H. (1988) Receptor-mediated gene delivery and expression in vivo. J. Biol Chem. 263, 14 621-14 624.
126. Wyman, T.B., Nicol, F., Zelphati, O., Scaria, P.V., Plank, C. and Szoka Jr., F.C. (1997) Design, synthesis and characterization of a cationic peptide that binds to nucleic acids and permeabilizes bilayers. Biochemistry 36, 3008-3017.
127. Xiao, C.-Y., Hubner, S. and Jans, D.A. (1997) SV40 large tumor antigen nuclear import is regulated by the double-stranded DNA-dependent protein kinase site (serine 120) flanking the nuclear localization sequence. J. Biol. Chem. 272, 22 191-22 198.
128. Yoneda, Y., Semba, T., Kaneda, Y., Noble, R.L., Matsuoka, Y., Kurihara, T., Okada, Y. and Imamoto, N. (1992) A long synthetic peptide containing a nuclear localization signal and its flanking sequences of SV40 T-antigen directs the transport of IgM into the nucleus efficiently. Exp. Cell Res. 201, 313-320.
129. Yoshikawa, Y. and Yoshikawa, K. (1995) Diaminoalkanes with an odd number of carbon atoms induce compaction of a single double-stranded DNA chain. FEBS Lett. 361, 277-281.
130. 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, 18 997-19 007.
131. Zamyatin, A.A. (1972) Protein volume in solution. Prog. Biophys. Mol. Biol. 24, 107-123.
132. 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.
133. Zatloukal, K., Wagner, E., Cotton, M., Phillips, S., Plank, C., Steinlein, P., Curriel, D.T. and Birnstiel, M.L. (1992) Transferrinfection: a highly efficient way to express gene constructs in eukaryotic cells. Ann. NY Acad. Sci. 660, 136-153.
134. Ziady, A.-G., Perales, J.C., Ferkol, T., Gerken, T., Beegen, H., Perlmutter, D.H. and Davis, P.B. (1997) Gene transfer into hepatoma cell lines via the serpin enzyme complex receptor. Am. J. Physiol. 273, G545-G552.
135. Ziady, A.-G., Ferkol, T., Dawson, D.V., Perlmutter, D.H. and Davis, P.B. (1999) Chain length of the polylysine in receptor-targeted gene transfer complexes affects duration of reporter gene expression both in vitro and in vivo. J. Biol. Chem. 274, 4908-4916.