Non-Viral Vectors for Gene Therapy

Biotechnology

Volumn 5, Issue , 2008, Pages 427-441

  Weir, Neil ^a  

^a UCB PHARMA   (Belgium)

Author keywords

Concentration; Demonstration; Efficiency; Linearization; Optimized

Indexed keywords

EID: 0002574512     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9783527620869.ch19     Document Type: Chapter

References (85)

1. ALTON, E. W., MIDDLETON, P. G., CAPLAN, N. J., SMITH, S. N., STEEL, D. M. et al. (1993), Noninvasive liposome-mediated gene delivery can correct the ion trasport defect in cystic fibrosis mutant mice, Nature Genetics 5, 135-142.
2. ANDERSON, W. F. (1992), Human gene therapy, Science 256, 808-813.
3. BAATZ, J. E., BRUNO, M. D., CIRAOLO, P. J., GLASSER, S. W., STRIPP, B. R. et al. (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.
4. BRIGHAM, K. L., MEYRICK, B., CHRISTMAN, B., MAGNUSON, M., KING, G., 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.
5. BUSCHLE, M., COTTEN, M., KIRLAPPOS, H., MECHTLER, K., SCHAFFNER, G. et al. (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.
6. CALOS, M. P. (1996), The potential of extrachromosomal replicating vectors for gene therapy, Trends Genet. 12, 463-466.
7. CANONICO, A. E., PLITMAN, J. D., CONARY, J. T., MEYRICK, B. O., BRIGHAM, K. L. (1994), No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes, J. Appl. Physiol. 77, 415-419.
8. CAPLAN, N. J., ALTON, E. W., MIDDLETON, P. G., DORIN, J. R., STEVENSON, B. J. et al. (1995), Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis, Nature Medicine 1, 39-46.
9. CAPPECCHI, M. R. (1980), High efficiency transformation by direct microinjection of DNA into cultured mammalian cells, Cell 22, 479-488.
10. CHEN, C, OKAYAMA, H. (1987), High-efficiency transformation of mammalian cells by plasmid DNA, Mol. Cell. Biol. 7, 2745-2752.
11. CHEN, J., GAMOU, S., TAKAYANAGI, A., SHIMIZU, N. (1994), A novel gene delivery system using EGF receptor-mediated endocytosis, FEBS Lett. 338, 167-169.
12. CHEN, S. Y., ZANI, C, KHOURI, Y., MARASCO, W. A. (1995), Design of a genetic immunotoxin to eliminate toxin immunogenicity, Gene Ther. 2, 116-123.
13. COTTEN, M., LANGLE-ROUAULT, F., KIRLAPPOS, H., WAGNER, E., MECHTLER, K. et al. (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.
14. COTTEN, M., WAGNER, E., ZATLOUKAL, K., PHILLIPS, S., CURIEL, D. T., 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.
15. CRAIG, R. K., ANTONIOU, M., DJEHA, H. (1995), International Patent Application WO 9506129.
16. CRISTIANO, R. J., ROTH, J. A. (1996), Epidermal growth factor mediated DNA delivery into lung cancer cells via the epidermal growth factor receptor, Cancer Gene Ther. 3, 4-10.
17. CURIEL, D. T., AGARWAL, S., WAGNER, E., COTTEN, M. (1991), Adenovirus enhancement of transferrin-polylysine-mediated gene delivery, Proc. Natl. Acad. Sci. USA 88, 8850-8854.
18. DILLON, N., GROSVELD, F. (1993), Transcriptional regulation of multigene loci: multilevel control, Trends Genet. 9, 134-137.
19. DING, Z. M, CRISTIANO, R. J., ROTH, J. A., TAKACS, B., Kuo, M. T. (1995), Malarial circum-sporozoite protein is a novel gene delivery vehicle to primary hepatocyte cultures and cultured cells, J Biol. Chem. 270, 3667-3676.
20. DUZGUNES, N., GOLDSTEIN, J. A., FRIEND, D. S., FELGNER, P. L. (1989), Fusion of liposomes containing a novel cationic lipid, N-[2,3-(dioleyl-oxy)propyl]-N,N,N trimethylammonium: induction by multivalent anions and asymmetric fusion with acidic phospholipid vesicles, Biochemistry 28, 9179-9184.
21. FARHOOD, H., SERBINA, N., HUANG, L. (1995), The role of dioleoyl phosphatidyl ethanolamine in cationic liposome-mediated gene transfer, Biochim. Biophys. Acta 1235, 289-295.
22. FEATHERSTONE, T., HUXLEY, C. (1993), Extrachromosomal maintenance and amplification of yeast artificial chromosome DNA in mouse cells, Genomics 17, 267-278.
23. FELGNER, P. L., GADEK, T. R., HOLM, M., ROMAN, R., CHAN, H. W. et al. (1987), Lipofection: a highly efficient, lipid-mediated DNA-transfec-tion procedure, Proc. Natl. Acad. Sci. USA 84, 7413-7417.
24. FELGNER, J. H., KUMAR, R., SRIDHAR, C. N., WHEELER, C. J., TSAI, Y. J. et al. (1994), Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations, J. Biol. Chem. 269, 2550-2561.
25. FERKOL, T., KAETZEL, C. S., DAVIS, P. B. (1993), Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor, J. Clin. Invest. 92, 2394-2400.
26. FERKOL, T., PERALES, J. C, ECKMAN, E., KAETZEL, C. S., HANSON, R. W., DAVIS, P. B. (1995), Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor, J. Clin. Invest. 95, 493-502.
27. FERKOL, T., PELLICENA-PALLE, A., ECKMAN, E., PERALES, J. C, TRZASKA, T. et al. (1996), Immunologic responses to gene transfer into mice via the polymeric immunoglobulin receptor, Gene Ther. 3, 669-678.
28. FRALEY, R., SUBRAMANI, S., BERG, P., PAPAHAD-JOPOULOS, D. (1980), Introduction of liposome-encapsulated SV40 DNA into cells, J. Biol. Chem. 255, 10431-10435.
29. GAO, X., HUANG, L. (1991), A novel cationic liposome reagent for efficient transfection of mammalian cells, Biochem. Biophys. Res. Commun. 179, 280-285.
30. GAO, X., HUANG, L. (1996), Potentiation of cationic liposome-mediated gene delivery by polycations, Biochemistry 35, 1027-1036.
31. GERSHON, H., GHIRLANDO, R., GUTTMAN, S. B., MINSKY, A. (1993), Mode of formation and structural features of DNA-cationic liposome complexes used for transfection, Biochemistry 32, 7143-7151.
32. GOTTSHALK, S., CRISTIANO, R. J., SMITH, L. C, Woo, S, L. C. (1994), Folate receptor-mediated DNA delivery into tumour cells: potosomal disruption results in enhanced gene expression, Gene Ther. 1, 185-191.
33. GOTTSHALK, S., SPARROW, J. T., HAUER, J., MIMMS, M. P., LELAND, F. E. et al. (1996), A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells, Gene Ther. 3, 448-457.
34. GRAHAM, F. L., VAN DER EB, A. J. (1973), A new technique for the assay of infectivity of human adenovirus 5 DNA, Virology 52, 456-460.
35. HAENSLER, J., SZOKA, F. C. Jr. (1993), Polyami-doamine cascade polymers mediate efficient transfection of cells in culture, Bioconj. Chem. 4, 372-379.
36. HAWLEY-NELSON, P., CICCARONE, V., GEBEYE-HU, G., JESSE, J., FELGNER, P. L. (1993), Lipo-fectamine TM reagent: a new, higher efficiency polycationic liposome transfection reagent, Focus 15, 73-79.
37. HAYNES, J. R., MCCABE, D. E., SWAIN, W. F., WIDERA, G., FULLER, J. T. (1996), Particle-mediated nucleic acid immunization, J. Biotechnol. 44, 37-42.
38. HENGGE, U. R., CHAN, E. F., FOSTER, R. A., WALKER, P. S., VOGEL, J. C (1995), Cytokine gene expression in epidermis with biological effects following injection of naked DNA, Nature Genetics 10, 161-166.
39. HOFFMAN, G. A. (1996), International Patent Application, WO 9612520.
40. HOLMBERG, E. G., REUER, Q. R., GEISERT, E. E., OWENS, J. L. (1994), Delivery of plasmid DNA to glial cells using pH-sensitive immunolipo-somes, Biochem. Biophys. Res. Commun. 201, 888-893.
41. HUXLEY, C. (1994), Mammalian artificial chromosomes: a new tool for gene therapy, Gene Ther. 1, 7-12.
42. ISHIKAWA, Y., HOMCY, C. J. (1992), High efficiency gene transfer into mammalian cells by a double transfection protocol, Nucleic Acids Res. 20, 4367.
43. KANEDA, Y., IWAI, K., UCHIDA, T. (1989), Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science 243, 375-378.
44. KAO, G. Y., LUNG -JI, C, ALLEN, T. M. (1996), Use of targeted cationic liposomes in enhanced DNA delivery to cancer cells, Cancer Gene Ther. 3, 250-256.
45. KAWAI, S., NISHIZAWA, M. (1984), New procedure for DNA transfection with polycation and dimethyl sulfoxide, Mol. Cell. Biol. 4, 1172-1174.
46. KINOSITA, K. Jr., TSONG, T. T. (1977), Hemolysis of human erythrocytes by transient electric field, Proc. Natl. Acad. Sci. USA 74, 1923-1927.
47. LABAT-MOLEUR, F., STEFFAN, A.-M., BRISSON, C, PERRON, H., FEUGAS, O. et al. (1996), An electron microscopy study into the mechanism of gene transfer with lipopolyamines, Gene Ther. 3, 1010-1017.
48. LEE, R. J., HUANG, L. (1996), Folate-targeted, anionic liposome-entrapped polylysine condensed DNA for tumor cell-specific gene transfer, J. Biol. Chem. 271, 8481-8487.
49. LOYTER, A., SCANGOS, G. A., RUDDLE, F. H. (1982), Mechanisms of DNA uptake by mammalian cells: fate of exogenously added DNA monitored by the use of fluorescent dyes, Proc. Natl. Acad. Sci. USA 79, 422-426.
50. MANNING, G. S. (1978), The molecular theory of polyelectrolyte solutions with applications to the electrostatic properties of polynucleotides, Q. Rev. Biophys. 11,179-246.
51. MCLACHLAN, G., DAVIDSON, D. J., STEVENSON, B. J., DICKINSON, P., DAVIDSON-SMITH, H. et al. (1995), Evaluation in vivo and in vivo of cationic liposome expression construct complexes for cystic fibrosis gene therapy, Gene Ther. 2, 614-622.
52. MERWIN, J. R., CARMICHAEL, E. P., NOELL, G. S., DEROME, M. E., THOMAS, W. L. et al. (1995), CD5-mediated specific delivery of DNA to T lymphocytes: compartmentalization augmented by adenovirus, J. Immunol. Methods 186, 257-266.
53. MIDOUX, P., MENDES, C., LEGRAND, A., RAIMOND, J., MAYER, R. et al. (1993), Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells, Nucleic Acids Res. 21, 871-878.
54. NEUMANN, E., SCHAEFER-RIDDER, M., WANG, Y., HOFSCHNEIDER, P. H. (1982), Gene transfer into mouse myeloma cells by electroporation in high electric fields, EMBO J. 1, 841-845.
55. NICOLAU, C, CUDD, A. (1989), Liposomes as carriers of DNA, Crit. Rev. Ther. Drug Carrier Syst. 6, 239-271.
56. PERALES, J. C., FERKOL, T., MOLAS, M. HANSON, R. W. (1994), An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes, Eur. J. Biochem. 226, 255-266.
57. PHILIP, R., BRUNETTE, E., KILINSKI, L., MURUGESH, D., MCNALLY, M. A. et al. (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.
58. PLANK, C, ZATLOUKAL, K., COTTEN, M., MECHTLER, K., WAGNER, E. (1992), Gene transfer into hepatocytes using asialoglycoprotein receptor-mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand, Bioconj. Chem. 3, 533-539.
59. RICHARDS, C A., AUSTIN, E. A., HUBER, B. E. (1995), Transcriptional regulatory sequences of carcinoembryonic antigen: identification and use with cytosine deaminase for tumour-specific gene therapy; chimeric gene construction for use in tumour-specific suicide therapy, Hum. Gene Ther. 6, 881-893.
60. ROSENKRANZ, A. A., YACHMENEV, S. V., JANS, D. A., SEREBRYAKOVA, N. V., MURAV'EV, V. I. et al. (1992), Receptor-mediated endocytosis and nuclear transport of a transfecting DNA construct, Exp. Cell Res. 199, 323-329.
61. Ross, G. F., MORRIS, R. E., CIRAOLO, G., HUELSMAN, K., BRUNO, M. et al. (1995), Surfactant protein A-polylysine conjugates for delivery of DNA to airway cells in culture, Hum. Gene Ther. 6, 31-40.
62. SENIOR, J. H., TRIMBLE, K. R., MASKIEWICZ. R. (1991), Interaction of positively charged liposomes with blood: implications for their application in vivo, Biochim. Biophys. Acta 1070, 173-179.
63. SHIMIZU, N., CHEN, J., GAMOU, S., TAKAYANAGI, A. (1996), Immunogene approach toward cancer therapy using erythrocyte growth factor receptor-mediated gene delivery, Cancer Gene Ther. 3, 113-120.
64. SORIANO, P., DIJKSTRA, J., LEGRAND, A., SPANJER, H., LONDOS-GAGLIARDI, D. et al. (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.
65. STERNBERG, B., SORGI, F. L., HUANG, L. (1994), New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy, FEBS Lett. 356, 361-366.
66. THURNHER, M, WAGNER, E., CLAUSEN, H., MECHTLER, K., RUSCONI, S. et al. (1994), Carbohydrate receptor-mediated gene transfer to human T leukemic cells, Glycobiology 4, 429-435.
67. TRUBETSKOY, V. S., TORCHILIN, V. P., KENNEL,S. J., HUANG, L. (1992a), 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.
68. TRUBETSKOY, V. S., TORCHILIN, V. P., KENNEL, S., HUANG,L. (1992b), 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.
69. ULMER, J. B., DONNELLY, J. J., PARKER, S. E., RHODES, G. H., FELGNER, P. L. et al. (1993), Heterologous protection against influenza by injection of DNA encoding a viral protein, Science 259, 1745-1749.
70. VITIELLO, L., CHONN, A., WASSERMAN, J. D., DUFF, C, 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.
71. WAGNER, E., ZENKE, M., COTTEN, M., BEUG, H., BIRNSTIEL, M. L. (1990), Transferrin-polycation conjugates as carriers for DNA uptake into cells, Proc. Natl. Acad. Sci. USA 87, 3410-3414.
72. WAGNER, E., COTTEN, M., FOISNER, R., BIRNSTIEL, M. L. (1991), 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.
73. WAGNER, E., ZATLOUKAL, K., COTTEN, M., KIRLAPPOS, H., MECHTLER, K. (1992a), Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes, Proc. Natl. Acad. Sci. USA 89, 6099-6103.
74. WAGNER, E., PLANK, C, ZATLOUKAL, K., COTTEN, M., BIRNSTIEL, M. L. (1992b), 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.
75. WANG, C. Y., HUANG, L. (1987), pH-Sensitive im-munoliposomes mediate target-cell specific delivery and controlled expression of a foreign gene in mouse, Proc. Natl. Acad. Sci. USA 84, 7851-7855.
76. WOLFF, J. A., MALONE, R. W., WILLIAMS, P., CHONG, W., ACSADI, G. et al. (1990), Direct gene transfer into mouse muscle in vivo, Science 247, 1465-1468.
77. WOLFF, J. A., LUDTKE, J. J., ACSADI, G., WILLIAMS, P., JAM, A. (1992), Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle, Hum. Mol. Genet. 1, 363-369.
78. WU, G. Y., Wu, C. H. (1987), Receptor-mediated in vivo gene transformation by a soluble DNA carrier system, J. Biol. Chem. 262, 4429-4432.
79. WU, G. Y., Wu, C. H. (1988), Receptor-mediated gene delivery and expression in vivo, J. Biol. Chem. 263, 14621-14624.
80. WU, G. Y., Wu, C. H. (1992), Specific inhibition of hepatitis B viral gene expression in vivo by targeted antisense oligonucleotides, J. Biol. Chem. 267, 12436-12439.
81. YANG, N. S., BURKHOLDER, J., ROBERTS, B., MARTINELL, B., MCCABE, D. (1990), In vivo and in vivo gene transfer to mammalian somatic cells by particle bombardment, Proc. Natl. Acad. Sci. USA 87, 9568-9572.
82. YOSHIMURA, K., ROSENFELD, M. A., NAKAMURA, H., SCHERER, E. M., PAVIRANI, A. et al. (1992), Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer, Nucleic Acids Res. 20, 3233-3240.
83. ZHANG, L., LI, L., HOFFMANN, G. A., HOFFMAN, R. M. (1996), Depth-targeted efficient gene delivery and expression in the skin by pulsed electric fields: an approach to gene therapy of skin aging and other diseases, Biochem. Biophys. Res. Commun. 220, 633-636.
84. ZHOU, X., HUANG, L. (1994), DNA transfection mediated by cationic liposomes containing lipo-polylysine: characterization and mechanism of action, Biochim. Biophys. Acta 1189, 195-203.
85. ZHOU, X. H, KLIBANOV, A. L., HUANG, L. (1991), Lipophilic polylysines mediate efficient DNA transfection in mammalian cells, Biochim. Biophys. Acta 1065, 8-14.