Vectors and delivery systems in gene therapy

Medical Science Monitor

Volumn 11, Issue 4, 2005, Pages

  Gardlík, Roman ^a,b   Pálffy, Roland ^a,b   Hodosy, Július ^a,b,c   Lukács, Ján ^b   Turňa, Ján ^b   Celec, Peter ^a,b,c,d  

^a BiomeD Research and Publishing Group   (Slovakia)

^b Physics and Informatics   (Slovakia)

^c Faculty of Medicine of Comenius University and University Hospital Bratislava   (Slovakia)

^d NONE   (Slovakia)

Author keywords

Alternative gene therapy; Gene therapy; Gene transfer; Oligodeoxynucleotides; Vectors

Indexed keywords

1,4 BIS[[2 (DIMETHYLAMINO N OXIDE)ETHYL]AMINO] 5,8 DIHYDROXYANTHRAQUINONE; FLUCYTOSINE; FLUOROURACIL; LIPOPLEX; NAKED DNA; OLIGODEOXYNUCLEOTIDE; TIRAPAZAMINE; VIRUS VECTOR;

ADENOVIRUS; DNA TRANSFER; GENE DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; HUMAN; MOLECULAR BIOLOGY; RETROVIRUS; REVIEW;

ANIMALS; ELECTROPORATION; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; OLIGODEOXYRIBONUCLEOTIDES; VIRUSES;

EID: 17444363398     PISSN: 12341010     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (101)

1. Orkin SH: Molecular genetics and potential gene therapy. Clin Immunol Immunopathol, 1986; 40: 151-6
2. Rubanyi GM: The future of human gene therapy. Mol Aspects Med, 2001; 22: 113-12
3. Hauser H, Spitzer D, Verhoeyen E et al: New approaches towards ex vivo and in vivo gene therapy. Cells Tissues Organs, 2000; 167: 75-80
4. Chan L, Fujimiya M, Kojima H: In vivo gene therapy for diabetes mellitus. Trends Mol Med, 2003; 9: 430-35
5. Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al: LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science, 2003; 302: 415-19
6. Walther W, Stein U: Viral vectors for gene transfer: a review of their use in the treatment of human diseases. Drugs, 2000; 60: 249-71
7. Kootstra NA, Verma IM: Gene therapy with viral vectors. Annu Rev Pharmacol Toxicol, 2003; 43: 413-39
8. Dando JS, Roncarolo MG, Bordignon C et al: A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors. Hum Gene Ther, 2001; 12: 1979-88
9. Von Seggern DJ, Kehler J, Endo RI et al: Complementation of a fibre mutant adenovirus by packaging cell lines stably expressing the adenovirus type 5 fibre protein. J Gen Virol, 1998; 79 (Pt 6): 1461-68
10. Lundstrom K: Latest development in viral vectors for gene therapy. Trends Biotechnol, 2003; 21: 117-22
11. Silman NJ, Fooks AR: Biophysical targeting of adenovirus vectors for gene therapy. Curr Opin Mol Ther, 2000; 2: 524-31
12. Einfeld DA, Roelvink PW: Advances towards targetable adenovirus vectors for gene therapy. Curr Opin Mol Ther, 2002; 4: 444-51
13. Gao GP, Yang Y, Wilson JM: Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J Virol, 1996; 70: 8934-43
14. Park MT, Lee MS, Kim SH et al: Influence of culture passages on growth kinetics and adenovirus vector production for gene therapy in monolayer and suspension cultures of HEK 293 cells. Appl Microbiol Biotechnol, 2004
15. Frauli M, Ribault S, Neuville P et al: Adenoviral-mediated skeletal muscle transcriptional targeting using chimeric tissue-specific promoters. Med Sci Monit, 2003; 9(2): BR78-84
16. Yang Y, Haecker SE, Su O et al: Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum Mol Genet, 1996; 5: 1703-12
17. Ritter T, Lehmann M, Volk HD: Improvements in gene therapy: averting the immune response to adenoviral vectors. BioDrugs, 2002; 16: 3-10
18. DeMatteo RP, Chu G, Ahn M et al: Immunologic barriers to hepatic adenoviral gene therapy for transplantation. Transplantation, 1997; 63: 315-19
19. Christ M, Lusky M, Stoeckel F et al: Gene therapy with recombinant adenovirus vectors: evaluation of the host immune response. Immunol Lett, 1997; 57: 19-25
20. Chia MC, Shi W, Li JH et al: A conditionally replicating adenovirus for nasopharyngeal carcinoma gene therapy. Mol Ther, 2004; 9: 804-17
21. Odaka M, Sterman DH, Wiewrodt R et al: Eradication of intraperitoneal and distant tumor by adenovirus-mediated interferon-beta gene therapy is attributable to induction of systemic immunity. Cancer Res, 2001; 61: 6201-12
22. Boris-Lawrie K, Temin HM: The retroviral vector. Replication cycle and safety considerations for retrovirus-mediated gene therapy. Ann N Y Acad Sci, 1994; 716: 59-70; discussion 71
23. Nafe C, Cao YJ, Quinones A et al: Expression of mutant non-cleavable Fas ligand on retrovirus packaging cells causes apoptosis of immunocompetent cells and improves prodnig activation gene therapy in a malignant glioma model. Life Sci, 2003; 73: 1847-60
24. Pannell D, Ellis J: Silencing of gene expression: implications for design of retrovirus vectors. Rev Med Virol, 2001; 11: 205-17
25. Scott-Taylor TH, Gallardo HF, Gansbacher B et al: Adenovirus facilitated infection of human cells with ecotropic retrovirus. Gene Ther, 1998; 5: 621-29
26. Hacein-Bey-Abina S, Le Deist F, Carlier F et al: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med, 2002; 346: 1185-93
27. Rainov NG, Ren H: Clinical trials with retrovirus mediated gene therapy-what have we learned? J Neurooncol, 2003; 65: 227-36
28. Quinonez R, Sutton RE: Lentiviral vectors for gene delivery into cells. DNA Cell Biol, 2002; 21: 937-51
29. Kafri T, Blomer U, Peterson DA et al: Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet, 1997; 17: 314-17
30. Tenenbaum L, Lehtonen E, Monahan PE: Evaluation of risks related to the use of adeno-associated virus-based vectors. Curr Gene Ther, 2003; 3: 545-65
31. Miller DG, Rutledge EA, Russell DW: Chromosomal effects of adeno-associated virus vector integration. Nat Genet, 2002; 30: 147-48
32. Satoh W, Hirai Y, Tamayose K et al: Site-specific integration of an adeno-associated virus vector plasmid mediated by regulated expression of rep based on Cre-loxP recombination. J Virol, 2000; 74: 10631-38
33. Conway JE, Rhys CM, Zolotukhin I et al: High-titer recombinant adeno-associated virus production utilizing a reconibinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap. Gene Ther, 1999; 6: 986-93
34. Wright JF, Qu G, Tang C et al: Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector. Curr Opin Drug Discov Devel, 2003; 6: 174-78
35. Wang XS, Yodel MC, Zhou SZ et al: Parvovirus B19 promoter at map unit 6 confers autonomous replication competence and erythroid specificity to adeno-associated virus 2 in primary human hematopoietic progenitor cells. Proc Natl Acad Sci USA, 1995; 92: 12416-20
36. Hargrove PW, Vanin EF, Kurtzman GJ et al: High-level globin gene expression mediated by a reconibinant adeno-associated virus genome that contains the 3′ gamma globin gene regulatory element and integrates as tandem copies in erythroid cells. Blood, 1997; 89: 2167-75
37. Ohi S, Kim BC: Synthesis of human globin polypeptides mediated by recombinant adeno-associated virus vectors. J Pharm Sci, 1996; 85: 274-81
38. Flotte TR: Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther, 2001; 3: 497-502
39. Kimura B, Mohuczy D, Tang X et al: Attenuation of hypertension and heart hypertrophy by adeno-associated virus delivering angiotensinogen antisense. Hypertension, 2001; 37: 376-80
40. Latchman DS: Gene delivery and gene therapy with herpes simplex virus-based vectors. Gene, 2001; 264: 1-9
41. Cotter MA, Robertson ES: Molecular genetic analysis of herpesviruses and their potential use as vectors for gene therapy applications. Curr Opin Mol Ther, 1999; 1: 633-44
42. Yeung SN, Tufaro F: Replicating herpes simplex virus vectors for cancer gene therapy. Expert Opin Pharmacother, 2000; 1: 623-31
43. Samoto K, Perng GC, Ehtesham M et al: A herpes simplex virus type 1 mutant deleted for gamma34.5 and LAT kills glioma cells in vitro and is inhibited for in vivo reactivation. Cancer Gene Ther, 2001; 8: 269-77
44. Carpenter DE, Stevens JG: Long-term expression of a foreign gene from a unique position in the latent herpes simplex virus genome. Hum Gene Ther, 1996; 7: 1447-54
45. Hu Y, Lee J, McCart JA et al: Yaba-like disease virus: an alternative replicating poxvirus vector for cancer gene therapy. J Virol, 2001; 75: 10300-8
46. Yamanaka R: Alphavirus vectors for cancer gene therapy (review). Int J Oncol, 2004; 24: 919-23
47. Lundstrom K: Alphavirus vectors for gene therapy applications. Curr Gene Ther, 2001; 1: 19-29
48. Lundstrom K: Alphavirus vectors as tools in cancer gene therapy. Technol Cancer Res Treat, 2002; 1: 83-88
49. Lundstrom K: Alphavirus vectors for vaccine production and gene therapy. Expert Rev Vaccines, 2003; 2: 447-59
50. Daemen T, Pries F, Bungener L et al: Genetic immunization against cervical carcinoma: induction of cytotoxic T lymphocyte activity with a recombinant alphavirus vector expressing human papillomavirus type 16 E6 and E7. Gene Ther, 2000; 7: 1859-66
51. Lundstrom K, Boulikas T: Viral and non-viral vectors in gene therapy: technology development and clinical trials. Technol Cancer Res Treat, 2003; 2: 471-86
52. Schatzlein AG: Non-viral vectors in cancer gene therapy: principles and progress. Anticancer Drugs, 2001; 12: 275-304
53. Nishitani M, Sakai T, Kanayama H et al: Cytokine gene therapy for cancer with naked DNA. Mol Urol, 2000; 4: 47-50
54. Molling K: Vaccination and gene therapy with naked DNA. Z Arztl Fortbild Qualitatssich, 1998; 92: 681-83
55. Lee Y, Park EJ, Yu SS et al: Improved expression of vascular endothelial growth factor by naked DNA in mouse skeletal muscles: implication for gene therapy of ischemic diseases. Biochem Biophys Res Commun, 2000; 272: 230-35
56. Perletti G, Marras E, Dondi D et al: Assessment of the biological activity of an improved naked-DNA vector for angiogenesis gene therapy on a novel non-mammalian model. Int J Mol Med, 2003; 11: 691-96
57. Baque P, Pierrefite-Carle V, Gavelli A et al: Naked DNA injection for liver metastases treatment in rats. Hepatology, 2002; 35: 1144-52
58. Ogris M: Non-viral cancer gene therapy - what is best? Drug Discov Today, 2003; 8: 63
59. Tupin E, Poirier B, Bureau MF et al: Non-viral gene transfer of murine spleen cells achieved by in vivo electroporation. Gene Ther, 2003; 10: 569-79
60. Gehl J: Electroporation: theory and methods, perspectives for drug delivery, gene therapy and research. Acta Physiol Scand, 2003; 177: 437-47
61. Kuriyama S, Mitoro A, Tsujinoue H et al: Particle-mediated gene transfer into murine livers using a newly developed gene gun. Gene Ther, 2000; 7: 1132-36
62. Qiu P, Ziegelhoffer P, Sun J et al: Gene gun delivery of mRNA in situ results in efficient transgene expression and genetic immunization. Gene Ther, 1996; 3: 262-68
63. Surman DR, Irvine KR, Shulman EP et al: Generation of polyclonal rabbit antisera to mouse melanoma associated antigens using gene gun immunization. J Immunol Methods, 1998; 214: 51-62
64. Darquet AM, Rangara R, Kreiss P et al: Minicircle: an improved DNA molecule for in vitro and in vivo gene transfer. Gene Ther, 1999; 6: 209-18
65. Stein CA, Cohen JS: Oligodeoxynucleotides as inhibitors of gene expression: a review. Cancer Res, 1988; 48: 2659-68
66. Miyake H, Hara I, Kamidono S et al: Novel therapeutic strategy for advanced prostate cancer using antisense oligodeoxynucleotides targeting anti-apoptotic genes upregulated after androgen withdrawal to delay androgen-independent progression and enhance chemosensitivity. Int J Urol, 2001; 8: 337-49
67. Robaczewska M, Guerret S, Remy JS et al: Inhibition of hepadnaviral replication by polyethylenimine-based intravenous delivery of antisense phosphodiester oligodeoxynucleotides to the liver. Gene Ther, 2001; 8: 874-81
68. Morishita R, Aoki M, Kaneda Y: Decoy oligodeoxynucleotides as novel cardiovascular drugs for cardiovascular disease. Ann N Y Acad Sci, 2001; 947: 294-301; discussion 301-2
69. Downward J: RNA interference. BMJ, 2004; 328: 1245-48
70. Montgomery MK: RNA interference: historical overview and significance. Methods Mol Biol, 2004; 265: 3-21
71. Wall NR, Shi Y: Small RNA: can RNA interference be exploited for therapy? Lancet, 2003; 362: 1401-3
72. Lai LW, Lien YH: Chimeric RNA/DNA oligonucleotide-based gene therapy. Kidney Int, 2002; 61: 47-51
73. Dass CR: Biochemical and biophysical characteristics of lipoplexes pertinent to solid tumour gene therapy. Int J Pharm, 2002; 241: 1-25
74. Gaucheron J, Santaella C, Vierling P: Improved in vitro gene transfer mediated by fluorinated lipoplexes in the presence of a bile salt surfactant. J Gene Med, 2001; 3: 338-44
75. Zhdanov RI, Podobed OV, Vlassov VV: Cationic lipid-DNA complexes-lipoplexes-for gene transfer and therapy. Bioelectrochemistry, 2002; 58: 53-64
76. Dass CR, Burton MA: Lipoplexes and tumours. A review. J Pharm Pharmacol, 1999; 51: 755-70
77. Omidi Y, Hollins AJ, Benboubetra M et al: Toxicogenomics of non-viral vectors for gene therapy: a microarray study of lipofectin- and oligo-fectamine-induced gene expression changes in human epithelial cells. J Drug Target, 2003; 11: 311-23
78. Lee LK, Siapati EK, Jenkins RG et al: Biophysical characterization of an integrin-targeted non-viral vector. Med Sci Monit, 2003; 9(1): BR54-61
79. Forrest ML, Pack DW: On the kinetics of polyplex endocytic trafficking: implications for gene delivery vector design. Mol Ther, 2002; 6: 57-66
80. Merlin JL, N'Doye A, Bouriez T et al: Polyethylenimine Derivatives as Potent Nonviral Vectors for Gene Transfer. Drug News Perspect, 2002; 15: 445-51
81. Rudolph C, Muller RH, Rosenecker J: Jet nebulization of PEI/DNA polyplexes: physical stability and in vitro gene delivery efficiency. J Gene Med, 2002; 4: 66-74
82. Mansouri S, Lavigne P, Corsi K et al: Chitosan-DNA nanoparticles as non-viral vectors in gene therapy: strategies to improve transfection efficacy. Eur J Pharm Biopharm, 2004; 57: 1-8
83. Schmidt-Wolf GD, Schmidt-Wolf IG: Non-viral and hybrid vectors in human gene therapy: an update. Trends Mol Med, 2003; 9: 67-72
84. Hodgson CP, Solaiman F: Virosomes: cationic liposomes enhance retroviral transduction. Nat Biotechnol, 1996; 14: 339-42
85. Lam PY, Breakefield XO: Hybrid vector designs to control the delivery, fate and expression of transgenes. J Gene Med, 2000; 2: 395-408
86. Tan BT, Wu L, Berk AJ: An adenovirus-Epstein-Barr virus hybrid vector that stably transforms cultured cells with high efficiency. J Virol, 1999; 73: 7582-89
87. Tu G, Kirchmaier AL, Liggitt D et al: Non-replicating Epstein-Barr virus-based plasmids extend gene expression and can improve gene therapy in vivo. J Biol Chem, 2000; 275: 30408-16
88. Bartoli A, Fettucciari K, Fetriconi I et al: Effect of trichostatin a and 5′-azacytidine on transgene reactivation in U937 transduced cells. Pharmacol Res, 2003; 48: 111-18
89. Brisson M, He Y, Li S et al: A novel T7 RNA polymerase autogene for efficient cytoplasmic expression of target genes. Gene Ther, 1999; 6: 263-70
90. Cooke H: Mammalian artificial chromosomes as vectors: progress and prospects. Cloning Stem Cells, 2001; 3: 243-49
91. Guo H, Kohlhaw GB: Regulation of transcription in mammalian cells by yeast Leu3p and externally supplied inducer. FEBS Lett, 1996; 390: 191-95
92. Wang J, Voutetakis A, Zheng C et al: Rapamycin control of exocrine protein levels in saliva after adenoviral vector-mediated gene transfer. Gene Ther, 2004; 11: 729-33
93. Nuyts S, Van Mellaert L, Theys J et al: The use of radiation-induced bacterial promoters in anaerobic conditions: a means to control gene expression in clostridium-mediated therapy for cancer. Radiat Res, 2001; 155: 716-23
94. Liu SC, Minton NP, Giaccia AJ et al: Anticancer efficacy of systemically delivered anaerobic bacteria as gene therapy vectors targeting tumor hypoxia/necrosis. Gene Ther, 2002; 9: 291-96
95. Jounaidi Y, Waxman DJ: Combination of the bioreductive drug tirapazamine with the chemotherapeutic prodrug cyclophosphamide for P450/P450-reductase-based cancer gene therapy. Cancer Res, 2000; 60: 3761-69
96. Nemunaitis J, Cunningham C, Senzer N et al: Pilot trial of genetically modified, attenuated Salmonella expressing the E. coli cytosine deaminase gene in refractory cancer patients. Cancer Gene Ther, 2003; 10: 737-44
97. Theys J, Landuyt AW, Nuyts S et al: Clostridium as a tumor-specific delivery system of therapeutic proteins. Cancer Detect Prev, 2001; 25: 548-57
98. Fujimori M, Amano J, Taniguchi S: The genus Bifidobacterium for cancer gene therapy. Curr Opin Drug Discov Devel, 2002; 5: 200-3
99. Li YH, Xie YM, Guo KY et al: Treatment of Tumor in Mice by Oral Administration of Cytosine Deaminase Gene Carried in Live Attenuated Salmonella. Sheng Wu Hua Xue Yu Sheng Wu Wu Li Xue Bao (Shanghai), 2001; 33: 233-37
100. Celec P, Gardlík R, Pálffy R et al: The use of transformed Escherichia coli for experimental angiogenesis induced by regulated in situ production of vascular endothelial growth factor - an alternative gene therapy. Med Hypotheses, 2005; 64: 505-11
101. Nabel GJ: Genetic, cellular and immune approaches to disease therapy: past and future. Nat Med, 2004; 10: 135-41