Viral vectors for gene therapy

Pharmacology and Therapeutics

Volumn 80, Issue 1, 1998, Pages 35-47

  Robbins, Paul D ^a   Ghivizzani, Steven C ^a  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Adenovirus; Gene therapy; Herpes simplex virus; Retrovirus; Vector

Indexed keywords

DNA; MIFEPRISTONE; NUCLEIC ACID; RAPAMYCIN;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ARTHRITIS; CANCER; CELL DIVISION; CLINICAL TRIAL; CONJUGATE; CYSTIC FIBROSIS; CYTOTOXICITY; DUCHENNE MUSCULAR DYSTROPHY; EXPRESSION VECTOR; GAUCHER DISEASE; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HERPES SIMPLEX VIRUS; HUMAN; IMMUNE RESPONSE; IMMUNOSURVEILLANCE; LYSOSOME STORAGE DISEASE; PRIORITY JOURNAL; RETROVIRUS; REVIEW; TRANSGENE; VIRUS DNA CELL DNA INTERACTION; VIRUS REPLICATION;

CYSTIC FIBROSIS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; NEOPLASMS; VIRUSES;

EID: 0031770243     PISSN: 01637258     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0163-7258(98)00020-5     Document Type: Review

References (75)

1. Albertini, M. R., Emler, C. A., Schell, K., Tans, K. J., King, D. M. and Sheehy, M. J. (1996) Dual expression of human leukocyte antigen molecules and the B7-1 costimulatory molecule (CD80) on human melanoma cells after particle-mediated gene transfer. Cancer Gene Ther. 3: 192-201.
2. Bahnson, A. B., Nimgaonkar, M., Fei, Y., Boggs, S. S., Robbins, P. D., Ohashi, T., Dunigan, J., Li, J., Ball, E. D. and Barranger, J. A. (1994) Transduction of CD34+ enriched cord blood and Gaucher bone marrow cells by a retroviral vector carrying the glucocerebrosidase gene. Gene Ther. 1: 176-184.
3. Bischoff, J. R., Kirn, D. H., Williams, A., Heise, C., Horn, S., Muna, M., Ng, L., Nye, A. J., Sampson-Johannes, A., Fattaey, A. and McCormick, F. (1996) An adenovirus mutant that replicates selectively in p53-deficient human tumor cells. Science 274: 373-376.
4. Blomer, U., Naldini, L., Verma, I. M., Trono, D. and Gage, F. H. (1996) Applications of gene therapy to the CNS [special issue]. Hum. Mol. Genet. 5: 1397-1404.
5. Carter, B. J. and Flotte, T. R. (1996) Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. Curr. Top. Microbiol. Immunol. 218: 119-144.
6. Conrad, C. K., Allen, S. S., Afione, S. A., Reynolds, T. C., Beck, S. E., Fee-Maki, M., Barrazza-Ortiz, X., Adams, R., Askin, F. B., Carter, B. J., Guggino, W. B. and Flotte, T. R. (1996) Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther. 3: 658-668.
7. Crystal, R. G. (1995) Transfer of genes to humans: early lessons and obstacles to success. Science 270: 404-410.
8. Douglas, J. T. and Curiel, D. T. (1997) Strategies to accomplish targeted gene delivery to muscle cells employing tropism-modified adenoviral vectors. Neuromuscul. Disord. 7: 284-298.
9. Dranoff, G., Jaffee, E., Lazenby, A., Golumbek, P., Levitsky, H., Brose, K., Jackson, V., Hamada, H., Pardoll, D. and Mulligan, R. C. (1993) Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting antitumor immunity. Proc. Natl. Acad. Sci. USA 90: 3539-3543.
10. Dranoff, G., Soiffer, R., Lynch, T., Mihm, M., Jung, K., Kolesar, K., Liebster, L., Lam, P., Duda, R., Mentzer, S., Singer, S., Tanabe, K., Johnson, R., Sober, A., Bhan, A., Clift, S., Cohen, L., Parry, G., Rokovich, J., Richards, L., Drayer, J., Berns, A. and Mulligan, R. C. (1997) A phase I study of vaccination with autologous, irradiated melanoma cells engineered to secrete human granulocyte-macrophage colony stimulating factor. Hum. Gene Ther. 8: 111-123.
11. Engelhardt, J. F., Yang, Y., Stratford-Perricaudet, L. D., Allen, E. D., Kozarsky, K., Perricaudet, M., Yankaskas, J. R. and Wilson, J. M. (1993) Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with El-deleted adenoviruses. Nat. Genet. 4: 27-34.
12. Evans, C. H., Robbins, P. D., Ghivizzani, S. C., Herndon, J. H., Kang, R., Bahnson, A. B., Barranger, J. A., Elders, E. M., Gay, S., Tomaino, M. M., Wasko, C., Watkins, S. C., Whiteside, T. L., Glorioso, J. C., Lotze, M. T. and Wright, T. M. (1996) Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis. Hum. Gene Ther. 7: 1261-1280.
13. Feero, W. G., Li, S., Rosenblatt, J. D., Sirianni, N., Morgan, J. E., Partridge, T. A., Huang, L. and Hoffman, E. P. (1997) Selection and use of ligands for receptor-mediated gene delivery to mygenic cells. Gene Ther. 4: 664-667.
14. Ferrari, F. K., Samulski, T., Shenk, T. and Samulski, R. J. (1996) Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70: 3227-3234.
15. Fink, D. J. and Glorioso, J. C. (1997a) Engineering herpes simplex virus vectors for gene transfer to neurons. Nat. Med. 3: 357-359.
16. Fink, D. J. and Glorioso, J. C. (1997b) Herpes simplex virus-based vectors: problems and some solutions. Adv. Neurol. 72: 149-156.
17. Fink, D. J., Ramakrishnan, R., Marconi, P., Goins, W. F., Holland, T. C. and Glorioso, J. C. (1995) Advances in the development of herpes simplex virus-based gene transfer vectors for the nervous system. Clin. Neurosci. 3: 284-291.
18. Fisher, K. J., Choi, H., Burda, J., Chen, S. J. and Wilson, J. M. (1996) Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 217: 11-22.
19. Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Haecker, S. E., High, K., Pathak, R., Raper, S. E. and Wilson, J. M. (1997) Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med. 3: 306-312.
20. Flotte, T., Carter, B., Conrad, C., Guggino, W., Reynolds, T., Rosenstein, B., Taylor, G., Walden, S. and Wetzel, R. (1996) A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 7: 1145-1159.
21. Gao, X. and Huang, L. (1995) Cationic liposome-mediated gene transfer. Gene Ther. 2: 710-722.
22. Geller, A. I. (1997) Herpes simplex virus-1 plasmid vectors for gene transfer into neurons. Adv. Neurol. 72: 143-148.
23. Gossen, M. and Bujard, H. (1992) Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc. Natl. Acad. Sci. USA 89: 5547-5551.
24. Graham, F. L. and Prevec, L. (1995) Methods for construction of adenovirus vectors. Mol. Biotechnol. 3: 207-220.
25. Guild, B. C., Finer, M. H., Housman, D. E. and Mulligan, R. C. (1988) Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo. J. Virol. 62: 3795-3801.
26. Gussoni, E., Blau, H. M. and Kunkel, L. M. (1997) The fate of individual myoblasts after transplantation into muscles of DMD patients. Nat. Med. 3: 970-977.
27. Hauser, M. A., Amalfitano, A., Kumarsingh, R., Hauschka, S. D. and Chamberlain, J. S. (1997) Improved adenoviral vectors for gene therapy of Duchene muscular dystrophy. Neuromuscul. Disord. 7: 277-283.
28. Hui, K. M. and Chia, T. F. (1997) Eradication of tumor growth via biolistic transformation with allogenic MHC genes. Gene Ther. 4: 762-767.
29. Kakulas, B. A. (1997) Problems and potential for gene therapy in Duchene muscular dystrophy. Neuromuscul. Disord. 7: 319-324.
30. Kaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O'Malley, K. L. and During, M. J. (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8: 148-154.
31. Kirn, D. H. and McCormick, F. (1996) Replicating viruses as selective cancer therapeutics. Mol. Med. Today 2: 519-527.
32. Kochanek, S., Clemens, P. R., Mitani, K., Chen, H. H., Chan, S. and Caskey, C. T. (1996) A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc. Natl. Acad. Sci. USA 93: 5731-5736.
33. Koeberl, D. D., Alexander, I. E., Halbert, C. L., Russell, D. W. and Miller, A. D. (1997) Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 94: 1426-1431.
34. Krasnykh, V. N., Mikheeva, G. V., Douglas, J. T. and Curiel, D. T. (1996) Generation of recombinant adenovirus vectors with modified fibers for altering viral tropism. J. Virol. 70: 6839-6846.
35. Krougliak, V. and Graham, F. L. (1995) Development of cell lines capable of complementing E1, E4, and protein IX defective adenovirus type 5 mutants. Hum. Gene Ther. 6: 1575-1586.
36. Liu, F., Yang, J., Huang, L. and Liu, D. (1996) New cationic lipid formulations for gene transfer. Pharm. Res. 13: 1856-1860.
37. Lotze, M. T., Zitvogel, L., Campbell, R., Robbins, P. D., Elder, E., Haluszczak, C., Martin, D., Whiteside, T. L., Storkus, W. J. and Tahara, H. (1996) Cytokine gene therapy of cancer using interleukin-12: murine and clinical trials. Ann. NY Acad. Sci. 795: 440-454.
38. Mack, C. A., Song, W. R., Carpenter, H., Wickham, T. J., Kovesdi, I., Harvey, G., Magovern, C. J., Isom, O. W., Rosengart, T., Falck-Pedersen, E., Hackett, N. R., Crystal, R. G. and Mastrangeli, A. (1997) Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype. Hum. Gene Ther. 8: 99-109.
39. Mader, S. and White, J. H. (1993) A steroid-indueible promoter for the controlled overexpression of clones genes in eukaryotic cells. Proc. Natl. Acad. Sci. USA 90: 5603-5607.
40. Marconi, P., Krisky, D., Oligino, T., Poliani, P. L., Ramakrishnan, R., Goins, W. F., Fink, D. J. and Glorioso, J. C. (1996) Replication-defective herpes simplex virus vectors for gene transfer in vivo. Proc. Natl. Acad. Sci. USA 93: 11319-11320.
41. Miller, A. D. (1992a) Human gene therapy comes of age. Nature 357: 455-460.
42. Miller, A. D. (1992b) Retroviral vectors. Curr. Top. Microbiol. Immunol. 158: 1-24.
43. Miller, A. D., Miller, D. G., Garcia, J. V. and Lynch, C. M. (1993) Use of retroviral vectors for gene transfer and expression. Methods Enzymol. 217: 581-599.
44. Miller, R. and Curiel, D. T. (1996) Towards the use of replicative adenoviral vectors for cancer gene therapy [editorial]. Gene Ther. 3: 557-559.
45. Mulligan, R. C. (1993) The basic science of gene therapy. Science 260: 926-932.
46. Naldini, L., Blomer, U., Gage, F. H., Trono, D. and Verma, I. M. (1996a) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 93: 11382-11388.
47. Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M. and Trono, D. (1996b) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267.
48. Neve, R. L. and Geller, A. I. (1995) A defective herpes simplex virus vector system for gene delivery into the brain: comparison with alternative gene delivery systems and usefulness for gene therapy. Clin. Neurosci. 3: 262-267.
49. Nguyen, D. M., Wiehle, S. A., Roth, J. A. and Cristiano, R. J. (1997) Gene delivery into malignant cells in vivo by a conjugated adenovirus DNA complex. Cancer Gene Ther. 4: 183-190.
50. Otani, K., Nita, I., Macaulay, W., Georgescu, H. I., Robbins, P. D. and Evans, C. H. (1996) Suppression of antigen-induced arthritis in rabbits by ex vivo gene therapy. J. Immunol. 156: 3558-3562.
51. Parks, R. J., Chen, L., Anton, M., Sankar, U., Rudnicki, M. A. and Graham, F. L. (1996) A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl. Acad. Sci. USA 93: 13565-13570.
52. Rakhmilevich, A. L., Janssen, K., Turner, J., Culp, J. and Yang, N. S. (1997) Cytokine gene therapy of cancer using gene gun technology, superior antitumor activity of interleukin-12. Hum. Gene Ther. 5: 1303-1311.
53. Rivera, V. M., Clackson, T., Natesan, S., Pollock, R., Amara, J. F., Keenan, T., Magari, S. R., Phillips, T., Courage, N. L., Cerasoli, F., Jr., Holt, D. A. and Gilman, M. (1996) A humanized system for pharmacologic control of gene expression. Nat. Med. 2: 1028-1032.
54. Riviere, I., Brose, K. and Mulligan, R. C. (1995) Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc. Natl. Acad. Sci. USA 92: 6733-6737.
55. Robbins, P. D., Tahara, H., Mueller, G., Hung, G., Bahnson, A., Zitvogel, L., Galea-Lauri, J., Ohashi, T., Patrene, K., Boggs, S. S., Evans, C. H. and Barranger, J. (1994) Retroviral vectors for use in human gene therapy for cancer, Gaucher disease, and arthritis. Ann. NY Acad. Sci. 716: 72-88. Discussion: 88-89.
56. Rolling, F. and Samulski, R. J. (1995) AAV as a viral vector for human gene therapy. Generation of recombinant virus. Mol. Biotechnol. 3: 9-15.
57. Rosenfeld, M. E. and Curiel, D. T. (1996) Gene therapy strategies for novel cancer therapeutics. Curr. Opin. Oncol. 8: 72-77.
58. Russell, D. W., Alexander, I. E. and Miller, A. D. (1995) DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 92: 5719-5723.
59. Samulski, R. J. (1993) Adeno-associated virus: integration at a specific chromosomal locus. Curr. Opin. Genet. Dev. 3: 74-80.
60. Shimizu, N., Chen, J., Gamou, S. and Takayanagi, A. (1996) Immunogenic approach toward cancer therapy using erythrocyte growth factor receptor-mediated gene delivery. Cancer Gene Ther. 3: 113-120.
61. Shockett, P., Defilippantonio, M., Hellman, N. and Schatz, D. G. (1995) A modified tetracycline-regulated system provides auto-regulatory, inducible gene expression in cultured cells and transgenic mice. Proc. Natl. Acad. Sci. USA 92: 6522-6526.
62. Starr, P. A., Lim, F., Grant, F. D., Trask, L., Lang, P., Yu, L. and Geller, A. I. (1996) Long-term persistence of defective HSV-1 vectors in the rat brain is demonstrated by reactivation of vector gene expression. Gene Ther. 3: 615-623.
63. Tahara, H., Zeh, H. J. R., Storkus, W. J., Pappo, I., Watkins, S. C., Gubler, U., Wolf, S. F., Robbins, P. D. and Lotze, M. T. (1994) Fibroblasts genetically engineered to secrete interleukin 12 can suppress tumor growth and induce antitumor immunity to a murine melanoma in vivo. Cancer Res. 54: 182-189.
64. Tahara, H., Zitvogel, L., Storkus, W. J., Zeh, H. J. R., McKinney, T. G., Schreiber, R. D., Gubler, U., Robbins, P. D. and Lotze, M. T. (1995) Effective eradication of established murine tumors with IL-12 gene therapy using a polycistronic retroviral vector. J. Immunol. 154: 6466-6474.
65. Varley, A. W., Geiszler, S. M., Gaynor, R. B. and Munford, R. S. (1997) A two-component expression system that responds to inflammatory stimuli in vivo. Nat. Biotechnol. 15: 1002-1006.
66. Wang, Q. and Finer, M. H. (1996) Second-generation adenovirus vectors. Nat. Med. 2: 714-716.
67. Wang, Y., Xu, J., Pierson, T., O'Malley, B. W. and Tsai, S. Y. (1997) Positive and negative regulation of gene expression in eukaryotic cells with an inducible transcriptional regulator. Gene Ther. 4: 432-441.
68. Wickham, T. J., Carrion, M. E. and Kovesdi, I. (1995) Targeting of adenovirus penton base to new receptors through replacement of its RGD motif with other receptor-specific peptide motifs. Gene Ther. 2: 750-756.
69. Wickham, T. J., Segal, D. M., Roelvink, P. W., Carrion, M. E., Lizonova, A., Lee, G. M. and Kovesdi, I. (1996) Targeted adenovirus gene transfer to endothelial and smooth muscle cells by using bispecific antibodies. J. Virol. 70: 6831-6838.
70. Wilson, J. M., Engelhardt, J. F., Grossman, M., Simon, R. H. and Yang, Y. (1994) Gene therapy of cystic fibrosis lung disease using E1 deleted adenoviruses: a phase I trial. Hum. Gene Ther. 5: 501-519.
71. Wu, N., Watkins, S. C., Schaffer, P. A. and DeLuca, N. A. (1996) Prolonged gene expression and cell survival after infection by a herpes simplex virus mutant defective in the immediate-early genes encoding ICP4, ICP27, and ICP22. J. Virol. 70: 6358-6369.
72. Yang, Y. and Wilson, J. M. (1995) Clearance of adenovirus-infected hepatocytes by MHC class I-restricted CD4+ CTLs in vivo. J. Immunol. 155: 2564-2570.
73. Yang, Y., Su, Q. and Wilson, J. M. (1996) Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs. J. Virol. 70: 7209-7212.
74. Zhu, Z., DeLuca, N. A. and Schaffer, P. A. (1996) Overexpression of the herpes simplex virus type 1 immediate-early regulatory protein, ICP27, is responsible for the aberrant localization of ICPO and mutant forms of ICP4 in ICP4 mutant virus-infected cells. J. Virol. 70: 5346-5356.
75. Zitvogel, L., Tahara, H., Robbins, P. D., Storkus, W. J., Clarke, M. R., Nalesnik, M. A. and Lotze, M. T. (1995) Cancer immunotherapy of established tumors with IL-12. Effective delivery by genetically engineered fibroblasts. J. Immunol. 155: 1393-1403.