Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products

Journal of Virology

Volumn 71, Issue 3, 1997, Pages 1897-1905

  Vincent, Karen A ^a   Piraino, Susan T ^a   Wadsworth, Samuel C ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CAPSID PROTEIN; GENE PRODUCT; HELICASE; VIRUS PROTEIN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; GENE EXPRESSION; GENE TRANSFER; HUMAN; HUMAN CELL; LONG TERMINAL REPEAT; PRIORITY JOURNAL; ROUS SARCOMA ONCOVIRUS; VIRUS CAPSID; VIRUS RECOMBINANT; VIRUS REPLICATION;

EID: 0031041173     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.71.3.1897-1905.1997     Document Type: Article

References (61)

1. Antoni, B. A., A. B. Rabson, I. L. Miller, J. P. Trempe, N. Chejanovsky, and B. J. Carter. 1991. Adeno-associated virus Rep protein inhibits human immunodeficiency virus type 1 production in human cells. J. Virol. 65:396-404.
2. Beaton, A., P. Palumbo, and K. I. Berns. 1989. Expression from the adeno-associated virus p5 and p19 promoters is negatively regulated in trans by the rep protein. J. Virol. 63:4450-4454.
3. Berns, K. I., and R. M. Linden. 1995. The cryptic life style of adeno-associated virus. Bioessays 3:237-245.
4. Blacklow, N. R., M. D. Hoggan, A. Z. Kapikian, J. B. Austin, and W. P. Rowe. 1968. Epidemiology of adeno-associated virus infection in a nursery population. Am. J. Epidemiol. 8:368-378.
5. Capone, J. P., P. A. Sharp, and U. L. RajBhandary. 1985. Amber, ochre and opal suppressor tRNA genes derived from a human serine tRNA gene. EMBO J. 4:213-221.
6. Carter, B. J. 1992. Adeno-associated virus vectors. Curr. Opin. Biotechnol. 3:533-539.
7. Chang, L.-S., Y. Shi, and T. Shenk. 1989. Adeno-associated virus p5 promoter contains an adenovirus E1A-inducible element and a binding site for the major late transcription factor. J. Virol. 63:3479-3488.
8. Chejanovsky, N., and B. J. Carter. 1989. Replication of a human parvovirus nonsense mutant in mammalian cells containing an inducible amber suppressor. Virology 171:239-247.
9. Cheung, A. K. M., M. D. Hoggan, W. W. Hauswirth, and K. I. Berns. 1980. Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J. Virol. 33:739-748.
10. Chiorini, J. A., C. M. Wendtner, E. Urcelay, B. Safer, M. Halles, and R. M. Kotin. 1995. High-efficiency transfer of the T cell costimulatory molecule B7-2 to lymphoid cells using high-titer recombinant adeno-associated virus vectors. Hum. Gene Ther. 6:1531-1541.
11. Clark, K. R., F. Voulgaropoulou, D. M. Fraley, and P. R. Johnson. 1995 .Cell lines for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6:1329-1341.
12. Einerhand, M. P. W., M. Antoniou, S. Zolotukhin, N. Muzyczka, K. I. Berns, F. Grosveld, and D. Valerio. 1995. Regulated high-level human β-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer. Gene Ther. 2:336-343.
13. Ensinger, M. J., and H. S. Ginsberg. 1972. Selection and preliminary characterization of temperature-sensitive mutants of type 5 adenovirus. J. Virol. 10:328-339.
14. Ferrari, F. K., T. Samulski, T. Shenk, and R. J. Samulski. 1996. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70:3227-3234.
15. Fisher, K. J., G.-P. Gao, M. D. Weitzman, R. DeMatteo, J. F. Burda, and J. M. Wilson. 1996. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70:520-532.
16. Flotte, T. R., S. A. Afione, C. Conrad, S. A. McGrath, R. Solow, H. Oka, P. L. Zeitlin, W. B. Guggino, and B. J. Carter. 1993. Stable in vivo expression of the cystic fibrosis transmembrane regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90:10613-10617.
17. Flotte, T. R., and B. J. Carter. 1995. Adeno-associated virus vectors for gene therapy. Gene Ther. 2:357-362.
18. Flotte, T. R., X. Barraza-Ortiz, R. Solow, S. A. Afione, B. J. Carter, and W. B. Guggino. 1995. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 2:29-37.
19. Graham, F. L., J. Smiley, W. C. Russell, and R. Nairn. 1977. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36:59-74.
20. Halbert, C. L., I. E. Alexander, G. M. Wolgamot, and A. D. Miller. 1995. Adeno-associated vectors transduce primary cells much less efficiently than immortalized cells. J. Virol. 69:1473-1479.
21. Handa, H., K. Shiroki, and H. Shimojo. 1977. Establishment and characterization of KB cell lines latently infected with adeno-associated virus type 1. Virology 82:84-92.
22. Heilbronn, R., A. Burkle, S. Stephan, and H. zur Hausen. 1990. The adeno-associated virus rep gene suppresses herpes simplex virus-induced DNA amplification. J. Virol. 64:3012-3018.
23. Higuchi, R., B. Krummel, and R. K. Saiki. 1988. A general method of in vitro preparation and specific mutagenesis of DNA fragments: study of protein and DNA interactions. Nucleic Acids Res. 16:7351-7367.
24. Hirt, B. 1967. Selective extraction of polyoma DNA from infected mouse cell cultures. J. Mol. Biol. 26:365-369.
25. Kölscher, C., M. Hörer, J. A. Kleinschmidt, H. Zentgraf, A. Bürkle, and R. Heilbronn. 1994. Cell lines inducibly expressing the adeno-associated virus (AAV) rep gene: requirements for productive replication of rep-negative AAV mutants. J. Virol. 68:7169-7177.
26. Hölscher, C., J. A. Kleinschmidt, and A. Bürkle. 1995. High-level expression of adeno-associated virus (AAV) Rep78 or Rep68 protein is sufficient for infectious-particle formation by a rep-negative AAV mutant. J. Virol. 69: 6880-6885.
27. Horer, M., S. Weger, K. Butz, F. Hoppe-Seyler, C. Geisen, and J. A. Kleinschmidt. 1995. Mutational analysis of adeno-associated virus Rep protein-mediated inhibition of heterologous and homologous promoters. J. Virol. 69:5485-5496.
28. Janik, J. E., M. M. Huston, K. Cho, and J. A. Rose. 1989. Efficient synthesis of adeno-associated virus structural proteins requires both adenovirus DNA binding protein and VA1 RNA. Virology 168:320-329.
29. Kaplitt, M. G., P. Leone, R. J. Samulski, X. Xiao, D. W. Pfaff, K. L. O'Malley, and M. J. During. 1994. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8:148-153.
30. Kotin, R. M., M. Siniscalco, R. J. Samulski, X. Zhu, L. Hunter, C. A. Laughlin, S. McLaughlin, N. Muzyczka, M. Rocchi, and K. I. Berns. 1990. Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87:2211-2215.
31. Kotin, R. M. 1994. Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther. 5:793-801.
32. 19 mRNA levels. J. Virol. 68:2947-2957.
33. 5 Rep binding site and the consensus ATP-binding motif of the AAV Rep68 protein. J. Virol. 69:6787-6796.
34. Labow, M. A., P. L. Hermonat, and K. I. Berns. 1986. Positive and negative autoregulation of adeno-associated virus type 2 genome. J. Virol. 60:251-258.
35. Labow, M. A., L. H. Graf, and K. I. Berns. 1987. Adeno-associated virus gene expression inhibits cellular transformation by heterologous genes. Mol. Cell. Biol. 7:1320-1325.
36. Laughlin, C. A., C. B. Cardellichio, and J. C. Coon. 1986. Latent infection of KB cells with adeno-associated virus type 2. J. Virol. 60:515-524.
37. Lebkowski, J.-S., M. M. McNally, T. B. Okarma, and L. B. Lerch. 1988. Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types. Mol. Cell. Biol. 8:3988-3996.
38. Lee, E. R., J. Marshall, C. S. Siegel, C. Jiang, N. S. Yew, M. R. Nichols, J. B. Nietupski, R. J. Ziegler, M. Lane, K. X. Wang, N. C. Wan, R. K. Scheule, D. J. Harris, A. E. Smith, and S. H. Cheng. 1996. Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum. Gene Ther. 7:1701-1717.
39. Lynn, D. E. 1992. A BASIC computer program for analyzing endpoint assays. BioTechniques 12:880-881.
40. Mamounas, M., M. Leavitt, M. Yu, and F. Wong-Staal. 1995. Increased titer of recombinant AAV vectors by gene transfer with adenovirus coupled to DNA-polylysine complexes. Gene Ther. 2:429-432.
41. McCarty, D. M., M. Christensen, and N. Muzyczka. 1991. Sequences required for coordinate induction of adeno-associated virus p19 and p40 by Rep protein. J. Virol. 65:2936-2945.
42. McCarty, D. M., D. J. Periera, I. Zolotukhin, X. Zhou, J. H. Ryan, and N. Muzyczka. 1994. Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein. J. Virol. 68:4988-4997.
43. McLaughlin, S. K., P. Collis, P. L. Hermonat, and N. Muzyczka. 1988. Adeno-associated virus general transduction vectors: analysis of proviral structures. J. Virol. 62:1963-1973.
44. Mendelson, E., J. P. Trempe, and B. J. Carter. 1986. Identification of the trans-acting Rep proteins of adeno-associated virus by antibodies to a synthetic oligopeptide. J. Virol. 60:823-832.
45. Muzyczka, N. 1992. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158:97-129.
46. Oelze, I., K. Rittner, and G. Sczakiel. 1994. Adeno-associated virus type 2 rep gene-mediated inhibition of basal gene expression of human immunodeficiency virus type 1 involves its negative regulatory functions. J. Virol. 68: 1229-1233.
47. Podsakoff, G., K. K. Wong, and S. Chatterjee. 1994. Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors. J. Virol. 68: 5656-5666.
48. rep and the HIV-1 long terminal repeat. J. Gen. Virol. 73:2977-2981.
49. Russell, D. W., A. D. Miller, and I. E. Alexander. 1994. Adeno-associated virus vectors preferentially transduce cells in S phase. Proc. Natl. Acad. Sci. USA 91:8915-8919.
50. Saiki, R. K., D. H. Gelfand, S. Stoffer, S. J. Scharf, R. Higuchi, G. T. Horn, K. B. Mullis, and H. A. Erlich. 1988. Primer-directed enzymatic amplification of DNA with a thermostable DNA polymerase. Science 239:487-491.
51. Sambrook, J., E. F. Fritsch, and T. Maniatis. 1989. Molecular cloning: a laboratory manual, 2nd ed. Cold Spring Harbor Laboratory, Cold Spring Harbor, N.Y.
52. Samulski, R. J., L.-S. Chang, and T. Shenk. 1989. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J. Virol. 63:3822-3828.
53. Samulski, R. J., X. Zhu, X. Xiao, J. D. Brook, D. E. Housman, N. Epstein, and L. A. Hunter. 1991. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J. 10:3941-3950.
54. Shi, Y., E. Seto, L.-S. Chang, and T. Shenk. 1991. Transcriptional repression by YY1, a human GLI-Kruppel-related protein, and relief of repression by adenovirus E1A protein. Cell 76:377-388.
55. Stillman, B. W., F. Tamanoi, and M. B. Mathews. 1982. Purification of an adenovirus-coded DNA polymerase that is required for initiation of DNA replication. Cell 31:613-623.
56. Thrasher, A. J., M. de Alwis, C. M. Casimir, C. Kinnow, K. Page, J. Lebkowski, A. W. Segal, and R. J. Levinsky. 1995. Generation of recombinant adeno-associated virus (rAAV) from an adenoviral vector and functional reconstitution of the NADPH-oxidase. Gene Ther. 2:481-485.
57. Tratschin, J. D., J. Tal, and B. J. Carter. 1986. Negative and positive regulation in trans of gene expression from adeno-associated virus vectors in mammalian cells by a viral rep gene product. Mol. Cell. Biol. 6:2884-2894.
58. Trempe, J. P., and B. J. Carter. 1988. Regulation of adeno-associated virus gene expression in 293 cells: control of mRNA abundance and translation. J. Virol. 62:68-74.
59. Vincent, K. A., G. K. Moore, and N. L. Haigwood. 1990. Replication and packaging of HIV envelope genes in a novel adeno-associated virus vector system, p. 353-359. In F. Brown, R. M. Chanock, H. S. Ginsberg, and R. A. Lerner (ed.), Vaccines 90: modern approaches to new vaccines including prevention of AIDS. Cold Spring Harbor Laboratory Press, Cold Spring Harbor, N.Y.
60. West, M. H. P., J. P. Trempe, J.-D. Tratschin, and B. J. Carter. 1987. Gene expression in adeno-associated virus vectors: the effects of chimeric mRNA structure, helper virus, and adenovirus VA1 RNA. Virology 160:38-47.
61. Yang, Q., F. Chen, and J. P. Trempe. 1994. Characterization of cell lines that inducibly express the adeno-associated virus Rep proteins. J. Virol. 68:4847-4856.