Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

Journal of Virology

Volumn 70, Issue 1, 1996, Pages 520-532

  Fisher, Krishna J ^a   Gao, Guang Ping ^a   Weitzman, Matthew D ^a   Dematteo, Ronald ^a   Burda, John F ^a   Wilson, James M ^b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b WISTAR INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ARTICLE; CONTROLLED STUDY; GENE THERAPY; HUMAN; HUMAN CELL; PARVOVIRUS; PRIORITY JOURNAL; VIRUS REPLICATION;

ADENOVIRUS E1 PROTEINS; ADENOVIRUS E4 PROTEINS; ANIMAL; CELL LINE; CERCOPITHECUS AETHIOPS; DEPENDOVIRUS; DNA, SINGLE-STRANDED; DNA, VIRAL; GENE THERAPY; HELA CELLS; HUMAN; LAC OPERON; LIVER; LUNG; MICE; MICE, INBRED BALB C; OPEN READING FRAMES; RECOMBINATION, GENETIC; SUPPORT, NON-U.S. GOV'T; SUPPORT, U.S. GOV'T, P.H.S.; TRANSFORMATION, GENETIC; VERO CELLS;

EID: 0029655894     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.70.1.520-532.1996     Document Type: Article

References (48)

1. Alexander, I. E., D. W. Russell, and A. D. Miller. 1994. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J. Virol. 68:8282-8287.
2. Babiss, L. E., and H. S. Ginsberg. 1984. Adenovirus type 5 early region 1b gene product is required for efficient shutoff of host protein synthesis. J. Virol. 50:202-212.
3. Berk, A. J. 1986. Functions of adenovirus E1A. Cancer Surv. 5:367-387.
4. Bridge, E., and G. Ketner. 1989. Redundant control of adenovirus late gene expression by early region 4. J Virol. 63:631-638.
5. Bridge, E., S. Medgbalchi, S. Ubol, M. Leesong, and G. Ketner. 1993. Adenovirus early region 4 and viral DNA synthesis. Virology 193:794-801.
6. Carter, B. J. 1990. The growth cycle of adeno-associated virus, p. 155-168. In P. Tijsser (ed.), Handbook of parvoviruses. CRC Press, Inc., Boca Raton, Fla.
7. Carter, B. J., C. J. Marcus-Sekura, C. A. Laughlin, and G. Ketner. 1983. Properties of an adenovirus type 2 mutant, Ad2dl807, having a deletion near the right-hand genome terminus: failure to help AAV replication. Virology 126:505-516.
8. 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.
9. Fields-Berry, S. C., A. L. Halliday, and C. L. Cepko. 1992. A recombinant retrovirus encoding alkaline phosphatase confirms clonal boundary assignment in lineage analysis of murine retina Proc. Natl. Acad. Sci. USA 89:693-697
10. Flotte, T. R., S. A. Afione, R. Solow, M. L. Drumm, D. Markakis, W. B. Guggino, P. L. Zeitlin, and B. J. Carter. 1993. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J. Biol. Chem. 268:3781-3790.
11. Gao, G. Unpublished data.
12. Halbert, D. N., J. R. Cutt, and T. Shenk. 1985. Adenovirus early region 4 encodes functions required for efficient DNA replication, late gene expression, and host cell shutoff. J. Virol. 56:250-257.
13. Hermonat, P. L., M. A. Labow, R. Wright, K. I. Berns, and N. Muzyczka. 1984 Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants. J. Virol. 51:329-339.
14. Hermonat, P. L., and N. Muzyczka, 1984. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc. Natl. Acad. Sci. USA 81:6466-6470.
15. Hirt, B. 1967. Selective extraction of polyoma DNA from infected mouse cell cultures. J. Mol. Biol. 26:365-369.
16. 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.
17. Kaplitt, M. G., R. 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. Nature Genet. 8:148-154.
18. 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.
19. 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.
20. Kozarsky, K. F., and J. M. Wilson. 1993. Gene therapy: adenovirus vectors. Curr. Opin. Genet. Dev. 3:499-503.
21. LaFace, D., P. Hermonat, E. Wakeland, and A. Peck. 1988 Gene transfer into hematopoietic progenitor cells mediated by an adeno-associated virus vector. Virology 162:483-490.
22. 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.
23. McLaugblin, 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.
24. McPherson, R. A., H. S. Ginsberg, and J. A. Rose. 1982. Adeno-associated virus helper activity of adenovirus DNA binding protein. J. Virol. 44:666-673.
25. Myers, M. W., C. A. Laughlin, F. T. Jay, and B. J. Carter. 1980. Adenovirus helper function for growth of adeno-associated virus: effect of temperature-sensitive mutants in adenovirus early region 2. J. Virol. 35:65-75.
26. Pina, M., and M. Green. 1965. Biochemical studies un adenovirus multiplication. IX. Chemical and base composition of 28 human adenoviruses. Proc. Natl. Acad. Sci. USA 54:547-551.
27. Rice, S. A., and D. F. Klessig. 1985. Isolation and analysis of adenovirus type 5 mutants containing deletions in the gene encoding the DNA-binding protein. J. Virol. 56:767-778.
28. Richardson, W. D., and H. Westphal. 1983. Adenovirus early gene regulation and the adeno-associated virus helper effect. Curr. Top. Microbiol. Immunol. 109:147-165.
29. Richardson, W. D., and H. Westphal. 1984. Requirement for either early region la or early region 1b adenovirus gene products in the helper effect for adeno-associated virus. J. Virol. 51:404-410.
30. Samulski, R. J., L.-S. Chang, and T. Shenk. 1987. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61:3096-3101.
31. 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.
32. r polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs. J. Virol. 62:206-210.
33. 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. (Erratum, 11:1228, 1992.)
34. Sarasin, A. 1985. SOS response in mammalian cells. Cancer Invest. 3:163-174
35. Sarnow, P., P. Hearing, C. W. Auderson, D. N. Halbert, T. Shenk, and A. J. Levine. 1984. Adenovirus early region 1B 58,000-dalton tumor antigen is physically associated with an early region 4 25,000-dalton protein in productively infected cells. J. Virol. 49:692-700.
36. Sarnow, P., Y. Ho, J. Williams, and A. J. Levine. 1982. Adenovirus E1B-55 kD tumor antigen and SV40 large tumor antigen are physically associated with the same 54 kD cellular protein in transformed cells Cell 28:387-394
37. Schlehofer, J. R., M. Ehrbar, and H. zur Hausen. 1986. Vaccinia virus. herpes simplex virus, and carcinogens induce DNA amplification in a human cell line and support replication of a helper virus dependent parvoirus. Virology 152:110-121.
38. Shepherd, S. E., J. A. Howe, J. S. Mymryk, and S. T. Bayley. 1993. Induction of the cell cycle in baby rat kidney cells by adenovirus type 5 E1A in the absence of E1B and a possible influence of p53. J. Virol. 67:2944-2949
39. Straus, S. E., H. S. Ginsberg, and J. A. Rose. 1976. DNA-minus temperature-sensitive mutants of adenovirus type 5 help adenovirus-associated virus replication. J. Virol 17:140-148.
40. Tratschin, J.-D., I. L. Miller, and B. J. Carter. 1984. Genetic analysis of adeno-associated virus, properties of deletion mutants constructed in vitro and evidence for an adeno-associated virus replication function J Virol. 51:611-619.
41. Tratschin, J.-D., I. L. Miller, M. G. Smith, and B. J. Carter. 1985. Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells. Mol. Cell. Biol. 5:3251-3260.
42. Weinberg, D. H., and G. Ketner. 1983. A cell line that supports the growth of a defective early region 4 deletion mutant of human adenovirus type 2. Proc. Natl. Acad. Sci USA 80:5383-5386.
43. Weinberg, D. H., and G. Ketner. 1986. Adenoviral early region 4 is required for efficient viral DNA replication and for late gene expression. J. Virol. 57:833-838.
44. 43a.Weitzman, M. D., et al. Unpublished data.
45. West, M. H., 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
46. Wilson, J. M., D. M. Jefferson, J. R. Chowdhury, P. M. Novikoff, D. E. Johnston, and R. C. Mulligan. 1988. Retrovirus-mediated transduction of adult hepatocytes. Proc Natl. Acad. Sci. USA 85:3014-3018.
47. Yakobson, B., T. Koch, and E. Winocour. 1987. Replication of adeno-associated virus in synchronized cells without the addition of a helper virus. J. Virol. 61:972-981.
48. Yankinoglu, A. O., R. Heilbronn, A. Burkle, J. R. Schlehofer, and H. zur Hausen. 1988. DNA amplification of adeno-associated virus as a response to cellular genotoxic stress. Cancer Res. 48:3123-3130.