An adenoviral expression system for AAV rep78 using homologous recombination

Molecular Therapy

Volumn 6, Issue 1, 2002, Pages 91-98

  Carlson, Cheryl A ^a   Shayakhmetov, Dmitry M ^b   Lieber, André ^a,b  

^a UNIVERSITY OF WASHINGTON   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

AAV rep78; AAV rescue; Adenoviral vectors; Homologous recombination

Indexed keywords

ADENOVIRUS VECTOR; PLASMID DNA; REP78 PROTEIN; UNCLASSIFIED DRUG; VIRUS DNA; VIRUS PROTEIN;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ANIMAL CELL; ARTICLE; CELL DIVISION; CONTROLLED STUDY; CYTOTOXICITY; DNA REPLICATION; GENE AMPLIFICATION; GENE EXPRESSION SYSTEM; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; INVERTED TERMINAL REPEAT; NONHUMAN; OPEN READING FRAME; PROGENY; TRANSGENE; VIRUS GENOME; VIRUS INFECTION; VIRUS REPLICATION;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; DNA VIRUSES;

EID: 0036665599     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0634     Document Type: Article

References (50)

1. Hitt, M. M., Addison, C. L., and Graham, F. L. (1997). Human adenovirus vectors for gene transfer into mammalian cells. Adv. Pharmacol. 40: 137-206.
2. Wang, Q., Greenburg, G., Bunch, D., Farson, D., and Finer, M. H. (1997). Persistent transgene expression in mouse liver following in vivo gene transfer with a Δ E1/Δ E4 adenovirus vector. Gene Ther. 4: 393-400.
3. Yang, Y., Ertl, H. C., and Wilson, J. M. (1994). MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunity 1: 433-442.
4. Kochanek, S. (1999). High-capacity adenoviral vectors for gene transfer and somatic gene therapy. Hum. Gene Ther. 10: 2451-2459.
5. Sandig, V., et al. (2000). Optimization of the helper-dependent adenovirus system for production and potency in vivo. Proc. Natl. Acad. Sci. USA 97: 1002-1007.
6. Srivastava, A., Lusby, E. W., and Berns, K. I. (1983). Nucleotide sequence and organization of the adeno-associated virus 2 genome. J. Virol. 45: 555-564.
7. Berns, K. I. (1990). Parvovirus replication. Microbiol. Rev. 54: 316-329.
8. Kotin, R. M., et al. (1990). Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87: 2211-2215.
9. Samulski, R. J., et al. (1991). Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J. 10: 3941-3950.
10. Nahreini, P., and Srivastava, A. (1989). Rescue and replication of the adeno-associated virus 2 genome in mortal and immortal human cells. Intervirology 30: 74-85.
11. Hauswirth, W. W., and Berns, K. I. (1977). Origin and termination of adeno-associated virus DNA replication. Virology 78: 488-499.
12. Srivastava, A. (1987). Replication of the adeno-associated virus DNA termini in vitro. Intervirology 27: 138-147.
13. Mendelson, E., Trempe, J. P., and Carter, B. J. (1986). Identification of the trans-acting Rep proteins of adeno-associated virus by antibodies to a synthetic oligopeptide. J. Virol. 60: 823-832.
14. Im, D. S., and Muzyczka, N. (1992). Partial purification of adeno-associated virus Rep78, Rep52, and Rep40 and their biochemical characterization. J. Virol. 66: 1119-1128.
15. Pereira, D. J., McCarty, D. M., and Muzyczka, N. (1997). The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection. J. Virol. 71: 1079-1088.
16. Labow, M. A., Graf, L. H., Jr., and Berns, K. I. (1987). Adeno-associated virus gene expression inhibits cellular transformation by heterologous genes. Mol. Cell. Biol. 7: 1320-1325.
17. Ni, T. H., Zhou, X., McCarty, D. M., Zolotukhin, I., and Muzyczka, N. (1994). In vitro replication of adeno-associated virus DNA. J. Virol. 68: 1128-1138.
18. Nahreini, P., Woody, M. J., Zhou, S. Z., and Srivastava, A. (1993). Versatile adeno-associated virus 2-based vectors for constructing recombinant virions. Gene 124: 257-262.
19. Linden, R. M., Ward, P., Giraud, C., Winocour, E., and Berns, K. I. (1996). Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 93: 11288-11294.
20. Surosky, R. T., et al. (1997). Adeno-associated virus Rep proteins target DNA sequences to a unique locus in the human genome. J. Virol. 71: 7951-7959.
21. Lamartina, S., Roscilli, G., Rinaudo, D., Delmastro, P., and Toniatti, C. (1998). Lipofection of purified adeno-associated virus Rep68 protein: toward a chromosome-targeting nonviral particle. J. Virol. 72: 7653-7658.
22. Casto, B. C., Atchison, R. W., and Hammon, W. M. (1967). Studies on the relationship between adeno-associated virus type I (AAV-1) and adenoviruses. I. Replication of AAV-1 in certain cell cultures and its effect on helper adenovirus. Virology 32: 52-59.
23. Casto, B. C., Armstrong, J. A., Atchison, R. W., and Hammon, W. M. (1967). Studies on the relationship between adeno-associated virus type 1 (AAV-1) and adenoviruses. II. Inhibition of adenovirus plaques by AAV; its nature and specificity. Virology 33: 452-458.
24. Weitzman, M. D., Fisher, K. J., and Wilson, J. M. (1996). Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers. J. Virol. 70: 1845-1854.
25. Carlson, C. A., Shayakhmetov, D. M., and Lieber, A. (2002). Restoration of a functional open reading frame by homologous recombination between two adenoviral vectors. Mol. Ther. 6: 99-105, doi:10.1006/mthe.2001.0635.
26. Okuyama, T., et al. (1996). Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-α 1-antitrypsin promoter dramatically increases expression of human α1-antitrypsin in vivo. Hum. Gene Ther. 7: 637-645.
27. Steinwaerder, D. S., and Lieber, A. (2000). Insulation from viral transcriptional regulatory elements improves inducible transgene expression from adenovirus vectors in vitro and in vivo. Gene Ther. 7: 556-567.
28. Fisher, K. J., Kelley, W. M., Burda, J. F., and Wilson, J. M. (1996). A novel adenovirus-adeno-associated virus hybrid vector that displays efficient rescue and delivery of the AAV genome. Hum. Gene Ther. 7: 2079-2087.
29. Lieber, A., Steinwaerder, D. S., Carlson, C. A., and Kay, M. A. (1999). Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. J. Virol. 73: 9314-9324.
30. Babiss, L. E., Friedman, J. M., and Darnell, J. E., Jr. (1986). Cellular promoters incorporated into the adenovirus genome: effects of viral regulatory elements on transcription rates and cell specificity of albumin and β-globin promoters. Mol. Cell. Biol. 6: 3798-3806.
31. Palla, F., Melfi, R., Anello, L., Di Bernardo, M., and Spinelli, G. (1997). Enhancer blocking activity located near the 3′ end of the sea urchin early H2A histone gene. Proc. Natl. Acad. Sci. USA 94: 2272-2277.
32. Parnell, T. J., and Geyer, P. K. (2000). Differences in insulator properties revealed by enhancer blocking assays on episomes. EMBO J. 19: 5864-5874.
33. Steinwaerder, D. S., Carlson, C. A., and Lieber, A. (2000). DNA replication of first-generation adenovirus vectors in tumor cells. Hum. Gene Ther. 11: 1933-1948.
34. Graham, F. L., Smiley, J., Russell, W. C., and Nairn, R. (1977). Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36: 59-74.
35. Zhang, H. G., et al. (2001). Recombinant adenovirus expressing adeno-associated virus cap and rep proteins supports production of high-titer recombinant adeno-associated virus. Gene Ther. 8: 704-712.
36. Recchia, A., et al. (1999). Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 96: 2615-2620.
37. Ward, P., Urcelay, E., Kotin, R., Safer, B., and Berns, K. I. (1994). Adeno-associated virus DNA replication in vitro: activation by a maltose binding protein/Rep 68 fusion protein. J. Virol. 68: 6029-6037.
38. Leonard, C. J., and Berns, K. I. (1994). Cloning, expression, and partial purification of Rep78: an adeno-associated virus replication protein. Virology 200: 566-573.
39. Fisher, K. J., et al. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70: 520-532.
40. Ni, T. H., et al. (1998). Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection. J. Virol. 72: 2777-2787.
41. Miao, C. H., et al. (2000). Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol. Ther. 1: 522-532, doi:10.1006/mthe.2000.0075.
42. McGrory, W. J., Bautista, D. S., and Graham, F. L. (1988). A simple technique for the rescue of early region I mutations into infectious human adenovirus type 5. Virology 163: 614-617.
43. Lieber, A., He, C. Y., Kirillova, I., and Kay, M. A. (1996). Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J. Virol. 70: 8944-8960.
44. Shayakhmetov, D. M., and Lieber, A. (2000). Dependence of adenovirus infectivity on length of the fiber shaft domain. J. Virol. 74: 10274-10286.
45. Lieber, A., et al. (1997). The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors. J. Virol. 71: 8798-8807.
46. Kay, M. A., Graham, F., Leland, F., and Woo, S. L. (1995). Therapeutic serum concentrations of human α-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology 21: 815-819.
47. Alexander, I. E., Russell, D. W., Spence, A. M., and Miller, A. D. (1996). Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors. Hum. Gene Ther. 7: 841-850.
48. Nakai, H., Iwaki, Y., Kay, M. A., and Couto, L. B. (1999). Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J. Virol. 73: 5438-5447.
49. Chung, J. H., Bell, A. C., and Felsenfeld, G. (1997). Characterization of the chicken β-globin insulator. Proc. Natl. Acad. Sci. USA 94: 575-580.
50. Bett, A. J., Krougliak, V., and Graham, F. L. (1995). DNA sequence of the deletion/insertion in early region 3 of Ad5 dl309. Virus Res. 39: 75-82.