Herpes simplex virus type 1/adeno-associated virus rep+ hybrid amplicon vector improves the stability of transgene expression in human cells by site-specific integration

Journal of Virology

Volumn 76, Issue 14, 2002, Pages 7150-7162

  Wang, Y ^a   Camp, S M ^b   Niwano, M ^a,c   Shen, X ^a   Bakowska, J C ^b   Breakefield, X O ^b   Allen, P D ^a  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c KYOTO UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

AMPLICON; ARTICLE; CONTROLLED STUDY; DNA RECOMBINATION; GENE EXPRESSION; HERPES SIMPLEX VIRUS 1; HUMAN; HUMAN CELL; INVERTED TERMINAL REPEAT; MYOBLAST; PRIORITY JOURNAL; ADENO ASSOCIATED VIRUS; CELL LINE; EVALUATION; GENE AMPLIFICATION; GENE TRANSFER; GENE VECTOR; GENETIC RECOMBINATION; GENETIC TRANSDUCTION; GENETICS; LONG TERMINAL REPEAT; METABOLISM; TRANSGENE; VIRUS DNA CELL DNA INTERACTION;

ADENO-ASSOCIATED VIRUS; HUMAN HERPESVIRUS 1;

DNA BINDING PROTEIN; REP PROTEINS, ADENO ASSOCIATED VIRUS 2; REP PROTEINS, ADENO-ASSOCIATED VIRUS 2; VIRUS PROTEIN;

CELL LINE; DEPENDOVIRUS; DNA-BINDING PROTEINS; GENE AMPLIFICATION; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HERPESVIRUS 1, HUMAN; HUMANS; RECOMBINATION, GENETIC; TERMINAL REPEAT SEQUENCES; TRANSDUCTION, GENETIC; TRANSGENES; VIRAL PROTEINS; VIRUS INTEGRATION;

EID: 0036632792     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/JVI.76.14.7150-7162.2002     Document Type: Article

References (61)

1. Balague, C., M. Kalla, and W. W. Zhang. 1997. Adeno-associated virus Rep78 protein and terminal repeats enhance integration of DNA sequences into the cellular genome. J. Virol. 71:3299-3306.
2. Berns, K. I., and C. Giraud. 1996. Biology of adeno-associated virus. Curr. Top. Microbiol. Immunol. 218:1-23.
3. Brister, J. R., and N. Muzyczka. 2000. Mechanism of Rep-mediated adeno-associated virus origin nicking. J. Virol. 74:7762-7771.
4. Buller, R. M., J. E. Janik, E. D. Sebring, and J. A. Rose. 1981. Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J. Virol. 40:241-247.
5. Cheung, A. K., 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.
6. Costantini, L. C., D. R. Jacoby, S. Wang, C. Fraefel, X. O. Breakefield, and O. Isacson. 1999. Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors. Hum. Gene Ther. 10:2481-2494.
7. Duan, D., P. Sharma, J. Yang, Y. Yue, L. Dudus, Y. Zhang, K. J. Fisher, and J. F. Engelhardt. 1998. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J. Virol. 72:8568-8577.
8. During, M. J., J. R. Naegele, K. L. O'Malley, and A. I. Geller. 1994. Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266:1399-1403.
9. Federoff, H. J., A. Brooks, B. Muhkerjee, and T. Corden. 1997. Somatic gene transfer approaches to manipulate neural networks. J. Neurosci. Methods 71:133-142.
10. Fraefel, C., D. R. Jacoby, C. Lage, H. Hilderbrand, J. Y. Chou, F. W. Alt, X. O. Breakefield, and J. A. Majzoub. 1997. Gene transfer into hepatocytes mediated by helper virus-free HSV/AAV hybrid vectors. Mol. Med. 3:813-825.
11. Fraefel, C., S. Song, F. Lim, P. Lang, L. Yu, Y. Wang, P. Wild, and A. I. Geller. 1996. Helper virus-free transfer of herpes simplex virus type 1 plasmid vectors into neural cells. J. Virol. 70:7190-7197.
12. Geller, A. I., and X. O. Breakefield. 1988. A defective HSV-1 vector expresses Escherichia coli beta-galactosidase in cultured peripheral neurons. Science 241:1667-1669.
13. Hamilton, D. L., and K. Abremski. 1984. Site-specific recombination by the bacteriophage P1 lox-Cre system. Cre-mediated synapsis of two lox sites. J. Mol. Biol. 178:481-486.
14. Handa, H., and B. J. Carter. 1979. Adeno-associated virus DNA replication complexes in herpes simplex virus or adenovirus-infected cells. J. Biol. Chem. 254:6603-6610.
15. 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.
16. 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.
17. Heister, T., I. Heid, M. Ackermann, and C. Fraefel. 2002. Herpes simplex virus type 1/adeno-associated virus hybrid vectors mediate site-specific integration at the adeno-associated virus preintegration site, AAVS1, on human chromosome 19. J. Virol. 76:7163-7173.
18. Heller, R. A., K. Song, D. Villaret, R. Margolskee, J. Dunne, H. Hayakawa, and G. M. Ringold. 1990. Amplified expression of tumor necrosis factor receptor in cells transfected with Epstein-Barr virus shuttle vector cDNA libraries. J. Biol. Chem. 265:5708-5717.
19. Hoggan, M. D., N. R. Blacklow, and W. P. Rowe. 1966. Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc. Natl. Acad. Sci. USA 55:1467-1474.
20. Huard, J., G. Acsadi, A. Jani, B. Massie, and G. Karpati. 1994. Gene transfer into skeletal muscles by isogenic myoblasts. Hum. Gene Ther. 5:949-958.
21. Huard, J., G. Akkaraju, S. C. Watkins, M. Pike-Cavalcoli, and J. C. Glorioso. 1997. LacZ gene transfer to skeletal muscle using a replication-defective herpes simplex virus type 1 mutant vector. Hum. Gene Ther. 8:439-452.
22. Jin, B. K., M. Belloni, B. Conti, H. J. Federoff, R. Starr, J. H. Son, H. Baker, and T. H. Joh. 1996. Prolonged in vivo gene expression driven by a tyrosine hydroxylase promoter in a defective herpes simplex virus amplicon vector. Hum. Gene Ther. 7:2015-2024.
23. Johnston, K. M., D. Jacoby, P. A. Pechan, C. Fraefel, P. Borghesani, D. Schuback, R. J. Dunn, F. I. Smith, and X. O. Breakefield. 1997. HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells. Hum. Gene Ther. 8:359-370.
24. Kotin, R. M., R. M. Linden, and K. I. Berns. 1992. Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J. 11:5071-5078.
25. Kotin, R. M., J. C. Menninger, D. C. Ward, and K. I. Berns. 1991. Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter. Genomics 10:831-834.
26. Kotin, R. M., M. Siniscalco, R. J. Saraulski, X. D. 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.
27. Lam, P. Y., and X. O. Breakefield. 2000. Hybrid vector designs to control the delivery, fate and expression of transgenes. J. Gene Med. 2:395-408.
28. Lamartina, S., G. Roscilli, D. Rinaudo, P. Delmastro, and C. Toniatti. 1998. Lipofection of purified adeno-associated virus Rep68 protein: toward a chromosome-targeting nonviral particle. J. Virol. 72:7653-7658.
29. Laughlin, C. A., J. D. Tratschin, H. Coon, and B. J. Carter. 1983. Cloning of infectious adeno-associated virus genomes in bacterial plasmids. Gene 23: 65-73.
30. Liu, H. S., M. S. Jan, C. K. Chou, P. H. Chen, and N. J. Ke. 1999. Is green fluorescent protein toxic to the living cells? Biochem. Biophys. Res. Commun. 260:712-717.
31. Miao, C. H., R. O. Snyder, D. B. Schowalter, G. A. Patijn, B. Donahue, B. Winther, and M. A. Kay. 1998. The kinetics of rAAV integration in the liver. Nat. Genet. 19:13-15.
32. Nakai, H., S. R. Yant, T. A. Storm, S. Fuess, L. Meuse, and M. A. Kay. 2001. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol. 75:6969-6976.
33. Neill, J. C., M. R. Sarkisian, Y. Wang, Z. Liu, L. Yu, P. Tandon, G. Zhang, G. L. Holmes, and A. I. Geller. 2001. Enhanced auditory reversal learning by genetic activation of protein kinase C in small groups of rat hippocampal neurons. Brain Res. Mol. Brain Res. 93:127-136.
34. Palombo, F., A. Monciotti, A. Recchia, R. Cortese, G. Ciliberto, and N. La Monica. 1998. Site-specific integration in mammalian cells mediated by a new hybrid baculovirus-adeno-associated virus vector. J. Virol. 72:5025-5034.
35. Ponnazhagan, S., D. Erikson, W. G. Kearns, S. Z. Zhou, P. Nahreini, X. S. Wang, and A. Srivastava. 1997. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum. Gene Ther. 8:275-284.
36. Recchia, A., R. J. Parks, S. Lamartina, C. Toniatti, L. Pieroni, F. Palombo, G. Ciliberto, F. L. Graham, R. Cortese, N. La Monica, and S. Colloca. 1999. Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 96:2615-2620.
37. Rutledge, E. A., and D. W. Russell. 1997. Adeno-associated virus vector integration junctions. J. Virol. 71:8429-8436.
38. Samulski, R. J., K. I. Berns, M. Tan, and N. Muzyczka. 1982. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc. Natl. Acad. Sci. USA 79:2077-2081.
39. 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.
40. Satoh, W., Y. Hirai, K. Tamayose, and T. Shimada. 2000. Site-specific integration of an adeno-associated virus vector plasmid mediated by regulated expression of rep based on Cre-loxP recombination. J. Virol. 74:10631-10638.
41. Sena-Esteves, M., Y. Saeki, C. Fraefel, and X. O. Breakefield. 2000. HSV-1 amplicon vectors - simplicity and versatility. Mol. Ther. 2:9-15.
42. Song, S., Y. Wang, S. Y. Bak, M. J. During, J. Bryan, O. Ashe, D. B. Ullrey, L. E. Trask, F. D. Grant, K. L. O'Malley, H. Riedel, D. S. Goldstein, K. A. Neve, G. J. LaHoste, J. F. Marshall, J. W. Haycock, R. L. Neve, and A. I. Geller. 1998. Modulation of rat rotational behavior by direct gene transfer of constitutively active protein kinase C into nigrostriatal neurons. J. Neurosci. 18:4119-4132.
43. Spaete, R. R., and N. Frenkel. 1982. The herpes simplex virus amplicon: a new eucaryotic defective-virus cloning-amplifying vector. Cell 30:295-304.
44. Spaete, R. R., and N. Frenkel. 1985. The herpes simplex virus amplicon: analyses of cis-acting replication functions. Proc. Natl. Acad. Sci. USA 82: 694-698.
45. Srivastava, A., E. W. Lusby, and K. I. Berns. 1983. Nucleotide sequence and organization of the adeno-associated virus 2 genome. J. Virol. 45:555-564.
46. Starr, P. A., F. Lim, F. D. Grant, L. Trask, P. Lang, L. Yu, and A. I. Geller. 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.
47. Sternberg, N., and D. Hamilton. 1981. Bacteriophage P1 site-specific recombination. I. Recombination between loxP sites. J. Mol. Biol. 150:467-486.
48. Surosky, R. T., M. Urabe, S. G. Godwin, S. A. McQuiston, G. J. Kurtzman, K. Ozawa, and G. Natsoulis. 1997. Adeno-associated virus Rep proteins target DNA sequences to a unique locus in the human genome. J. Virol. 71:7951-7959.
49. Thyagarajan, B., M. J. Guimaraes, A. C. Groth, and M. P. Calos. 2000. Mammalian genomes contain active recombinase recognition sites. Gene 244:47-54.
50. Tsai, D. J., J. J. Ho, C. R. Ozawa, and R. M. Sapolsky. 2000. Long-term expression driven by herpes simplex virus type-1 amplicons may fail due to eventual degradation or extrusion of introduced transgenes. Exp. Neurol. 165:58-65.
51. Wade-Martins, R., E. R. Smith, E. Tyminski, E. A. Chiocca, and Y. Saeki. 2001. An infectious transfer and expression system for genomic DNA loci in human and mouse cells. Nat. Biotechnol. 19:1067-1070.
52. Wang, X. S., S. Ponnazhagan, and A. Srivastava. 1996. Rescue and replication of adeno-associated virus type 2 as well as vector DNA sequences from recombinant plasmids containing deletions in the viral inverted terminal repeats: selective encapsidation of viral genomes in progeny virions. J. Virol. 70:1668-1677.
53. Wang, Y., C. Fraefel, F. Protasi, R. A. Moore, J. D. Fessenden, I. N. Pessah, A. DiFrancesco, X. Breakefield, and P. D. Allen. 2000. HSV-1 amplicon vectors are a highly efficient gene delivery system for skeletal muscle myoblasts and myotubes. Am. J. Physiol. Cell. Physiol. 278:C619-C626.
54. Wang, Y., L. Yu, and A. I. Geller. 1999. Diverse stabilities of expression in the rat brain from different cellular promoters in a helper virus-free herpes simplex virus type 1 vector system. Hum. Gene Ther. 10:1763-1771.
55. Weindler, F. W., and R. Heilbronn. 1991. A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication. J. Virol. 65:2476-2483.
56. Weitzman, M. D., K. J. Fisher, and J. M. Wilson. 1996. Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers. J. Virol. 70:1845-1854.
57. Weitzman, M. D., S. R. Kjostio, R. M. Kotin, and R. A. Owens. 1994. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc. Natl. Acad. Sci. USA 91:5808-5812.
58. Yakinoglu, 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. 48:3123-3129.
59. Yakobson, B., T. A. Hrjnko, M. J. Peak, and E. Winocour. 1989. Replication of adeno-associated virus in cells irradiated with UV light at 254 nm. J. Virol. 63:1023-1030.
60. Yang, C. C., X. Xiao, X. Zhu, D. C. Ansardi, N. D. Epstein, M. R. Frey, A. G. Matera, and R. J. Samulski. 1997. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J. Virol. 71:9231-9247.
61. Zhang, G. R., X. Wang, T. Yang, M. Sun, W. Zhang, Y. Wang, and A. I. Geller. 2000. A tyrosine hydroxylase-neurofilament chimeric promoter enhances long-term expression in rat forebrain neurons from helper virus-free HSV-1 vectors. Brain Res. Mol. Brain Res. 84:17-31.