Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells

Human Gene Therapy

Volumn 8, Issue 3, 1997, Pages 275-284

  Ponnazhagan, Selvarangan ^a   Erikson, Dana ^b   Kearns, William G ^b,c   Zhou, Shang Zhen ^a   Nahreini, Piruz ^a   Wang, Xu Shan ^a   Srivastava, Arun ^a  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CENTER FOR PEDIATRIC RESEARCH   (United States)

^c JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NEOMYCIN; TETRACYCLINE; THYMIDINE KINASE; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANTIBIOTIC RESISTANCE; ARTICLE; CHROMOSOME 19; DNA INTEGRATION; FLUORESCENCE IN SITU HYBRIDIZATION; GENE THERAPY; HERPES VIRUS; HUMAN; HUMAN CELL; NASOPHARYNX CARCINOMA; NONHUMAN; POLYMERASE CHAIN REACTION; PROMOTER REGION; SOUTHERN BLOTTING; VIRUS CAPSID; VIRUS DNA CELL DNA INTERACTION; VIRUS RECOMBINANT;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 2; ADENOVIRIDAE; HERPES; HERPESVIRIDAE; MIRIDAE;

EID: 0030850930     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1997.8.3-275     Document Type: Article

References (50)

1. ASHKTORAB, H., and SRIVASTAVA, A. (1989). Identification of nuclear proteins that specifically interact with the adeno-associated virus type 2 inverted terminal repeat hairpin DNA. J. Virol. 63, 3034-3039.
2. BERNS, K.J. (1990). Parvovirus replication. Microbiol. Rev. 54, 316-329.
3. BERNS, K.I., and BOHENZKY, R.A. (1987). Adeno-associated viruses: An update. Adv. Virus Res. 32, 243-307.
4. BERNS, K.I., PINKERTON, T.C., THOMAS, G.F., and HOGGAN, M.D. (1975). Detection of adeno-associated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells. J. Virol. 68, 556-560.
5. CARTER, B.J. (1992). Adeno-associated virus vectors. Curr. Opin. Biotechnol. 3, 533-538.
6. CHATTERJEE, S., JOHNSON, P.R., and WONG, K.K. (1992). Dual-target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 258, 1485-1488.
7. CHEUNG, A.K., HOGGAN, M.D., HAUSWIRTH, W.W., and BERNS, K.I. (1980). Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J. Virol. 33, 739-748.
8. CHIORINI, J.A., WEINER, S.M., OWENS, R.A., KYOSTIO, S.R.M., KOTIN, R.M., and SAFER, B. (1994). Sequence requirements for stable binding and function of Rep68 on the adeno-associated virus type 2 inverted terminal repeats. J. Virol. 68, 7448-7457.
9. DONAHUE, R.E., KESSLER, S.W., BODINE, D., MCDONAGH, K., DUNBAR, C., GOODMAN, S., AGRICOLA, B., BYRNE, E., RAFFELD, M., MOEN, R., BACHER, J., ZSEBO, K.M., and NIENHUIS, A.W. (1992). Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J. Exp. Med. 176, 1125-1135.
10. FLOTTE, T.R., AFIONE, S.A., CONRAD, C., MCGRATH, S.A., SOLOW, R., OKA, H., ZEITLIN, P.L., GUGGINO, W.B., and CARTER, B.J. (1993). Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90, 10613-10617.
11. GIRAUD, C., WINOCOUR, E., and BERNS, K.I. (1994). Site-specific integration by adeno-associated virus is directed by a cellular DNA sequence. Proc. Natl. Acad. Sci. USA 91, 10039-10043.
12. GIRAUD, C., WINOCOUR, E., and BERNS, K.I. (1995). Recombinant junctions formed by site-specific integration of adeno-associated virus into an episome. J. Virol. 69, 6917-6924.
13. GOODMAN, S., XIAO, X., DONAHUE, R.E., MOULTON, A., MILLER, J., WALSH, C.E., YOUNG, N.S., SAMULSKI, R.J., and NIENHUIS, A.W. (1994). Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 84, 1492-1500.
14. IM, D.-S., and MUZYCZKA, N. (1989). Factors that bind to adeno-associated virus terminal repeats. J. Virol. 63, 3095-3104.
15. IM, D.-S., and MUZYCZKA, N. (1990). The AAV origin binding protein rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity. Cell 61, 447-457.
16. 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.
17. KEARNS, W.G., AFIONE, S.A., FULMER, S.B., PANG, M.G., ERIKSON, D., EGAN, M., LANDRUM, M.J., FLOTTE, T.R., and CUTTING, G.R. (1996). Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther. 3, 748-755.
18. KOTIN, R.M., and BERNS, K.I. (1989). Organization of adeno-associated virus DNA in latently infected Detroit 6 cells. Virology 170, 460-467.
19. KOTIN, R.M., SINISCALCO, M., SAMULSKI, R.J., ZHU, X.D., HUNTER, L.A., LAUGHLIN, C.A., MCLAUGHLIN, S.K., MUZYCZKA, N., ROCCHI, M., and BERNS, K.I. (1990). Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211-2215.
20. KOTIN, R.M., MENNINGER, J.C., WARD, D.C., and BERNS, K.I. (1991). Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter. Genomics 10, 831-834.
21. KOTIN, R.M., LINDEN, R.M., and BERNS, K.I. (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.
22. LUSBY, E.W., FIFE, K.H., and BERNS, K.I. (1980). Nucleotide sequence of the inverted terminal repetition in adeno-associated virus DNA. J. Virol. 34, 402-409.
23. MCLAUGHLIN, S.K., COLLIS, P., HERMONAT, P., and MUZYCZKA, N. (1988). Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 62, 1963-1973.
24. MORGAN, R.A., and ANDERSON, W.F. (1993). Human gene therapy. Annu. Rev. Biochem. 62, 191-217.
25. MURO-CACHO, C.A., SAMULSKI, R.J., and KAPLAN, D. (1992). Gene transfer in human lymphocytes using a vector based on adeno-associated virus. J. Immunother. 11, 231-235.
26. MUZYCZKA, N. (1992). Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158, 98-117.
27. 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.
28. NAHREINI, P., and SRIVASTAVA, A. (1992). Rescue of the adeno-associated virus 2 genome correlates with alterations in DNA-modifying enzymes in human cells. Intervirology 33, 109-115.
29. NAHREINI, P., LARSEN, S.H., and SRIVASTAVA, A. (1992). Cloning and integration of DNA fragments in human cells via the inverted terminal repeats of the adeno-associated virus 2 genome. Gene 119, 265-272.
30. 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.
31. PONNAZHAGAN, S., NALLARI, M., and SRIVASTAVA, A. (1994). Suppression of human α-globin gene expression mediated by the recombinant adeno-associated virus 2-based antisense vectors. J. Exp. Med. 179, 733-738.
32. SAMBROOK, R.J., FRITSCH, E.F., and MANIATIS, T. (1989). Molecular Cloning: A Laboratory Manual. (Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY).
33. SAMULSKI, R.J. (1993). Adeno-associated virus: Integration at a specific chromosomal locus. Curr. Opin. Genet. Dev. 3, 74-80.
34. SAMULSKI, R.J., SRIVASTAVA, S., BERNS, K.I., and MUZYCZKA, N. (1983). Rescue of adeno-associated virus from recombinant plasmids: Gene correction within the terminal repeats of AAV. Cell 33, 135-143.
35. SAMULSKI, R.J., CHANG, L.-S., and SHENK, T. (1989). Helper-free stocks of adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63, 3822-3828.
36. SAMULSKI, R.J., ZHU, X., XIAO, X., BROOK, J.D., HOUSEMAN, D.E., EPSTEIN, N., and HUNTER, L.A. (1991). Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J. 10, 3941-3950.
37. - mutant and correction of terminal palindrome deletions. J. Mol. Biol. 179, 1-20.
38. SNYDER, R.O., IM, D.-S., and MUZYCZKA, N. (1990a). In vitro resolution of covalently joined AAV chromosome ends. Cell 60, 105-113.
39. SNYDER, R.O., IM, D.-S., and MUZYCZKA, N. (1990b). Evidence for covalent attachment of the adeno-associated virus rep protein to the ends of the AAV genome. J. Virol. 64, 6204-6213.
40. SOUTHERN, E.M. (1975). Detection of specific sequences among DNA fragments separated by gel electrophoresis. J. Mol. Biol. 98, 503-517.
41. SRIVASTAVA, A. (1987). Replication of the adeno-associated virus DNA termini in vitro. Intervirology 27, 138-147.
42. SRIVASTAVA, A. (1994). Parvovirus-based vectors for human gene therapy. Blood Cells 20, 531-538.
43. SRIVASTAVA, A., LUSBY, E.W., and BERNS, K.I. (1983). Nucleotide sequence and organization of the adeno-associated virus 2 genome. J. Virol. 45, 555-562.
44. SRIVASTAVA, C.H., SAMULSKI, R.J., LU, L., LARSEN, S.H., and SRIVASTAVA, A. (1989). Construction of a recombinant human parvovirus B19: Adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proc. Natl. Acad. Sci. USA 86, 8078-8082.
45. WALSH, C.E., LIU, J.M., XIAO, X., YOUNG, N.S., NIENHUIS, A.W., and SAMULSKI, R.J. (1992). Regulated high level expression of a human γ-globin introduced into erythroid cells by an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 89, 7257-7261.
46. WALZ, C., and SCHLEHOFER, J.G. (1992). Modification of some biological properties of HeLa cells containing adeno-associated virus DNA integrated into chromosome 17. J. Virol. 66, 2990-3002.
47. WEITZMAN, M.D., KYOSITO, S.R.M., KOTIN, R.M., and OWENS, R.A. (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.
48. XIAO, X., DEVLAMINCK, W., and MONAHAN, J. (1993). Adeno-associated virus (AAV) vectors for gene transfer. Adv. Drug Del. Rev. 12, 201-215.
49. ZHOU, S.Z., BROXMEYER, H.E., COOPER, S., HARRINGTON, M.A., and SRIVASTAVA, A. (1993). Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp. Hematol. 20, 928-933.
50. ZHOU, S.Z., COOPER, S., KANG, L.Y., RUGGIERI, L., HEIMFELD, S., SRIVASTAVA, A., and BROXMEYER, H.E. (1994). Adeno-associated virus 2-mediated high-efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J. Exp. Med. 179, 1867-1875.