In vivo model of adeno-associated virus vector persistence and rescue

Journal of Virology

Volumn 70, Issue 5, 1996, Pages 3235-3241

  Afione, Sandra A ^a   Conrad, Carol K ^a   Kearns, William G ^a   Chunduru, Suryaprabha ^a   Adams, Robert ^a   Reynolds, Thomas C ^b   Guggino, William B ^a   Cutting, Garry R ^a   Carter, Barrie J ^b   Flotte, Terence R ^a,c  

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

^b Targeted Genetics Corporation   (United States)

^c JOHNS HOPKINS HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; GENE THERAPY; LOWER RESPIRATORY TRACT; NONHUMAN; PRIORITY JOURNAL; RHESUS MONKEY; VIRUS RECOMBINANT;

EID: 9244234931     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.70.5.3235-3241.1996     Document Type: Article

References (26)

1. Brough, D. E., S. A. Rice, S. Sell, and D. F. Klessig. 1985. Restricted changes in the adenovirus DNA-binding protein that lead to extended host range or temperature-sensitive phenotypes. J. Virol. 55:206-212.
2. 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
3. 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.
4. Crystal, R. G., N. G. McElvaney, M. A. Rosenfeld, C.-S. Chu, A. A. Mastrangeli, G. H. Jogn, S. L. Brody, H. A. Jaffe, N. T. Eissa, and C. Danel. 1994. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis Nat. Genet. 8:42-51
5. Engelhardt, J. F., R. H. Simon, Y. Yang, M. Zepeda, S. Weber-Pendelton, B. Doranz, M. Grossman, and J. M. Wilson. 1993 Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: biological efficacy study. Hum. Gene Ther. 4:759-769.
6. Flotte, T. R., S. A. Afione, C. K. 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 conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90:10613-10617.
7. 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.
8. S. Flotte, T. R., S. A. Afione, and P. L. Zeitlin. 1994. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am. J. Respir Cell. Mol. Biol. 11:517-521.
9. Flotte, T. R., X. Barrazza-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. 2:39-47.
10. Ginsberg, H. E., U. Lundholm-Beauchamp, R. L. Hoswood, B. Pernis, W. S. M. Wold, R. M. Chanock, and G. A. Prince. 1989. Role of early region 3 (E3) in pathogenesis of adenovirus disease. Proc. Natl. Acad. Sci. USA 86:3823-3827.
11. Giraud, C., E. Winocour, and K. I. Berns. 1994. Site-specific integration by adeno-associated virus is directed by a cellular DNA sequence. Proc Natl. Acad Sci. USA 91:10039-10043.
12. Kearns, W. G., S. Afione, S. B. Fulmer, J. Caruso, P. Pearson, T. Flotte, and G. R. Cutting. 1994. Recombinant AAV vectors demonstrate episomal expression in immortalized human bronchial epithelial cells. (Abstract.; Presented at the 1994 American Society of Human Genetics Meeting.
13. Kotin, R. M., R. M. Linden, and K. I. Berns. 1992 Characterization of a preferred site on chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J. 11:5071-5076
14. 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 10:831-534.
15. 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 87:2210-2215.
16. McLaughlin, S. K., P. Collis, P. L. Hermonat, and N. Muzyezka. 1988. Adeno-associated virus general transduction vectors: analysis of proviral structures. J. Virol. 62:1963-1973.
17. Rosenfeld, M. A., W. Seigfried, K. Yoshimura, K. Yoneyama, M. Fukayama, L. E. Stier, P. K. Paakko, P. Gilardi, L. D. Stratford-Perricaudet, M. Perricaudet, S. Jallat, A. Pavirani, J.-P. Lecocq, and R. G. Crystal. 1991 Adenovirus-mediated transfer a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 252:431-434.
18. Rosenfeld, M. A., K. Yoshimura, B. C. Trapnell, K. Yoneyama, E. R. Rasenthal, W. Dalemans, M. Fukayama, J. Bargon, L. E. Stier, L. Stratford-Perricaudet, M. Perricaudet, W. B. Guggino, J.-P. Lecoeq, and R. G. Crystal. 1992 In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68:143-155.
19. 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 91:8915-5919.
20. Samulski, R. J. 1993. Adeno-associated virus: integration at a specific chromosomal locus. Curr. Opin. Biotechnol. 3:74-80.
21. Samulski, R. J., L.-S. Chang, and R. Shenk. 1989. Helper-tree stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J. Virol. 63:3822-3828.
22. 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.
23. Simon, R. H., J. F. Engelhardt, Y. Yang, M. Zepeda, S. Weber-Pendleton, M. Grossman, and J. M. Wilson. 1993. Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: toxicity study. Hum Gene Ther. 4:771-780.
24. 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.
25. Weitzmann, M. D., S. R. M. Kyöstiö, 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.
26. Zabner, L., L. A. Couture, R. J. Gregory, S. M. Graham, A. E. Smith, and M. J. Welsh. 1993. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75:207-216.