Gene therapy vectors based on adeno-associated virus type 1

Journal of Virology

Volumn 73, Issue 5, 1999, Pages 3994-4003

  Xiao, Weidong ^a   Chirmule, Narendra ^a   Berta, Scott C ^a   McCullough, Beth ^a   Gao, Guangping ^a   Wilson, James M ^a,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; ADULT; ARTICLE; GENE SEQUENCE; GENE TARGETING; HUMAN; HUMAN EXPERIMENT; MOLECULAR CLONING; NONHUMAN; NORMAL HUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; SEQUENCE ANALYSIS; VIRUS NEUTRALIZATION;

EID: 0032924185     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.73.5.3994-4003.1999     Document Type: Article

References (33)

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. Bantel-Schaal, U., and H. zur Hausen. 1984. Characterization of the DNA of a defective human parvovirus isolated from a genital site. Virology 134:52-63.
3. Berns, K. I. 1995. Parvoviridae: the viruses and their replication, p. 1007-1041. In B. N. Fields, D. M. Knipe, and P. M. Howley (ed.), Fundamental virology, 3rd ed., vol. 2. Lippincott-Raven Publishers, Philadelphia, Pa.
4. Chlorini, J. A., L. Yang, Y. Liu, B. Safer, and R. M. Kotin. 1997. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J. Virol. 71:6823-6833.
5. Clark, K. R., F. Voulgaropoulou, D. M. Fraley, and P. R. Johnson. 1995. Cell lines for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6:1329-1341.
6. Clark, K. R., F. Voulgaropoulou, and P. R. Johnson. 1996. A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors. Gene Ther. 3:1124-1132.
7. Fisher, K. J., G. P. Gao, M. D. Weitzman, R. DeMatteo, J. F. Burda, and J. M. Wilson. 1996. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70:520-532.
8. Fisher, K. J., K. Jooss, J. Alston, Y. Yang, S. E. Haecker, K. High, R. Pathak, S. E. Raper, and J. M. Wilson. 1997. Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med. 3:306-312.
9. Flotte, T., B. Carter, C. Conrad, W. Guggino, T. Reynolds, B. Rosenstein, G. Taylor, S. Walden, and R. Wetzel. 1996. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 7:1145-1159.
10. Flotte, T. R., and B. J. Carter. 1997. In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. Adv. Pharmacol. 40:85-101.
11. Gao, G., G. Qu, L. Z. Faust, R. K. Engdahl, W. Xiao, J. V. Hughes, P. W. Zoltick, and J. M. Wilson. 1998. High-liter adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum. Gene Ther. 9:2353-2362.
12. Halbert, C. L., T. A. Standaert, C. B. Wilson, and A. D. Miller. 1998. Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure. J. Virol. 72:9795-9805.
13. Kaplitt, M. G., P. Leone, R. J. Samulski, X. Xiao, D. W. Pfaf, 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. Nat. Genet. 8:148-154.
14. Kotin, R. M., M. Siniscalco, R. J. Samulski, 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.
15. Li, J., R. J. Samulski, and X. Xiao. 1997. Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71:5236-5243.
16. Manning, W. C., S. Zhou, M. P. Bland, J. A. Escobedo, and V. Dwarki. 1998. Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors. Hum. Gene Ther. 9:477-485.
17. Muramatsu, S., H. Mizukami, N. S. Young, and K. E. Brown. 1996. Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3. Virology 221:208-217.
18. Murphy, F. A., C. M. Fauquet, M. A. Mayo, A. W. Jarvis, S. A. Ghabrial, M. D. Summers, G. P. Martelli, and D. H. L. Bishop. 1995. Classification and nomenclature of viruses: sixth report of the International Committee on Taxonomy of Viruses. Arch. Virol. 1995:169-175.
19. Muzyczka, N. 1992. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158:97-129.
20. Parks, W. P., D. W. Boucher, J. L. Melnick, L. H. Taber, and M. D. Yow. 1970. Seroepidemiological and ecological studies of the adenovirus-associated satellite viruses. J. Virol. 2:716-722.
21. Rubenstein, R. C., U. McVeigh, T. R. Flotte, W. B. Guggino, and P. L. Zeitlin. 1997. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther. 4:384-392.
22. Rutledge, E. A., C. L. Halbert, and D. W. Russell. 1998. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J. Virol. 72:309-319.
23. Samulski, R. J. 1993. Adeno-associated virus: integration at a specific chromosomal locus. Curr. Opin. Genet. Dev. 3:74-80.
24. 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.
25. Samulski, R. J., A. Srivastava, K. I. Berns, and N. Muzyczka. 1983. Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell 33:135-143.
26. 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.)
27. Snyder, R. O., C. H. Miao, G. A. Patijn, S. K. Spratt, O. Danos, D. Nagy, A. M. Gown, B. Winther, L. Meuse, L. K. Cohen, A. R. Thompson, and M. A. Kay. 1997. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat. Genet. 16:270-276.
28. Song, S., M. Morgan, T. Ellis, A. Poirier, K. Chesnut, J. Wang, M. Brantly, N. Muzyczka, B. J. Byrne, M. Atkinson, and T. R. Flotte. 1998. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 95:14384-14388.
29. 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.
30. Xiao, W., S. C. Berta, M. M. Lu, A. D. Muscioni, J. Tazelaar, and J. M. Wilson. 1998. Adeno-associated virus as a vector for liver-directed gene therapy. J. Virol. 72:10222-10226.
31. Xiao, W., and J. M. Wilson. Unpublished data.
32. Xiao, X., J. Li, and R. J. Samulski. 1996. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70:8098-8108.
33. Xiao, X., J. Li, and R. J. Samulski. 1998. Production of high-liter recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72:2224-2232.