Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line

Journal of Virology

Volumn 71, Issue 3, 1997, Pages 1776-1783

  Malik, Punam ^a   McQuiston, Susan A ^b   Yu, Xiao Jin ^a   Pepper, Karen A ^a   Krall, Wanda J ^a   Podsakoff, Gregory M ^b   Kurtzman, Gary J ^b   Kohn, Donald B ^a,c  

^a UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^b Avigen Inc   (United States)

^c Division of Research Immunology/Bone Marrow Transplantation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; NERVE GROWTH FACTOR RECEPTOR; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ARTICLE; CONTROLLED STUDY; GENE TRANSFER; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; MOLONEY LEUKEMIA ONCOVIRUS; PRIORITY JOURNAL; VIRUS INFECTION; VIRUS RECOMBINANT;

EID: 0031058475     PISSN: 0022538X     EISSN: None     Source Type: Journal    
DOI: 10.1128/jvi.71.3.1776-1783.1997     Document Type: Article

References (43)

1. Alexander, I. E., D. W. Russell, and D. Miller. 1994. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J. Virol. 68:8282-8287.
2. Antoni, B. A., A. B. Rabson, I. L. Miller, J. P. Trempe, N. Chejanovsky, and B. J. Carter. 1991. Adeno-associated virus Rep protein inhibits human immunodeficiency virus type 1 production in human cells. J. Virol. 65:396-404.
3. Berg, M. M., D. W. Sternberg, B. L. Hempstead, and M. V. Chao. 1991. The low-affinity p75 nerve growth factor (NGF) receptor mediates NGF-induced tyrosine phosphorylation. Proc. Natl. Acad. Sci. USA 88:7106-7110.
4. Berns, K. I., and R. A. Bohenzky. 1987. Adeno-associated viruses: an update. Adv. Virus Res. 32:243-307.
5. Bertran, J., J. L. Miller, Y. Yang, A. Fenimore-Justman, F. Rueda, E. F. Vanin, and A. W. Neinhuis. 1996. Recombinant adeno-associated virus-mediated high efficiency transient expression of the murine cationic amino acid transporter (ecotropic retroviral receptor) permits stable transduction of human HeLa cells by ecotropic retroviral vectors. J. Virol. 70:6759-6766.
6. Challita, P., and D. Kohn. 1994. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc. Natl. Acad. Sci. USA 91:2567-2571.
7. Chatterjee, S., P. R. Johnson, and K. K. Wong, Jr. 1992. Dual-target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 258:1485-1488.
8. Einerhand, M. P., M. Antoniou, S. Zolotukhin, N. Muzyczka, K. I. Berns, F. Grosveld, and D. Valerio. 1995. Regulated high-level human beta-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer. Gene Ther. 2:336-343.
9. Ferrari, F. K., T. Samulski, T. Shenk, and R. J. Samulski. 1996. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70:3227-3234.
10. 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.
11. 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.
12. Goodman, S., X. Xiao, R. E. Donahue, A. Moulton, J. Miller, C. Walsh, N. S. Young, R. J. Samulski, and A. W. Nienhuis. 1994. Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 84:1492-1500. (Erratum, 85:862, 1995.)
13. Halbert, C. L., I. E. Alexander, G. M. Wolgamot, and A. D. Miller. 1995. Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J. Virol. 69:1473-1479.
14. Kessler, P. D., G. M. Podsakoff, X. Chen, S. A. McQuiston, P. C. Colosi, L. A. Matelis, G. J. Kurtzman, and B. J. Byrne. 1996. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. USA 93:14082-14087.
15. Kotin, R. M. 1994. Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther. 5:793-801.
16. Kotin, R. M., M. Siniscalco, R. J. Samulski, X. Zhu, L. Hunter, K. A. Laughlin, S. K. 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.
17. Lebkowski, J. S., M. M. McNally, T. B. Okarma, and L. B. Lerch. 1988. Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types. Mol. Cell. Biol. 8:3988-3996.
18. McLachlin, J. R., N. Mittereder, M. B. Daucher, M. Kadan, and M. A. Eglitis. 1993. Factors affecting retroviral vector function and structural integrity. Virology 195:1-5.
19. Mclaughlin, S. K., P. Collis, P. L. Hermonat, and N. Muzyczka. 1988. Adeno-associated virus general transduction vectors: analysis of proviral structures. J. Virol. 62:1963-1973.
20. Miller, A. D., and C. Buttimore. 1986. Redesign of retrovirus packaging lines to avoid recombination leading to helper virus production. Mol. Cell. Biol. 6:2895-2902.
21. Miller, A. D., J. V. Garcia, N. V. Suhr, C. M. Lynch, C. Wilson, and M. V. Eiden. 1991. Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J. Virol. 65:2220-2224.
22. Miller, D., and G. Rosman. 1989. Improved retroviral vectors for gene transfer and expression. BioTechniques 7:980-986.
23. Miller, J. L., R. E. Donahue, S. E. Sellers, R. J. Samulski, N. S. Young, and A. W. Nienhuis. 1993. Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells. Proc. Natl. Acad. Sci. USA 91:10183-10187.
24. Miller, J. L., C. E. Walsh, P. A. Ney, R. J. Samulski, and A. W. Nienhuis. 1993. Single-copy transduction and expressions of human gamma-globin in K562 erythroleukemia cells using recombinant adeno-associated virus vectors: the effect of mutations in NF-E2 and GATA-1 binding motifs within the hypersensitivity site 2 enhancer. Blood 82:1900-1906.
25. Muzyczka, N. 1992. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158:97-129.
26. Natsoulis, G., R. Suroski, S. Godwin, S. McQuiston, and G. Kurtzman. 1995. A System for specifically targeting genes into chromosome 19. Blood 86(Suppl. 1):236a.
27. Nolta, J. A., X. Yu, I. Bahner, and D. Kohn. 1992. Retroviral mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow. J. Clin. Invest 90:343-348.
28. Philip, R., E. Brunette, L. Kilinski, D. Murugesh, M. A. McNally, K. Ucar, J. Rosenblatt, T. B. Okarma, and J. S. Lebkowski. 1994. Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes. Mol. Cell. Biol. 14:2411-2418.
29. Podsakoff, G., K. K. Wong, Jr., and S. Chatterjee. 1994. Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors. J. Virol. 68:5656-5666.
30. Ponnazhagan, S., M. L. Nallari, and A. Srivastava. 1994. Suppression of human alpha-globin gene expression mediated by the recombinant adeno-associated virus 2-based antisense vectors. J. Exp. Med. 179:733-738.
31. Radeke, M. J., T. P. Misko, C. Hsu, L. A. Herzenberg, and E. M. Shooter. 1987. Gene transfer and molecular cloning of the rat nerve growth factor receptor. Nature 325:593-597.
32. 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 91:8915-8919.
33. Samulski, R. J., L. Chang, and T. Shenk. 1989. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J. Virol. 63:3822-3828.
34. 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.
35. 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.
36. Sun, X., B. R. Murphy, Q. Li, S. Gullapalli, J. Mackins, H. N. Jayaram, A. Srivastava, and A. C. Antony. 1995. Transduction of folate receptor cDNA into cervical carcinoma cells using recombinant adeno-associated virions delays cell proliferation in vitro and in vivo. J. Clin. Invest. 96:1535-1547.
37. Thrasher, A. J., M. de Alwis, C. M. Casimir, C. Kinnon, K. Page, J. S. Lebkowski, A. W. Segal, and R. J. Levinsky. 1995. Functional reconstitution of the NADPH-oxidase by adeno-associated virus gene transfer. Blood 86:761-765.
38. 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.
39. Walsh, C. E., J. M. Liu, X. Xiao, N. S. Young, A. W. Nienhuis, and R. J. Samulski. 1992. Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 89:7257-7261.
40. Walsh, C. E., A. W. Nienhuis, R. J. Samulski, M. G. Brown, J. L. Miller, N. S. Young, and J. M. Liu. 1994. Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J. Clin. Invest. 94:1440-1448.
41. Welcher, A. A., C. M. Bitler, M. J. Radeke, and E. M. Shooter. 1991. Nerve growth factor binding domain of the nerve growth factor receptor. Proc. Natl. Acad. Sci. USA 88:159-163.
42. Zhou, S. Z., H. E. Broxmeyer, S. Cooper, M. A. Harrington, and A. Srivastava. 1993. Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp. Hematol. 21:928-933.
43. Zhou, S. Z., S. Cooper, L. Y. Kang, L. Ruggieri, S. Heimfeld, A. Srivastava, and H. E. Broxmeyer. 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.