Adeno-Associated Viral Vectors

Concepts in Gene Therapy

Volumn , Issue , 2020, Pages 61-78

  McCarty, D M ^a   Samulski, Richard Jude ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 2642684884     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1515/9783110808636-005     Document Type: Chapter

References (71)

1. Laughlin, C. A., Tratschin, J.-D., Coon, H., and Carter, B. J. (1983). Cloning of infectious adenoassociated virus genomes in bacterial plasmids. Gene, 23, 65-73.
2. Samulski, R. J., Bems, K. I., Tan, M., and Muzyczka, N. (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.
3. Hermonat, P. L., Labow, M. A., Wright, R., Berns, K. I., and Muzyczka, N. (1984). Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants. J Virol, 51, 329-333.
4. Tratschin, J.-D., Miller, I. L., and Carter, B. J. (1984). Genetic analysis of adeno-associated virus: properties of deletion mutants constructed in vitro and evidence for an adeno-associated virus replication function. J Virol, 51, 611-619.
5. Tratschin, J.-D., Tal, J., and Carter, B. J. (1986). Negative and positive regulation in trans of gene expression from adeno-associated virus vectors in mammalian cells by a viral Rep gene product. Mol Cell Biol, 6, 2884-2894.
6. Labow, M. A., Hermonat, R L., and Berns, K. I. (1986). Positive and negative autoregulation or the adeno-associated virus type 2 genome. J Virol, 60, 251-258.
7. Samulski, R. J., Chang, L.-S., and Shenk, T. (1989). Helper-free stocks of recombinant adenoassociated viruses: normal integration does not require viral gene expression. J Virol, 63, 3822-3828.
8. Muzyczka, N. (1992). Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol, 158, 97-129.
9. Holscher, C., et al. (1994). Cell lines inducibly expressing the adeno-associated virus (AAV) rep gene: requirements for productive replication of rep-negative AAV mutants. J Virol, 68(11), 7169-77.
10. Trempe, J. P. and Yang, Q. (1993). Characterization of a cell line that expresses the AAV replication proteins (abstract). In: Fifth Parvovirus Workshop. Crystal River, FL.
11. Clark, K. R., Voulgaropoulou, F., Fraley, D. M., and Johnson, P. R. (1995). Cell lines for the production of recombinant adeno-associated virus. Human Gene Therapy, 6(10), 1329-41.
12. Tamayose, T., Yukihiko, H., and Shimada, T. (1996). A New Strategy for Large-Scale Preparation of High-Titer Recombinant Adeno-Associated Virus Vectors by Using Packaging Cell Lines and Sulfonated Cellulose Column Chromatography. Human Gene Therapy, 7, 507-513.
13. Flotte, T. R. et al. (1995). An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Therapy, 2(1), 29-37.
14. Mamounas, M., Leavitt, M., Yu, M., and Wong-Staal, F. (1995). Increased titer of recombinant AAV vectors by gene transfer with adenovirus coupled to DNA-polylysine complexes. Gene Therapy, 2(6), 429-32.
15. Snyder, R. O., Xiao, X., and Samulski, R. J. (1996). Production of recombinant adeno-associated virus vectors. In: Current protocols in Human Genetics, N. Dracopoli et al. (eds.). John Wiley and Sons Ltd.: New York, 12.1.1-12.2.23.
16. Yakobson, B., Koch, T., and Winocour, E. (1987). Replication of adeno-associated virus in synchronized cells without the addition of a helper virus. J Virol, 61(4), 972-81.
17. McLaughlin, S. K., Collis, P., Hermonat, P. L., and Muzyczka, N. (1988). Adeno-associated virus general transduction vectors: analysis of proviral structures. J Virol, 62(6), 1963-73.
18. de la Maza, L. M. and Carter, B. J. (1980). Heavy and light particles of adeno-associated virus. J Virol, 33, 1129-1137.
19. Rose, J. A. and Koczot, F. J. (1972). Adenovirus-associated virus multiplication: VII. Helper requirement for viral deoxyribonucleic acid and ribonucleic acid synthesis. J Virol, 10(1), 1-8.
20. Ferrari, F. K., Samulski, T., Shenk, T., and Samulski, R. J. (1996). Second-Strand Synthesis is a Rate Limiting Step for Efficient Transduction by Recombinant Adeno-Associated Virus Vectors. Journal of Virology, 70(5).
21. Fisher, K. J. et al. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. Journal of Virology, 70(1), 520-32.
22. Ohman, K., Nordqvist, K., and Akusjarvi, G. (1993). Two adenovirus proteins with redundant activities in virus growth facilitates tripartite leader mRNA accumulation. Virology, 194(1), 50-8.
23. Weiden, M. D. and Ginsberg, H. S. (1994). Deletion of the E4 region of the genome produces adenovirus DNA concatemers. Proceedings of the National Academy of Sciences of the United States of America, 91(1), 153-7.
24. McCarty, D. M., Christensen, M., and Muzyczka, N. (1991). Sequences required for the coordinate induction of the AAV pl9 and p40 promoters by the Rep protein. J Virol, 65, 2936-2945.
25. Beaton, A., Palumbo, P., and Berns, K. I. (1989). Expression from the adeno-associated virus p5 and p19 promoters is negatively regulated in trans by the rep protein. J Virol, 63, 4450-4454.
26. Dobner, T., Horikoshi, N., Rubenwolf, S., and Shenk, T. (1996). Blockage by Adenovirus E4orf6 of Transcriptional Activation by the p53 Tumor Suppressor. Science, 272, 1470-1473.
27. Nordqvist, K., Ohman, K., and Akusjarvi, G. (1994). Human adenovirus encodes two proteins which have opposite effects on accumulation of alternatively spliced mRNAs. Molecular and Cellular Biology, 14(1), 437-45.
28. Samulski, R. J. and Shenk, T. (1988). Adenovirus E1B 55-M, polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs. J Virol, 62, 206-210.
29. Sarnow, P. et al. (1984). Adenovirus early region IB 58, 000-dalton tumor antigen is physically associated with an early region 4 25, 000-dalton protein in productively infected cells. Journal of Virology, 49(3), 692-700.
30. Bridge, E., Medghalchi, S., Ubol, S., Leesong, M., and Ketner, G. (1993). Adenovirus early region 4 and viral DNA synthesis. Virology, 193(2), 794-801.
31. Russell, D. W., Alexander, I. A., and Miller, A. D. (1995). DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA, 92, 5719-5723.
32. Yalkinoglu, A. O., Heilbronn, R., Burkle, A., Schlehofer, J. R., and zur-Hausen, H. (1988). DNA amplification of adeno-associated virus as a response to cellular genotoxic stress. Cancer Res, 48 (11), 3123-9.
33. Yakobson, B., Hrynko, T. A., Peak, M. J., and Winocour, E. (1989). Replication of adeno-associated virus in cells irradiated with UV light at 254 nm. J Virol, 63(3), 1023-30.
34. Yalkinoglu, A. O., Zentgraf, H., and Hubscher, U. (1991). Origin of adeno-associated virus DNA replication is a target of carcinogen-inducible DNA amplification. Journal of Virology, 65(6), 3175-84.
35. Russell, D. W., Miller, A. D., and Alexander, I. E. (1994). Adeno-associated virus vectors preferentially transduce cells in S phase. Proc Natl Acad Sci USA, 91(19), 8915-9.
36. Halbert, C. L., Alexander, I. E., Wolgamot, G. M., and Miller, A. D. (1995). Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J Virol, 69(3), 1473-9.
37. McCown, T. J., Xiao, X., Li, J., Breeze, G. R., and Samulski, R. J. (1996). Differential and Persistent Expression Patterns of CNS Gene Transfer by an Adeno-Associeated Virus (AAV) Vector. Brain Research, 713, 99-107.
38. Xiao, X., Li, J., and Samulski, R. J. (1996). Efficient Long-Term Gene Transfer into Muscle Tissue of Immunocompetent Mice by Adeno-Associated Virus Vector. J. Virol., 70(11), 8098-8108.
39. Bertran, J. et al. (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 Retrovirual Vectors. J. Virol., 70(10), 6759-6766.
40. Flotte, T. R., Afione, S. A., and Zeitlin, P. L. (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(5), 517-21.
41. Goodman, S. et al. (1994). Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood, 84(5), 1492-500.
42. Keams, W. G. et al. (1996). Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Therapy, 3, 748-755.
43. Walsh, C. E. et al. (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(15), 7257-61.
44. Weitzman, M. D., Kyostio, S. R., 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(13), 5808-12.
45. 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(13), 5071-8.
46. McCarty, D. M. et al. (1994). Identification of linear DNA sequences that specifically bind the adenoassociated virus Rep protein. J Virol, 68(8), 4988-97.
47. Snyder, R. O. et al. (1993). Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein. J Virol, 67(10), 6096-104.
48. Muzyczka, N. (1991). In vitro replication of adeno-associated virus DNA. Seminars in VIROLOGY, 2, 281-290.
49. Lusby, E., 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.
50. Hauswirth, W. W. and Berns, K. I. (1979). Adeno-associated virus DNA replication. Virology, 93, 57-68.
51. Hauswirth, W. W. and Berns, K. I. (1977). Origin and termination of adeno-associated virus DNA replication. Virology, 78, 488-499.
52. Straus, S. E., Sebring, E. D., and Rose, J. A. (1976). Concatemers of alternating plus and minus strands are intermediates in adenovirus-associated virus DNA synthesis. Proceedings of the National Academy of Sciences USA, 73, 742-746.
53. Ward, P. and Berns, K. I. (1995). Minimum origin requirements for linear duplex AAV DNA replication in vitro. Virology, 209(2), 692-5.
54. Ni, T. H., Zhou, X., McCarty, D. M., Zolotukhin, I., and Muzyczka, N. (1994). In vitro replication of adeno-associated virus DNA. J Virol, 68(2), 1128-38.
55. Koczot, F. J., Carter, B. J., Garon, C. F., and Rose, I. A. (1973). Self-complementarity of terminal sequences within plus or minus strands of adenovirus-associated virus DNA. Proceedings of the National Academy of Sciences of the United States of America, 70(1), 215-9.
56. Berns, K. I. and Kelly, T. J. J. (1974). Visualization of the inverted terminal repetition in adenoassociated virus DNA. J Mol Biol, 82, 267-271.
57. Berns, K. I., Pinkerton, T. C., Thomas, G. F., and Hoggan, M. D. (1975). Detection of adenoassociated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells. Virology, 68, 556-560.
58. Laughlin, C. A., Cardellichio, C. B., and Coon, H. C. (1986). Latent infection of KB cells with adeno-associated virus type 2. J Virol, 60(2), 515-24.
59. Handa, H., Shiroki, K., and Shimojo, H. (1977). Establishment and characterization of KB cell lines latently infected with adeno-associated virus type 1. Virology, 82, 84-92.
60. Hermonat, P. L. and Muzyczka, N. (1984). Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA, 81, 6466-6470.
61. Tratschin, J. D., Miller, I. L., Smith, M. G., and Carter, B. J. (1985). Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells. Mol Cell Biol, 5(11), 3251-60.
62. Urcelay E., Ward, P., Wiener, S. M., Safer, B., and Kotin, R. M. (1995). Asymmetric replication in vitro from a human sequence element is dependent on adeno-associated virus Rep protein. Journal of Virology, 69(4), 2038-46.
63. 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(21), 10039-43.
64. Kotin, R. M. and Berns, K. I. (1989). Organization of adeno-associated virus DNA in latently infected Detroit 6 cells. Virology, 170, 460-467.
65. Linden, R. M., Winocour, E., and Berns, K. I. (1996). The recombination signals for adenoassociated virus site-specific integration. P.N.A.S, 93, 7966-7972.
66. Prasad, K. M. and Trempe, J. P. (1995). The adeno-associated virus Rep78 protein is covalently linked to viral DNA in a preformed virion. Virology, 214(2), 360-70.
67. McCarty, D. M., Ryan, J. H., Zolotukhin, S., Zhou, X., and Muzyczka, N. (1994). Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat. J Virol, 68(8), 4998-5006.
68. Kaplitt, M. G. et al. (1994). Long-term gene expression and phenotypic correction using adenoassociated virus vectors in the mammalian brain. Nature Genetics, 8, 148-154.
69. Zolotukhin S., Potter, M., Hauswirth, W. W., Guy, J., and Muzyczka, N. (1996). A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells. Journal of Virology, 70(7), 4646-54.
70. Lalwani, A. K., Walsh, B. J., Reilly, P. G., Muzyczka, N., and Mhatre, A. N. (1996). Development of in vivo gene therapy for hearing disorders: introduction of adeno-associated virus into the cochlea of the guinea pig. Gene Therapy, 3, 588-592.
71. Alexander, I. E., Rüssel, D. W., Spence, A. M., and Miller, A. D. (1996). Effects of Gamma Irradiation on the Transduction of Dividing and Nondividing Cells in Brain and Muscle of Rats by Adeno-Associated Virus Vectors. Human Gene Therapy, 7, 841-850.