Adeno-associated viral vectors for gene transfer and gene therapy

Biological Chemistry

Volumn 380, Issue 6, 1999, Pages 613-622

  Büeler, Hansruedi ^a  

^a UNIVERSITY OF ZURICH   (Switzerland)

Author keywords

Gene therapy; Gene transfer; Recombinant adeno associated virus; Retargeting; Transcriptional regulation

Indexed keywords

VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HERPES VIRUS; HUMAN; IMMUNE RESPONSE; NONHUMAN; PRIORITY JOURNAL; REVIEW; TOXICITY; VIRUS GENE; VIRUS RECOMBINANT; VIRUS TITRATION;

ANIMALS; BRAIN; DEPENDOVIRUS; DNA REPLICATION; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HELPER VIRUSES; HUMANS; RECOMBINATION, GENETIC;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; HERPES; HERPESVIRIDAE; PARVOVIRUS;

EID: 0141636324     PISSN: 14316730     EISSN: None     Source Type: Journal    
DOI: 10.1515/BC.1999.078     Document Type: Review

References (103)

1. Allen, J.M., Debelak, D.J., Reynolds, T.C., and Miller, A.D. (1997). Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomologous recombination during AAV vector production. J. Virol. 71, 6816-6822.
2. Atchison, R.W., Casto, B.C., and Hammond, W.M. (1965). Adenovirus-associated defective virus particles. Science 149, 754-756.
3. Bartlett J.S., Samulski, R.J., and McCown, T.J. (1998). Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum. Gene Ther. 9, 1181-1186.
4. Bartlett, J.S., Kleinschmidt, J., Boucher, R.C., and Samulski, R.J. (1999). Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody. Nature Biotechnol. 17, 181-186.
5. Berns, K.I., and Linden, M. (1995). The cryptic life cycle of adeno-associated virus. Bio Essays 17, 237-245.
6. 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. Virology 68, 556-560.
7. Bohl, D., Salvetti, A., Mouiller, P., and Heard, J.M. (1998). Control of erythropoietin delivery by doxycycline in mice after intra-muscular injection of adeno-associated vector. Blood 92, 1512-1517.
8. Buller, P.M., Janik, J.E., Sebring, E.D., and Rose, J.A. (1981). Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J. Virol. 40, 241-247.
9. Carter, B.J., Marcus-Sekura, C.J., Laughlin, C.A., and Ketner, G. (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.
10. Chang, L.S., Shi, Y., and Shenk, T. (1989). Adeno-associated virus P5 promoter contains an adenovirus E1A-inducible element and a binding site for the major late transcription factor. J. Virol. 63, 3479-3488.
11. Clark, K.R., Voulgaropoulou, F., Fraley, D.M., and Johnson, P.R. (1995). Cell lines for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6, 1329-1341.
12. Daly, T.M., Vogler, C., Levy, B., Haskins, M.E., and Sands, M.S. (1999). Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc. Natl. Acad. Sci. USA 96, 2296-2300.
13. Ding, L., Lu, S., and Munshi, N.C. (1997). In vitro packaging of an infectious recombinant adeno-associated virus 2. Gene Ther. 4, 1167-1172.
14. Duan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K.J., and Engelhardt, J.F. (1998). Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J. Virol. 72, 8568-8577. (Published erratum appears in J. Virol. 73, 861).
15. During, M.J., Samulski, R.J., Elsworth, J.D., Kaplitt, M.G., Leone, P., Xiao, X., Li, J., Freese, A., Taylor, J.R., Roth, R.H., Sladek, J.R. Jr., O'Malley, K.L., and Redmond, D.E. Jr. (1998a). In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther. 5, 820-827.
16. During, M.J., Xu, R., Young, D., Kaplitt, M.G., Sherwin, R.S., and Leone, P. (1998b). Peroral gene therapy of lactose intolerance using an adeno-associated virus vector. Nat. Med. 4, 1131-1135.
17. Fan, D.S., Ogawa, M., Fujimoto, K.I., Ikeguchi, K., Ogasawara, Y., Urabe, M., Nishizawa, M., Nakano, I., Yoshida, M., Nagatsu, I., Ichinose, H., Nagatsu, T., Kurtzman, G.J., and Ozawa, K. (1998). Behavioral recovery in 6-hydroxydopamine lesioned rats by cotransduction of striatum with tyrosine hydroxylase and aromatic L-amino acid decarboxylase genes using two separate adeno-associated virus vectors. Hum. Gene Ther. 9, 2527-2535.
18. 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. J. Virol. 70, 3227-3234.
19. Fisher, K.J., Gao, G.P., Weitzman, M.D., DeMatteo, R., Burda, J.F., and Wilson, J.M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532.
20. Fisher, K.J., Jooss, K., Alston, J., Yang, Y., Haecker, S.E., High, K., Pathak, R., Raper, S.E., and Wilson, J.M. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 3, 306-312.
21. Flannery, J.G., Zolotukhin, S., Vaquero, M.I., LaVail, M.M., Muzyczka, N., and Hauswirth, W.W. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA 94, 6916-6921.
22. Flotte, T.R., Barraza-Ortiz, X., Solow, R., Afione, S.A., Carter, B.J., and Guggino, W.B. (1995). An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 2, 29-37.
23. Gao, G.P., Qu, G., Faust, L.Z, Engdahl, R.K., Xiao, W., Hughes, J.V., Zoltick, P.W., and Wilson, J.M. (1998). High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum. Gene Ther. 9, 2353-2362.
24. Greelish, J.P., Su, L.T., Lankford, E.B., Burkman, J.M., Chen, H., Konig, S.K., Mercier, I.M., Desjardins, P.R., Mitchell, M.A., Zheng, X.G., Leferovich, J., Gao, G.P., Balice-Gordon, R.J., Wilson, J.M., and Stedman, H.H. (1999). Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat. Med. 5, 439-443.
25. Grimm, D., Kern, A., Rittner, K., and Kleinschmidt, J.A. (1998). Novel tools for production and purification of recombinant adeno-associated virus vectors. Hum. Gene Ther. 9, 2745-2760.
26. Haberman, R.P., McCown, T.J., and Samulski R.J. (1998). Inducible long-term gene expression in brain with adeno-associated virus gene transfer. Gene Ther. 5, 1604-1611.
27. 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, 329-339.
28. Herzog, R.W., Hagstrom, J.N., Kung, S.H., Tai, S.J., Wilson, J.M., Fisher, K.J., and High, K.A. (1997). Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA 94, 5804-5809.
29. Herzog, R.W., Yang, E.Y., Couto, L.B., Hagstrom, J.N., Elwell, D., Fields, P.A., Burton, M., Bellinger, D.A., Read, M.S., Brinkhous, K.M., Podsakoff, G.M., Nichols, T.C., Kurtzman, G.J., and High, K.A. (1999). Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med. 5, 56-63.
30. Huang, M.M., and Hearing, P. (1989). Adenovirus early region 4 encodes two gene products with redundant effects in lytic infection. J. Virol. 63, 2605-2615.
31. Im, D.-S., and Muzyczka, N. (1989). Factors that bind to the AAV terminal repeats. J. Virol. 63, 3095-3104.
32. 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.
33. Inoue, N., and Russell, D.W. (1998). Packaging cells based on inducible gene amplification for the production of adeno-associated virus vectors. J. Virol. 72, 7024-7031.
34. Janik, J.E., Huston, M.M., Cho, K., and Rose, J.A. (1989). Efficient synthesis of adeno-associated virus structural proteins requires both adenovirus DNA binding protein and VA I RNA. Virology 168, 320-329.
35. Jay, F.T., Laughlin, C.A., and Carter, B.J. (1981). Eukaryotic translational control: adeno-associated virus protein synthesis is affected by a mutation in the adenovirus DNA-binding protein. Proc. Natl. Acad. Sci. USA 78, 2927-2931.
36. Jomary, C., Vincent, K.A., Grist, J., Neal, M.J., and Jones, S.E. (1997). Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther. 4, 683-690.
37. Jooss, K., Yang, Y., Fisher, K.J., and Wilson, J.M. (1998). Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J. Virol. 72, 4212-4223.
38. Kaplitt, M.G., Leone, P., Samulski, R.J., Xiao, X., Pfaff, D.W., O'Malley, K.L., and During, M.J. (1994). Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8, 148-154.
39. Kessler, P.D., Podsakoff, G.M., Chen, X., McQuiston, S.A., Colosi, P.C., Matelis, L.A., Kurtzman, G.J., and Byrne, B.J. (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.
40. Klein, R.L., Meyer, E.M., Peel, A.L., Zolotukhin, S., Meyers, C., Muzyczka, N., and King, M.A. (1998). Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Exp. Neurol. 150, 183-194.
41. Koeberl, D.D., Alexander, I.E., Halbert, C.L., Russell, D.W., and Miller, A.D. (1997). Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 94, 1426-1431.
42. Kotin, R.M., Siniscalco, M., Samulski, R.J., Zhu, X.D., Hunter, L., Laughlin, C.A., McLaughlin, S., Muzyczka, N., Rocchi, M., and Berns, K.I. (1990). Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211-2215.
43. Kyostio, S.R., Owens, R.A., Weitzman, M.D., Antoni, B.A., Chejanovsky, N., and Carter, B.J. (1994). Analysis of adeno-associated virus (AAV) wild-type and mutant Rep proteins for their abilities to negatively regulate AAV p5 and p19 mRNA levels. J. Virol. 68, 2947-2957.
44. Laughlin, C.A., Jones, N., and Carter, B.J. (1982). Effect of deletions in adenovirus early region 1 genes upon replication of adeno-associated virus. J. Virol. 41, 868-876.
45. Lewin, A.S., Drenser, K.A., Hauswirth, W.W., Nishikawa, S., Yasumura, D., Flannery, J.G., and LaVail, M.M. (1998). Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nature Med. 4, 967-971. (Published erratum appears in Nat. Med. 4, 1081)
46. Lewis, B.A., Tullis, G., Seto, E., Horikoshi, N., Weinmann, R., and Shenk, T. (1995). Adenovirus E1A proteins interact with the cellular YY1 transcription factor. J. Virol. 69, 1628-1636.
47. Li, J., Samulski, R.J., and Xiao, X. (1997). Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71, 5236-5243.
48. Li, J., Dressman, D., Tsao, Y.P., Sakamoto, A., Hoffman, E.P., and Xiao, X. (1999a). rAAV vector-mediated sarcoglycan gene transfer in a hamster model for limb girdle muscular dystrophy. Gene Ther. 6, 74-82.
49. Li, X., Eastman, E.M., Schwartz, R.J., and Draghia-Akli, R. (1999b). Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences. Nature Biotechnol. 17, 241-245.
50. Linden, R.M., Winocour, E., and Berns, K.I. (1996). The recombination signals for adeno-associated virus site-specific integration. Proc. Natl. Acad. Sci. USA 93, 7966-7972.
51. Mah, C., Qing, K., Khuntirat, B., Ponnazhagan, S., Wang, X.S., Kube ,D.M., Yoder, M.C., and Srivastava, A. (1998). Adeno-associated virus type2-mediated gene transfer: role of epidermal growth factor receptor protein tyrosine kinase in transgene expression. J. Virol. 72, 9835-9843.
52. Mandel, R.J., Spratt, S.K., Snyder, R.O., and Leff, S.E. (1997). Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc. Natl. Acad. Sci. USA 94, 14083-14088.
53. Matsushita, T., Elliger, S., Elliger, C., Podsakoff, G., Villarreal, L., Kurtzman, G.J., Iwaki, Y., and Colosi, P. (1998). Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5, 938-945.
54. McCarty, D.M., Christensen, M., and Muzyczka, N. (1991). Sequences required for coordinate induction of adeno-associated virus p19 and p40 promoters by Rep protein. J. Virol. 65, 2936-2945
55. McCarty, D.M., Pereira, D.J., Zolotukhin, I., Zhou, X., Ryan, J.H., and Muzyczka, N. (1994). Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein. J. Virol. 68, 4988-4997.
56. McCown, T.J., Xiao, X., Li, J., Breese, G.R., and Samulski, R.J. (1996). Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain. Res. 713, 99-107.
57. Murphy, J.E., Zhou, S., Giese, K., Williams, L.T., Escobedo, J.A., Dwarki, V.J. (1998). Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin. Proc. Natl. Acad. Sci. USA 94, 13921-13926.
58. 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, 1128-1138.
59. Ni, T.H., McDonald, W.F., Zolotukhin, I., Melendy, T., Waga, S., Stillman, B., and Muzyczka, N. (1998). Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection. J. Virol. 72, 2777-2787.
60. Ogasawara, Y., Urabe, M., and Ozawa, K. (1998). The use of heterologous promoters for adeno-associated virus (AAV) protein expression in AAV vector production. Microbiol. Immunol. 42, 177-185.
61. Palombo, F., Monciotti, A., Recchia, A., Cortese, R., Ciliberto, G., and La Monica, N. (1999). Site-specific integration in mammalian cells mediated by a new hybrid baculovirus-adeno-associated virus vector. J. Virol. 72, 5025-5034.
62. Pereira, D.J., McCarty, D.M., and Muzyczka, N. (1997). The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection. J. Virol. 71, 1079-1088.
63. Ponnazhagan, S., Erikson, D., Kearns, W.G., Zhou, S.Z., Nahreini, P., Wang, X.S., and Srivastava, A. (1997). Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum. Gene Ther. 8, 275-284.
64. Qing, K., Khuntirat, B., Mah, C., Kube, D.M., Wang, X.S., Ponnazhagan, S., Zhou, S., Dwarki, V.J., Yoder, M.C., and Srivastava, A. (1998). Adeno-associated virus type 2-mediated gene transfer: correlation of tyrosine phosphorylation of the cellular single-stranded D sequence-binding protein with transgene expression in human cells in vitro and murine tissues in vivo. J. Virol. 72, 1593-1599.
65. Qing, K., Mah, C., Hansen, J., Zhou, S., Dwarki, V., and Srivastava, A. (1999). Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2. Nature Med. 5, 71-77.
66. Recchia, A., Parks, R.J., Lamartina, S., Toniatti, C., Pieroni, L., Palombo, F., Ciliberto, G., Graham, F.L., Cortese, R., La Monica, N., and Colloca, S. (1999). Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 96, 2615-2620.
67. Rendahl, K.G., Leff, S.E., Otten, G.R., Spratt, S.K., Bohl, D., Van Roey, M., Donahue, B.A, Cohen, L.K., Mandel, R.J., Danos, O., and Snyder, R.O. (1998). Regulation of gene expression in vivo following transduction by two separate rAAV vectors. Nature Biotechnol. 16, 757-761.
68. Richardson, W.D., and Westphal, H. (1981). A cascade of adenovirus early functions is required for expression of adeno-associated virus. Cell 27, 133-141.
69. Richardson, W.D., and Westphal, H. (1984). Requirement for either early region 1a or early region 1b adenovirus gene products in the helper effect for adeno-associated virus. J. Virol. 51, 404-410.
70. Rizzuto, G., Gorgoni, B., Cappelletti, M., Lazzaro, D., Gloaguen, I., Poli, V., Sgura, A., Cimini, D., Ciliberto, G., Cortese, R., Fattori, E., and La Monica, N. (1999). Development of animal models for adeno-associated virus site-specific integration. J. Virol. 73, 2517-2526.
71. Salvetti, A., Oreve, S., Chadeuf, G., Favre, D., Cherel, Y., Champion-Arnaud, P., David-Ameline, J., and Moullier, P. (1998). Factors influencing recombinant adeno-associated virus production. Hum. Gene Ther. 9, 695-706.
72. Samulski, R.J., and Shenk, T. (1988). Adenovirus E1B 55-Mr polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs. J. Virol. 62, 206-210.
73. Samulski, R.J., Chang, L.S., and Shenk, T. (1989). Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J. Virol. 63, 3822-3828.
74. Samulski, R.J., Zhu, X., Xiao, X., Brook, J.D., Housman, 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. (Published erratum appears in EMBO J. 11, 1228).
75. Sanlioglu, S., Duan, D., and Engelhardt, J.F. (1999). Two independent molecular pathways for recombinant adeno-associated virus genome conversion occur after UV-C and E4orf6 augmentation of transduction. Hum. Gene Ther. 10, 591-602.
76. Schimmenti, S., Boesen, J., Claassen, E.A., Valerio, D., and Einerhand, M.P. (1998). Long-term genetic modification of rhesus monkey hematopoietic cells following transplantation of adenoassociated virus vector-transduced CD34+ cells. Hum. Gene Ther. 9, 2727-2734.
77. Shi, Y., Seto, E., Chang, L.S., and Shenk, T. (1991). Transcriptional repression by YY1, a human GLI-Kruppel-related protein, and relief of repression by adenovirus E1A protein. Cell 18, 377-388.
78. Smith, R.H., and Kotin, R.M. (1998). The Rep52 gene product of adeno-associated virus is a DNA helicase with 3′-to-5′ polarity. J. Virol. 72, 4874-4881.
79. Snyder, R.O., Miao, C.H., Patijn, G.A., Spratt, S.K., Danos, O., Nagy, D., Gown, A.M., Winther, B., Meuse, L., Cohen, L.K., Thompson, A.R., and Kay, M.A. (1997). Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-276.
80. Snyder, R.O., Miao, C., Meuse, L., Tubb, J., Donahue, B.A., Lin, H.F., Stafford, DW., Patel, S., Thompson, A.R, Nichols, T., Read, M.S., Bellinger, D.A., Brinkhous, K.M., and Kay, M.A. (1999). Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nature Med. 5, 64-70.
81. Song S., Morgan, M., Ellis, T., Poirier, A., Chesnut, K., Wang, J., Brantly, M., Muzyczka, N., Byrne, B.J., Atkinson, M., and Flotte, T.R. (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.
82. Srivastava, A., Lusby, E.W., and Berns, K.I. (1983). Nucleotide sequence and organization of the adeno-associated virus 2 genome. J. Virol. 45, 555-564.
83. Summerford, C., and Samulski, R.J. (1998). Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72, 1438-1445.
84. Summerford, C., Bartlett, J.S., and Samulski, R.J. (1999). AlphaV-beta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat. Med. 5, 78-82.
85. Surosky, R.T., Urabe, M., Godwin, S.G., McQuiston, S.A., Kurtzman, G.J., Ozawa, K., and Natsoulis, G. (1997). Adeno-associated virus Rep proteins target DNA sequences to a unique locus in the human genome. J. Virol. 71, 7951-7959.
86. Szczypka, M.S., Mandel, R.J., Donahue, B.A., Snyder, R.O., Left, S.E., and Palmiter, R.D. (1999). Viral gene delivery selectively restores feeding and prevents lethality of dopamine-deficient mice. Neuron 22, 167-178.
87. Tamayose, K., Hirai, Y., 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. Hum. Gene Ther. 7, 507-513.
88. Teramoto, S., Bartlett, J.S., McCarty, D., Xiao, X., Samulski, R.J., and Boucher, R.C. (1998). Factors influencing adeno-associated virus-mediated gene transfer to human cystic fibrosis airway epithelial cells: comparison with adenovirus vectors. J. Virol 72, 8904-8912.
89. Vincent K.A., Piraino, S.T., and Wadsworth, S.C. (1997). Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. J. Virol. 71, 1897-1905.
90. Vincent-Lacaze, N., Snyder, R.O., Gluzman, R., Bohl, D., Lagarde, C., and Danos, O. (1999). Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle. J. Virol. 73, 1949-1955.
91. Walker, S.L., Wonderling, R.S., and Owens, R.A. (1997). Mutational analysis of the adeno-associated virus type 2 Rep68 protein helicase motifs. J. Virol. 71, 6996-7004.
92. Wang, X.S., Khuntirat, B., Qing, K., Ponnazhagan, S., Kube, D.M., Zhou, S., Dwarki, V.J., and Srivastava, A. (1998). Characterization of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination. J. Virol. 72, 5472-5480.
93. Wang, L., Takabe, K., Bidlingmaier, S.M., III C.R., Verma, I.M. (1999). Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl. Acad. Sci. USA 96, 3906-3910.
94. 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, 5808-5812.
95. West, M.H., Trempe, J.P., Tratschin, J.D., and Carter, B.J. (1987). Gene expression in adeno-associated virus vectors: the effects of chimeric mRNA structure, helper virus, and adenovirus VA1 RNA. Virology 160, 38-47.
96. 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, 8098-8108.
97. Xiao X., Li J., and Samulski, R.J. (1998a). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.
98. Xiao, W., Berta, S.C., Lu, M.M., Moscioni, A.D., Tazelaar, J., Wilson, J.M. (1998b). Adeno-associated virus as a vector for liver-directed gene therapy. J Virol 72, 10222-10226.
99. Yakinoglu, 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, 3123-3129.
100. Yang, Q., Mamounas, M., Yu G., Kennedy, S., Leaker, B., Merson, J., Wong-Staal, F., Yu, M., and Barber, J.R. (1998). Development of novel cell surface CD34-targeted recombinant adeno-associated virus vectors for gene therapy. Hum. Gene Ther. 9, 1929-1937.
101. Ye, X., Rivera, V.M., Zoltick, P., Cerasoli, F. Jr., Schnell, M.A., Gao, G., Hughes, J.V., Gilman, M., and Wilson, J.M. (1999). Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science 283, 88-91.
102. Zhou, X., and Muzyczka, N. (1998). In vitro packaging of adeno-associated virus DNA. J. Virol. 72, 3241-3247.
103. Zhou, S., Murphy, J.E., Escobedo, J.A., and Dwarki, V.J. (1998). Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. Gene Ther. 5, 665-670.