Adeno-Associated Viruses

Methods in Molecular Biology

Volumn 737, Issue , 2011, Pages 211-234

  Mezzina, Mauro ^b   Merten, Otto Wilhelm ^a  

^a GÉNÉTHON   (France)

^b NONE

Author keywords

Dot blot; ELISA; Gene therapy; Gene transfer; PEI; Purification; rAAV vectors; Triple transfection method; Ultracentrifugation

Indexed keywords

ADENO ASSOCIATED VIRUS; ANIMAL; ARTICLE; CELL LINE; DNA PROBE; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; GENETICS; GROWTH, DEVELOPMENT AND AGING; HELPER VIRUS; HUMAN; ISOLATION AND PURIFICATION; METHODOLOGY; SYNTHESIS; TITRIMETRY; ULTRACENTRIFUGATION; VIRION; VIRUS GENE;

ANIMALS; CELL LINE; DEPENDOVIRUS; DNA PROBES; GENE THERAPY; GENES, VIRAL; GENETIC VECTORS; HELPER VIRUSES; HUMANS; TITRIMETRY; TRANSDUCTION, GENETIC; TRANSFECTION; ULTRACENTRIFUGATION; VIRION;

EID: 80052340259     PISSN: 10643745     EISSN: 19406029     Source Type: Book Series    
DOI: 10.1007/978-1-61779-095-9_9     Document Type: Chapter

References (66)

1. Berns, K. I. (1996) Parvoviridae: The virus and their replication. In: Fields BN, Knipe DM, Howley PM (eds). Fields in Virology. Lippincott – Raven, Philadelphia, pp. 2173–2197.
2. Rutledge, E.A., Halbert, C. L., and Russell, D. W. (1998) Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J. Virol. 72, 309–319.
3. Gao, G. P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., and Wilson, J. M. (2002) Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. USA 99, 11854–11859.
4. Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., and Wilson, J. M. (2004) Clades of adeno-associated viruses are widely disseminated in human tissues. J. Virol. 78, 6381–6388.
5. Mori, S., Wang, L., Takeuchi, T., and Kanda, T. (2004) Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein. Virology 330, 375–383.
6. Gao, G., Vandenberghe, L. H., and Wilson J. M. (2005) New recombinant serotypes of AAV vectors. Curr. Gene Ther. 5, 285–297.
7. Qiu, J. and Pintel, D. J. (2004) Alternative polyadenylation of adeno-associated virus type 5 RNA within an internal intron is governed by the distance between the promoter and the intron and is inhibited by U1 small nuclear RNP binding to the intervening donor. J. Biol. Chem. 279, 14889–14898.
8. Erles, K., Sebokova, P., and Schlehofer, J. R. (1999) Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J. Med. Virol. 59, 406–411.
9. Tobiasch, E., Burguete, T., Klein-Bauernschmitt, P., Heilbronn, R., and Schlehofer, J. R. (1998) Discrimination between different types of human adeno-associated viruses in clinical samples by PCR. J. Virol. Methods 71, 17–25.
10. Kotin, R. M., Siniscalco, M., Samulski, R. J., Zhu, X. D., Hunter, L., Laughlin, C. A., et al. (1990) Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211–2215.
11. Samulski, R. J., Zhu, X., Xiao, X., Brook, J. D., Housman, D. E., Epstein, N., et al. (1991) Targeted integration of adeno-associated virus (AAV) into human chromosome 19. Embo J. 10, 3941–3950.
12. Shelling, S. N. and Smith, M. G. (1994) Targeted integration of transfected and infected adeno-associated virus vectors containing the neomycin gene. Gene Ther. 1, 165–169.
13. Samulski, R. J., Berns, 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.
14. Samulski, R. J., Chang, L. S., and Shenk, T. (1987) A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61, 3096–3101.
15. 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, 3251–3260.
16. Grimm, D. and Kay, M. A. (2003) From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr. Gene Ther. 3, 281–304.
17. Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., et al. (2008) In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J. Virol. 82, 5887–5911.
18. 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 adenoassociated virus replication function. J. Virol. 51, 611–619.
19. 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.
20. 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. U S A 91, 5808–5812.
21. Horer, M., Weger, S., Butz, K., Hoppe-Seyler, F., Geisen, C., and Kleinschmidt, J. A. (1995) Mutational analysis of adeno-associated virus Rep protein-mediated inhibition of heterologous and homologous promoters. J. Virol. 69, 5485–5496.
22. 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.
23. King, J. A., Dubielzig, R., Grimm, D., and Kleinschmidt, J. A. (2001) DNA helicasemediated packaging of adeno-associated virus type 2 genomes into preformed capsids. Embo J. 20, 3282–3291.
24. Timpe, J., Bevington, J., Casper, J., Dignam, J. D., and Trempe, J. P. (2005) Mechanisms of adeno-associated virus genome encapsidation. Curr. Gene Ther. 5, 273–284.
25. Schlehofer, J. R., Ehrbar, M., zur Hausen, H. (1986) Vaccinia virus, herpes simplex virus, and carcinogens induce DNA amplification in a human cell line and support replication of a helpervirus dependent parvovirus. Virology 152, 110–117.
26. Weindler, F. W. and Heilbronn, R. (1991) A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication. J. Virol. 65, 2476–2483.
27. McPherson, R. A., Rosenthal, L. J., and Rose, J. A. (1985) Human cytomegalovirus completely helps adeno-associated virus replication. Virology 147, 217–222.
28. Ogsten, P., Raj, K., and Beard, P. (2000) Productive replication of adeno-associated virus can occur in human papillomavirus type 16 (HPV-16) episome-containing keratinocytes and is augmented by the HPV-16 E2 protein. J. Virol. 74, 3494–3504.
29. Geoffroy, M.-C. and Salvetti, A. (2005) Helper functions required for wild type and recombinant adeno-associated virus growth. Curr. Gene Ther. 5, 265–271.
30. Wistuba, A., Kern, A., Weger, S., Grimm, D., and Kleinschmidt, J. A. (1997) Subcellular compartmentalization of adeno-associated virus type 2 assembly. J. Virol. 71, 1341–1352.
31. Grimm, D., Kern, A., Rittner, K., and Kleinschmidt, J. A. (1998) Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum. Gene Ther. 9, 2745–2760.
32. Xiao, X., Li, J., and Samulski, R.J. (1998) Production of high-titer recombinant adenoassociated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224–2323.
33. Chiorini, J. A., Yang, L., Liu, Y., Safer, B., and Kotin, R. M. (1997) Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J. Virol. 71, 6823–6833.
34. Chiorini, J. A., Kim, F., Yang, L., and Kotin, R. M. (1999) Cloning and characterization of adeno-associated virus type 5. J. Virol. 73, 1309–1319.
35. Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., and Wilson, J. M. (1999) Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73, 3994–4003.
36. Qu, G., McClelland, A., and Wright, J. F. (2000) Scaling-up production of recombinant AAV vectors for clinical applications. Curr. Opin. Drug Discov. Devel. 3, 750–755.
37. Brown, P., Barrett, S., Godwin, S., Trudinger, M., Marschak, T., Norboe, D., et al. (1998) Optimization of production of adeno-associated virus (AAV) for use in gene therapy, Presented at: Cell Culture Engineering VI, San Diego/CA.
38. Clark, K. R. (2002) Recent advances in recombinant adeno-associated virus vector production. Kidney Int. 61, 9–15.
39. Merten, O.-W., Gény, C., and Douar A. M. (2005) Current issues in adeno-associated viral vectors production. Gene Ther. 12, S51–S61.
40. Urabe, M., Ding, C., and Kotin, R. M. (2002) Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum. Gene Ther. 13, 1935–1943.
41. Urabe, M., Ozawa, K., Haast, S. J. P., and Hermens, W. T. J. M. C. (2007) Improved AAV vectors produced in insect cells. WO 2007/046703 A2.
42. Chen, H. (2008) Intron splicing-mediated expression of AAV Rep and Cap genes and production of AAV vectors in insect cells. Mol. Ther. 16, 924–930.
43. Smith, R. H., Levy, J. R., and Kotin, R. M. (2009) A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol. Ther. 17, 1888–1896.
44. Aslanidi, G., Lamb, K., and Zolotukhin, S. (2009) An inducible system for highly efficient production of recombinant adeno-associated virus (rAAV) vectors in insect Sf9 cells. Proc. Natl. Acad. Sci. U S A. 106, 5059–5064.
45. Cecchini, S., Virag, T., Negrete, A., and Kotin, R. M. (2009) Production and processing of rAAV-U7smOPT in 100 L bioreactors for canine models of Duchenne muscular dystrophy. Mol. Ther. 17, S1
46. Mena, Y. A., Aucoin, M. G., Chahal, P. S., and Kamen, A. A. (2008) Improvement of adenoassociated vector titers in high density insect cell cultures by combined feeding and asynchronous infection. Poster P12 presented at the ATGQ (Association de thérapie génique du Québec) Meeting, 26–27 May 2008, Montreal/Quebec.
47. Snyder, R. O., Xiao, X., and Samulski, R. J. (1996) Production of recombinant adenoassociated viral vectors. In: N. Dracopoli JH, B. Krof, D. Moir, C. Morton, C. Seidman, J. Seidman, and D. Smith (ed). Current Protocols in Human Genetics. John Wiley and Sons Publisher: New York, pp. 12.11.11–24.
48. Zolotukhin, S., Byrne, B. J., Mason, E., Zolotukhin, I., Potter, M., Chesnut, K., et al. (1999) Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973–985.
49. Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J. Jr., et al. (2002) Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28, 158–167.
50. Brument, N., Morenweiser, R., Blouin, V., Toublanc, E., Raimbaud, I., Chérel, Y., et al. (2002) A versatile and scalable two-step ionexchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and-5. Mol. Ther. 6, 678–686.
51. Kaludov, N., Handelman, B., and Chiorini, J. A. (2002) Scalable purification of adenoassociated virus type 2, 4, or 5 using ionexchange chromatography. Hum. Gene Ther. 13, 1235–1243.
52. Blouin, V., Brument, N., Toublanc, E., Raimbaud, I., Moullier, P., and Salvetti, A. (2004) Improving rAAV production and purification: towards the definition of a scalable process. J. Gen. Med. 6, S223–S228.
53. Burova, E. and Ioffe, E. (2005) Chromatographic purification of recombinant adenoviral and adeno-associated viral vectors: methods and implications. Gene Ther. 12, S5-S17.
54. Duffy, A. M., O’Doherty, A. M., O’Brian, T., and Strappe, P. M. (2005) Purification of adenovirus and adeno-associated virus: comparison of novel membrane-based technology to conventional techniques. Gene Ther. 12, S62–S72.
55. Drittanti, L., Jenny, C., Poulard, K., Samba, A., Manceau, P., Soria, N., et al. (2001) Optimised helper virus-free production of high-quality adeno-associated virus vectors. J. Gene Med. 3, 59–71.
56. Farson, D., Harding, T. C., Tao, L., Liu, J., Powell, S., Vimal, V., et al. (2004) Development and characterization of a cell line for large-scale, serum-free production of recombinant adeno-associated viral vectors. J. Gen. Med. 6, 1369–1381.
57. Salvetti, A., Oreve, S., Chadeuf, G., Favre, D., Cherel, Y., Champion-Arnaud, P., et al. (1998) Factors influencing recombinant adeno-associated virus production. Hum.Gene Ther. 9, 695–706.
58. Coecke, S., Balls, M., Bowe, G., Davis, J., Gstraunthaler, G., Hartung, T., et al. (2005) Guidance on Good Cell Culture Practice. A report of the second ECVAM task force on Good Cell Culture Practice. ATLA 33, 261–287.
59. Rohr, U. P., Wulf, M. A., Stahn, S., Steidl, U., Haas, R., and Kronenwett, R. (2002) Fast and reliable titration of recombinant adeno-associated virus type-2 using quantitative real-time PCR. J. Virol. Methods 106, 81–88.
60. Rohr, U. P., Heyd, F., Neukirchen, J., Wulf, M. A., Queitsch, I., Kroener-Lux, G., et al. (2005) Quantitative real-time PCR for titration of infectious recombinant AAV-2 particles. J. Virol. Methods 127, 40–45.
61. Matsushita, T., Elliger, S., Elliger, C., Podsakoff, G., Villarreal, L., Kurtzman, G.J., et al. (1998) Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther 5, 938–945.
62. Grimm, D., Kern, A., Pawlita, M., Ferrari, F., Samulski, R., Kleinschmidt, J. (1999) Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2. Gene Ther 6, 1322–1330.
63. Collaco, R.F., Cao, X., and Trempe, J.P. (1999) A helper virus-free packaging system for recombinant adeno-associated virus vectors. Gene 238, 397–405.
64. Jenny, C., Toublanc, E., Danos, O., and Merten, O.-W. (2005) Serum-free production of rAAV-2 using HeLa derived producer cells. Cytotechnology 49, 11–23.
65. Gao, G.P., Lu, F., Sanmiguel, J.C., Tran, P.T., Abbas, Z., Lynd, K.S., et al. (2002) Rep/Cap gene amplification and high-yield production of AAV in an A549 cell line expressing Rep/ Cap. Mol Ther 5, 644–649.
66. Farson, D., Harding, T.C., Tao, L., Liu, J., Powell, S., Vimal, V., et al. (2004) Development and characterization of a cell line for largescale, serum-free production of recombinant adeno-associated viral vectors. J Gene Med. 6, 1369–1381.