Transposon-based mutagenesis generates diverse adeno-associated viral libraries with novel gene delivery properties

Methods in Molecular Biology

Volumn 434, Issue , 2008, Pages 161-170

  Koerber, James T ^a   Schaffer, David V ^a  

^a University of California

Author keywords

Adeno associated virus; Gene therapy; Mutagenesis; Protein engineering; Transposase

Indexed keywords

ADENO-ASSOCIATED VIRUS;

EID: 84934444639     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-60327-248-3_10     Document Type: Article

References (29)

1. Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., et al. (2003) AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-72.
2. Kay, M. A., Manno, C. S., Ragni, M. V., Larson, P. J., Couto, L. B., McClelland, A., et al. (2000) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24, 257-61.
3. Fields, B. N., Knipe, D. M., Howley, P. M., and Griffin, D. E. (2001) Fields Virology, Lippincott Williams & Wilkins, Philadelphia.
4. Srivastava, A., Lusby, E. W., and Berns, K. I. (1983) Nucleotide sequence and organization of the adeno-associated virus 2 genome. J Virol 45, 555-64.
5. Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., et al. (1993) Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 90, 10613-7.
6. Kaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O'Malley, K. L., et al. (1994) Long-term gene expression and phenotypic correction using adenoassociated virus vectors in the mammalian brain. Nat Genet 8, 148-54.
7. Kaspar, B. K., Llado, J., Sherkat, N., Rothstein, J. D., and Gage, F. H. (2003) Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301, 839-42.
8. Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., et al. (2004) Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol 78, 6381-8.
9. Chiorini, J. A., Kim, F., Yang, L., and Kotin, R. M. (1999) Cloning and characterization of adeno-associated virus type 5. J Virol 73, 1309-19.
10. Wu, P., Xiao, W., Conlon, T., Hughes, J., Agbandje-McKenna, M., Ferkol, T., et al. (2000) Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol 74, 8635-47.
11. Rabinowitz, J. E., Xiao, W., and Samulski, R. J. (1999) Insertional mutagenesis of AAV2 capsid and the production of recombinant virus. Virology 265, 274-85.
12. Shi, W., Arnold, G. S., and Bartlett, J. S. (2001) Insertional mutagenesis of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors. Hum Gene Ther 12, 1697-711.
13. Lochrie, M. A., Tatsuno, G. P., Christie, B., McDonnell, J. W., Zhou, S., Surosky, R., et al. (2006) Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol 80, 821-34.
14. Grifman, M., Trepel, M., Speece, P., Gilbert, L. B., Arap, W., Pasqualini, R., et al. (2001) Incorporation of tumor-targeting peptides into recombinant adenoassociated virus capsids. Mol Ther 3, 964-75.
15. Girod, A., Ried, M., Wobus, C., Lahm, H., Leike, K., Kleinschmidt, J., et al. (1999) Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med 5, 1052-6.
16. Xie, Q., Bu, W., Bhatia, S., Hare, J., Somasundaram, T., Azzi, A., et al. (2002) The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc Natl Acad Sci USA 99, 10405-10.
17. Perabo, L., Buning, H., Kofler, D. M., Ried, M. U., Girod, A., Wendtner, C. M., et al. (2003) In vitro selection of viral vectors with modified tropism: the adenoassociated virus display. Mol Ther 8, 151-7.
18. Muller, O. J., Kaul, F., Weitzman, M. D., Pasqualini, R., Arap, W., Kleinschmidt, J. A., et al. (2003) Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 21, 1040-6.
19. Maheshri, N., Koerber, J. T., Kaspar, B. K., and Schaffer, D. V. (2006) Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 24, 198-204.
20. Guntas, G., and Ostermeier, M. (2004) Creation of an allosteric enzyme by domain insertion. J Mol Biol 336, 263-73.
21. Murakami, H., Hohsaka, T., and Sisido, M. (2002) Random insertion and deletion of arbitrary number of bases for codon-based random mutation of DNAs. Nat Biotechnol 20, 76-81.
22. Kazazian, H. H., Jr. (2004) Mobile elements: drivers of genome evolution. Science 303, 1626-32.
23. Brune, W., Menard, C., Hobom, U., Odenbreit, S., Messerle, M., and Koszinowski, U. H. (1999) Rapid identification of essential and nonessential herpesvirus genes by direct transposon mutagenesis. Nat Biotechnol 17, 360-4.
24. Hobom, U., Brune, W., Messerle, M., Hahn, G., and Koszinowski, U. H. (2000) Fast screening procedures for random transposon libraries of cloned herpesvirus genomes: mutational analysis of human cytomegalovirus envelope glycoprotein genes. J Virol 74, 7720-9.
25. Vilen, H., Aalto, J. M., Kassinen, A., Paulin, L., and Savilahti, H. (2003) A direct transposon insertion tool for modification and functional analysis of viral genomes. J Virol 11, 123-34.
26. Yu, J. H., and Schaffer, D. V. (2006) Selection of novel vesicular stomatitis virus glycoprotein variants from a peptide insertion library for enhanced purification of retroviral and lentiviral vectors. J Virol 80, 3285-92.
27. Bâtard, P., Jordan, M., and Wurm, F. (2001) Transfer of high copy number plasmid into mammalian cells by calcium phosphate transfection. Gene 270, 61-8.
28. Rabinowitz, J. E., Bowles, D. E., Faust, S. M., Ledford, J. G., Cunningham, S. E., and Samulski, R. J. (2004) Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol 78, 4421-32.
29. Gigout, L., Rebollo, P., Clement, N., Warrington, K. H., Jr., Muzyczka, N., Linden, R. M., et al. (2005) Altering AAV tropism with mosaic viral capsids. Mol Ther 11, 856-65.