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AAV as a viral vector for human gene therapy

Generation of recombinant virus

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Abstract

Investigation of the adeno-associated virus (AAV) life cycle has enabled the establishment of methodology and identification of critical cis-acting sequences required for recombinant AAV production. Vectors derived from the defective human parvovirus (AAV) have been used for successful gene transfer and expression in many diverse mammalian cell types, such as erythroid, airway epithelium, and neuronal cells. One of the crucial steps in the continued case of AAV as a vector is the development of packaging systems that will allow efficient encapsidation of foreign genes into AAV virions. For this reason, the focus of this article will be generation of recombinant AAV vectors.

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References

  1. Roemer, K. and Friedmann, T. (1992) Concepts and strategies for human gene therapy.Eur. J. Biochem. 208, 211–225.

    Article  PubMed  CAS  Google Scholar 

  2. Ostrove, J. M., Leonard, J., Weck, K. E., Rabson, A. B., and Gendelman, H. E. (1987) Activation of the human immunodeficiency virus by herpes simplex virus type 1.J. Virol. 61, 3726–3732.

    PubMed  CAS  Google Scholar 

  3. Atchison, R. W., Casto, B. C., and Hammon, W. Md. (1965) Adenovirus-associated defective virus particles.Science 149, 754–756.

    Article  PubMed  CAS  Google Scholar 

  4. McPherson, R. A., Rosenthal, L. J., and Rose, J. A. (1985) Human cytomegalovirus completely helps adeno-associated virus replication.Virology 147, 217–222.

    Article  PubMed  CAS  Google Scholar 

  5. Samulski, R. J. (1993) Adeno-associated virus: integration at a specific chromosomal locus.Curr. Opin. Genet. Devel. 3, 74–80.

    Article  CAS  Google Scholar 

  6. 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.

    PubMed  CAS  Google Scholar 

  7. 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.

    Article  PubMed  CAS  Google Scholar 

  8. Berns, K.I. (1990) Parvovirus replication.Microbiol. Rev. 54, 316–329.

    PubMed  CAS  Google Scholar 

  9. Cukor, G., Blacklow, N. R., Hoggan, D., and Berns, K. I. (1984) Biology of adeno-associated virus, inThe Parvoviruses (Berns, K. I., ed.), Plenum, New York, pp. 33–66.

    Google Scholar 

  10. Samulski, R. J., Bern, 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.

    Article  PubMed  CAS  Google Scholar 

  11. 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, 1963–1973.

    PubMed  CAS  Google Scholar 

  12. 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.

    PubMed  CAS  Google Scholar 

  13. Hunter, L. A. and Samulski, R. J. (1992) Colocalization of adeno-associated virus Rep and capsid proteins in the nuclei of infected cells.J. Virol. 66, 317–324.

    PubMed  CAS  Google Scholar 

  14. Carter, B. J., Laughlin, C. A., de la Maza, L. M., and Myers, M. (1979) Adeno-associated virus auto-interference.Virology 92, 449–462.

    Article  PubMed  CAS  Google Scholar 

  15. Tratschin, J.-D., West, M. H. P., Sandbank, T., and Carter, B. J. (1984) A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase.Mol. Cell. Biol. 4, 2072–2081.

    PubMed  CAS  Google Scholar 

  16. Graham, F. L. and Prevec, L. (1991) Manipulation of adenovirus vectors, inMethods in Molecular Biology: Gene Transfer and Expression Protocols, vol. 7, (Murray, E. J., ed.), Humana, Clifton, NJ, pp. 109–128.

    Chapter  Google Scholar 

  17. Graham, F. L., Smiley, J., Russell, W. C., and Nairn, R. (1977) Characteristics of a human cell line transformed by DNA from human adenovirus type 5.J. Gen. Virol. 36, 59–72.

    Article  PubMed  CAS  Google Scholar 

  18. Alwine, J. C. (1985) Transient gene expression control: effects of transfected DNA stability and transactivation by viral early proteins.Mol. Cell. Biol. 5, 1034–1042.

    PubMed  CAS  Google Scholar 

  19. Hirt, B. (1967) Selective extraction of polyoma DNA from infected mouse cell cultures.J. Mol. Biol. 26, 365–369.

    Article  PubMed  CAS  Google Scholar 

  20. Sanes, J. R., Rubenstein, J. L., and Nicolas, J. F. (1986) Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos.EMBO J. 5, 3133–3142.

    PubMed  CAS  Google Scholar 

  21. Maniatis, T., Fritsh, E. F., and Sambrook, J. (1982)Molecular Cloning: A Laboratory Manual, Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY.

    Google Scholar 

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Rolling, F., Samulski, R.J. AAV as a viral vector for human gene therapy. Mol Biotechnol 3, 9–15 (1995). https://doi.org/10.1007/BF02821330

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