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Viruses in Human Gene Therapy pp 53–76Cite as

Adeno-associated Viral Vectors

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Abstract

Viral vectors, which are capable of infecting virtually every cell in a target population, are the most efficient method for delivering nucleic acids into mammalian cells. Viral expression vectors, reviewed by Rigby (1983) have been developed using DNA viruses, such as papova (i.e., SV40), adeno, herpes, and pox viruses and RNA viruses, such as retrovirus. Recently, the most commonly used model vectors have been derived from murine and avian retroviruses (Mann, Mulligan and Baltimore 1983; Watanabe and Temin 1983; Cone and Mulligan 1984; Sorge et al. 1984). All steps required for use of these replication-defective vectors (infection, replication, integration and gene expression) have been thoroughly studied and characterized, thus providing well developed vector systems which can randomly deposit genes into the host genome. However, certain drawbacks currently restrict the usefulness of this popular system for gene therapy purposes. At present, recombinant retrovirus stock can only be made to low titers. While the retrovirus vectors can infect a broad class of cell types, cell replication and DNA synthesis are required for provirus integration, restricting their efficient use to replicating cells.

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  1. Richard Jude Samulski Ph.D.
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  1. Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, NC, USA

    Jean-Michel H. Vos D.Sc.

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© 1995 Jean-Michel H. Vos

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Samulski, R.J. (1995). Adeno-associated Viral Vectors. In: Vos, JM.H. (eds) Viruses in Human Gene Therapy. Springer, Dordrecht. https://doi.org/10.1007/978-94-011-0555-2_3

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  • DOI: https://doi.org/10.1007/978-94-011-0555-2_3

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