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Human gene targeting by viral vectors

Nature Genetics volume 18, pages 325–330 (1998)Cite this article

Abstract

Stable transduction of mammalian cells typically involves random integration of viral vectors by non-homologous recombination. Here we report that vectors based on adeno-associated virus (AAV) can efficiently modify homologous human chromosomal target sequences. Both integrated neomycin phosphotransferase genes and the hypoxanthine phosphoribosyltransferase gene were targeted by AAV vectors. Site-specific genetic modifications could be introduced into approximately 1% of cells, with the highest targeting rates occurring in normal human fibroblasts. These results suggest that AAV vectors could be used to introduce specific genetic changes into the genomic DNA of a wide variety of mammalian cells, including therapeutic gene targeting applications.

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Authors and Affiliations

  1. Markey Molecular Medicine Center and Division of Hematology, Department of Medicine, University of Washington School of Medicine, Seattle, Washington, 98195-7720, USA

    David W. Russell & Roll K. Hirata

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  1. David W. Russell
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  2. Roll K. Hirata
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Russell, D., Hirata, R. Human gene targeting by viral vectors. Nat Genet 18, 325–330 (1998). https://doi.org/10.1038/ng0498-325

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