Precise hit: adeno-associated virus in gene targeting

Nat Rev Microbiol. 2005 Nov;3(11):837-47. doi: 10.1038/nrmicro1266.


Vectors based on the adeno-associated virus (AAV) have attracted much attention as potent gene-delivery vehicles, mainly because of the persistence of this non-pathogenic virus in the host cell and its sustainable therapeutic gene expression. However, virus infection can be accompanied by potentially mutagenic random vector integration into the genome. A novel approach to AAV-mediated gene therapy based on gene targeting through homologous recombination allows efficient, high-fidelity, non-mutagenic gene repair in a host cell.

Publication types

  • Review

MeSH terms

  • Animals
  • DNA Repair
  • Dependovirus / genetics*
  • Eukaryotic Cells
  • Gene Targeting*
  • Genetic Therapy
  • Genetic Vectors*
  • Recombination, Genetic
  • Transduction, Genetic