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Nat Rev Microbiol. 2005 Nov;3(11):837-47.

Precise hit: adeno-associated virus in gene targeting.

Author information

1
Department of Gene and Cell Medicine, Mount Sinai School of Medicine, New York, USA.

Abstract

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.

PMID:
16261169
DOI:
10.1038/nrmicro1266
[Indexed for MEDLINE]

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