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Letter

Nature Genetics 34, 297–302 (1 July 2003) | doi:10.1038/ng1179

AAV serotype 2 vectors preferentially integrate into active genes in mice

Hiroyuki Nakai , Eugenio Montini , Sally Fuess , Theresa A Storm , Markus Grompe & Mark A Kay

Recombinant adeno-associated virus serotype 2 (rAAV2) is a promising vector for gene therapy because it can achieve long-term stable transgene expression in animals and human subjects after direct administration of vectors into various target tissues. In the liver, although stable transgene expression primarily results from extrachromosomal vector genomes, a series of experiments has shown that vector genomes integrate into host chromosomes in hepatocytes at a low frequency.