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

Research output: Contribution to journalArticlepeer-review


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. Despite the low integration efficiency, recent reports of retroviral insertional mutagenesis in mice6 and two human subjects have raised concerns about the potential for rAAV2-mediated insertional mutagenesis. Here we characterize rAAV2-targeted chromosomal integration sites isolated from selected or non-selected hepatocytes in vector-injected mouse livers. We document frequent chromosomal deletions of up to 2 kb at integration sites (14 of 14 integrations, 100%; most of the deletions were

Original languageEnglish
Pages (from-to)297-302
Number of pages6
JournalNature Genetics
Issue number3
Publication statusPublished - Jul 1 2003

ASJC Scopus subject areas

  • Genetics(clinical)
  • Genetics


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