Host cell genome integration via recombinant adeno-associated virus (rAAV) vector delivery of therapeutic transgene sequence (rAAV-mediated transgene integration)

This target refers to the process by which a therapeutic gene, delivered by a recombinant adeno-associated virus vector, becomes integrated into the genome of a host cell. The goal is stable and long-term expression of the therapeutic transgene for gene therapy or genome engineering applications. rAAV vectors are engineered from wild-type AAV, a small single-stranded DNA virus. Wild-type AAV can integrate site-specifically into the human genome at the AAVS1 locus. Recombinant AAV vectors used in therapy lack Rep proteins and thus have a greatly reduced ability to integrate site-specifically; instead, they predominantly persist as episomal DNA within the nucleus. Rarely (~0.1% frequency), rAAV genomes may randomly integrate into host chromosomal DNA at non-homologous sites.

Other names

rAAV genome integrationAAV-mediated gene delivery and integrationRecombinant AAV integration into host cell genome

rAAV vector delivers a therapeutic transgene to a host cell. While most of the rAAV vectors persist as episomes, a small fraction integrates randomly into the host cell genome, leading to long-term expression of the transgene.