Site-Specific Targeting of DNA Plasmids to Chromosome 19 Using AAV Cis and Trans Sequences

Adeno-associated virus (AAV) is of the genus Dependovirus and a member of the family Parvoviridae (1 ). AAV is unique, in that it requires co-infection of a second helper virus (i.e., adenovirus [Ad] or herpesvirus) to undergo productive infection. In the absence of a helper virus, AAV will integrate preferentially into the host chromosome (ch). Targeting of AAV to ch19.13.3 qter locus has been documented at a frequency of 70% or greater (2 ). Because of AAV’s unique ability to integrate site-specifically, extensive research has been devoted to understanding how AAV integrates, with the hope of creating targeting vectors for gene therapy.