AAV is a small virus that infects humans and other primate species. It is not currently known to cause any disease, however, it causes a very mild immune response, lending further support to its apparent lack of pathogenicity. Its genomic structure consists of a replication (rep, left) and capsid (cap, right) gene flanked by two inverted terminal repeats (ITR’s). The rep gene is important for integration of wild AAV onto a specific site on human chromosome 19. So, our goal is to create a recombinant AAV in which the cap gene (and situationally the rep gene) are deleted and replaced by the gene of interest (transgene).
For more details about the technology: Click here