scAAV DJ/8-GFP

Adeno-associated virus (AAV) was discovered as a contaminant in an adenovirus preparation in electron micrographs and soon became a subject of interest for scientists around the world. More than 50 years later, interest in AAV as a gene therapy vector continues to grow. AAV belongs to the parvovirus family, specifically the subfamily of dependoparvovirus. Members of this subfamily require a helper virus, such as adenovirus (Ad) or herpes simplex virus (HSV), to facilitate efficient infection and replication. Although an estimated 90% of humans are AAV seropositive, these viruses do not cause any known human disease, an important safety criterion for their use in gene therapy approaches.

Clinical trials using recombinant AAV (rAAV) vectors have shown impressive results for Leber congenital amaurosis, hemophilia B, spinal muscular atrophy, and other diseases. Production of rAAV in human cells (HEK293) transiently transfected with plasmids is probably the most common approach, but the use of insect cells and baculovirus is very convenient for industrial production. Other possible approaches include the use of a recombinant HSV complementation system in mammalian cells cultured in suspension (BHK21 or HEK293) or in mammalian-derived producer cell lines containing the rep and cap genes and the AAV vector integrated into the genome. In the latter case, amplification of the rAAV is initiated following infection with a helper virus such as Ad.