GFP Adeno-Associated Virus ( AAV-C4 )

AAV vectors are derived from adeno-associated viruses. AAVs are replication-defective members of the genus Dependoparvovirus in the family Parvoviridae. They were discovered in the 1960s as laboratory contamination of adenovirus (AdV) preparations. This close relationship is also found in nature, as AAVs rely on helper virus function to produce progeny. Although naturally occurring AAVs vary in the amino acid sequence of the capsid protein and genome length, all AAVs consist of an icosahedral protein capsid assembled from 60 subunits that protect the single-stranded DNA genome and mediate cellular infection.

Recombinant AAV vectors have been or are being used in 176 Phase I, II, and III clinical trials. AAV serotype 2 (AAV2) vectors have shown clinical efficacy in three human diseases: Leber congenital amaurosis (LCA), aromatic L-amino acid decarboxylase deficiency (AADC), and choroideremia. Despite these impressive achievements, it is becoming increasingly clear that the full potential of this vector system will only be realized once AAV vectors are engineered to improve cell transduction and evade host immune responses. The primary difference between GFP AAV-C4 and other AAV vectors is the insertion of the peptide PRGTNGP at position I587 of the capsid protein. This targeted genetic modification is designed to enhance specificity and affinity for tumor cells, making AAV-C4 particularly valuable in oncology research and potential cancer therapy.