GFP Adeno-Associated Virus ( rRGD587 )

Gene transfer vectors based on adeno-associated virus type 2 (AAV2) have shown great potential for human gene therapy, as AAV2-mediated gene expression is stable, there is no significant toxicity or immune response, and the efficiency of gene transfer in vivo is high. However, while AAV2 vectors can efficiently transfer genes into many different cell types, including muscle, brain, and liver, there appear to be some limitations that limit transduction of other cell types. Since the capsid protein is the sole mediator of cell entry and intracellular trafficking, recombinant AAV vectors can be targeted to alternative cellular receptors by genetically modifying the capsid. Many regions in the AAV capsid protein can be altered by incorporating small peptides. The RGD motif has in vivo targeting capabilities as a targeting peptide. Incorporation of this motif into the AAV VP3 monomer allows the virus to specifically use the RGD-integrin interaction as an alternative infection pathway, significantly improving the ability of the virus to transduce several cell types that are normally difficult to infect with the virus. Importantly, RGD-modified AAV2 vectors physically interacted with targeted integrin receptors both in solid-phase binding assays and on the cell surface, and this interaction was sufficient and necessary for targeted gene transduction.