GFP Adeno-Associated Virus ( AAV-NDVRAVS )

Different strategies have been developed to achieve higher transduction efficiency and specificity of AAV-based vectors, mainly by engineering the viral capsid. A successful screening system for targeted AAV2 vectors has been developed by displaying peptides directly on the viral capsid, allowing the selection of improved gene therapy vectors even without prior knowledge of the potential binding receptors and corresponding ligands. Combinatorial AAV2 libraries displaying random peptide ligands at amino acid position 588 of the capsid protein have been successfully used to select a large number of capsid mutants that efficiently transduce various cell types. The strong restriction to specific peptide motifs observed in these studies supports the hypothesis that target cell binding is only one of several factors influencing the selection process and suggests that peptide ligands inserted into the AAV2 capsid can influence different post-binding processes associated with gene transduction. A notable feature of GFP AAV-NDVRAVS is the insertion of a unique peptide sequence, NDVRAVS, at position I588 of the capsid protein. The main cells targeted by this capsid-engineered AAV are endothelial cells, which form the lining of blood vessels and play a key role in vascular biology, angiogenesis, and various diseases. By designing viral vectors that specifically target these cells, researchers can deliver genes of interest directly to endothelial cells, facilitating the study of gene function, the effects of genetic mutations, and the potential of gene therapy in treating vascular disease.