GFP Adeno-Associated Virus ( AAV-CNH )

Endothelial cells form the lining of blood vessels and play a key role in a variety of physiological processes, including angiogenesis, blood pressure regulation, and barrier function. Targeting endothelial cells is particularly beneficial in studying and treating cardiovascular disease, cancer, and other diseases involving vascular abnormalities. AAV vectors are attractive for in vivo vascular gene therapy because they confer sustained transgene expression and are less immunogenic than first-generation adenoviruses. Unfortunately, they have a poor natural tropism for endothelial cells (ECs). Alternative serotypes do not appear to improve EC transduction levels. Insertion of engineered peptides into the AAV capsid allows for more selective and efficient endothelial cell transduction, maximizing therapeutic potential while minimizing off-target effects. AAV-CNH is derived from AAV serotype 2 (AAV2). Capsids are critically important because they determine the ability of the virus to infect different cell types and tissues by interacting with cellular receptors. For AAV-CNH, the capsid was carefully engineered by inserting the peptide sequence CNHRYMQMC at isoleucine residue 587 (I587). This modification enhances the specificity of AAV-CNH particles for endothelial cells, thereby expanding their utility in vascular-targeted gene therapy.