GFP Adeno-Associated Virus ( AAV-PENSV )

Vectors based on adeno-associated viruses (AAV) are being developed as clinically relevant gene transfer systems because they combine a number of advantageous properties. They are derived from non-pathogenic viruses, are able to transduce both dividing and non-dividing cells, and induce only moderate immune responses. In addition, AAV vectors have been shown to integrate at low frequencies in a semi-random manner and provide persistent transgene expression mainly in an episomal state. However, poor transduction of many therapeutic target cells, widespread humoral immunity against AAV2, and especially broad and nonspecific tropism remain major obstacles limiting widespread clinical application. Different strategies have been developed to achieve higher transduction efficiency and specificity by eliminating natural tropism and re-targeting vectors to specific target cell receptors, mainly by engineering the AAV capsid.

GFP AAV-PENSV is derived from AAV serotype 2 (AAV2) and has undergone significant capsid engineering to improve its specificity and efficiency. One of the key modifications is the insertion of the peptide sequence PENSVRRYGLEE at position I588 of the capsid protein. Engineered peptides in the AAV-PENSV capsid enhance the ability of the virus to effectively bind to and enter endothelial cells, allowing for precise gene delivery and expression.