GFP Adeno-Associated Virus ( AAV-LSLAS )

Numerous in vitro and in vivo studies have shown that the 12 different human and non-human primate AAV serotypes sequenced to date (AAV1-12) exhibit different tissue and cell tropisms. Therefore, pseudotyping the AAV2 vector genome with capsids from different AAV variants or generating chimeric capsids may enable the delivery of recombinant AAV (rAAV) to previously refractory cell types. However, the use of pseudotyped or chimeric vectors does not imply greater cell type specificity nor does it help to overcome pre-existing immunity to the parental serotype. Gene transfer efficiency and specificity can be improved by rational vector targeting, for example, through bispecific linker molecules or chemical modifications of the AAV capsid. A notable feature of GFP AAV-LSLAS is the insertion of a specific peptide sequence, LSLASNRPTATS, at position I588 of the viral capsid. This peptide insertion is a complex engineering manipulation designed to alter the tropism of the virus, enabling it to selectively target endothelial cells. Endothelial cells form the lining of blood vessels and play a key role in vascular biology, making them an important target for therapeutic intervention in diseases such as cardiovascular disease, inflammatory diseases, and tumor angiogenesis. Engineered capsids with the LSLASNRPTATS peptide can be precisely delivered to endothelial cells and combined with a GFP reporter gene to achieve efficient and observable transgene expression. These advances are expected to improve treatment outcomes for diseases that require endothelial cell targeting and have the potential to transform future gene therapy applications and vascular biology research.