CAG-FLEX-GCaMP6f AAV (Serotype 8)

Adeno-associated virus (AAV) remains a promising viral vector for delivering therapeutic genes in human gene therapy. In 2012, the European Commission approved Glybera, an AAV vector carrying the human lipoprotein lipase gene, for commercial use in the treatment of lipoprotein lipase deficiency. In addition, over the past five years, AAV has achieved many successes in preclinical, early, and late clinical trials for the treatment of various diseases, including muscular dystrophy, hemophilia, Leber's congenital amaurosis, age-related macular degeneration, heart disease, cancer, and neurodegenerative diseases such as Parkinson's disease and Canavan disease. However, despite these advances, the infection pathways of AAV vectors continue to evolve. At least 12 AAV serotypes from humans and primates have been identified. Many viruses bind to different receptors on the surface of host cells. Several serotypes have been reported to bind to primary cell surface receptors and then interact with secondary receptors that can facilitate viral entry. The major cell surface attachment receptors identified to date include heparan sulfate proteoglycans of AAV serotypes 2, 3, and 6; N-terminal galactose of AAV9; and specific N- or O-linked sialic acid moieties of AAV1, 4, 5, and 6. Secondary receptors include fibroblast growth factor receptor (FGFR) and integrins of AAV2; hepatocyte growth factor receptor (c-Met) of AAV2 and 3, and platelet-derived growth factor of AAV5, the latter also modified by sialic acid.