Syn-FLEX-GCaMP6s AAV (Serotype 8)

Adeno-associated virus (AAV) is a small, nonpathogenic, replication-defective parvovirus with a single-stranded DNA genome. Among the various serotypes, AAV serotype 2 is the most extensively studied of AAV biology and the subject of exploration as a gene delivery vector. Although recombinant AAV serotype 2 (rAAV2) is a promising vector, in vivo gene transfer using rAAV2 is suboptimal in many cases. To overcome these shortcomings of rAAV2 vectors, many researchers have begun to explore novel naturally occurring or artificially engineered AAV capsids, which are the major determinants of tropism and transduction efficiency.

To date, more than 100 naturally occurring primate AAVs have been reported. Many in vivo studies have shown that rAAV vectors packaged in alternative AAV serotype capsids exhibit serotype-specific tissue or cell type tropism and/or improved transduction efficiency. Peripheral intravenous injection of single-stranded rAAV8 vectors can transduce not only all hepatocytes in mice, but also all skeletal muscle and the entire myocardium of the mouse body. In addition to skeletal and cardiac muscle, rAAV8 can also transduce pancreatic acinar and islet cells, smooth muscle cells in various tissues, and neurons and glial cells in the brain with considerable efficiency following intravenous administration of the vector. As a result, a large number of applications utilizing systemic intravenous administration of rAAV8 vectors are being used to treat animal models of various human diseases that were previously considered refractory to gene therapy.