Syn-GCaMP6s AAV (Serotype 8)

Recombinant adeno-associated viruses (rAAV), such as AAV8, are essential for the delivery of gene therapy. These viral vectors can effectively and safely deliver therapeutic genes to target cells in various tissues, including liver, muscle, and brain, in clinical trials. AAV8 is a special model because it has high transduction efficiency, low immunogenicity, reduced pathogenicity, and establishes long-term transgene expression. In addition, the AAV genome is considered easy to obtain and edit. AAV particles can be efficiently purified at very high titers and then lyophilized for convenient handling and storage. Therefore, it has become a promising tool for gene therapy applications.

Of all AAV vectors, AAV8 has the lowest observed seroprevalence of neutralizing factors and better transduction efficiency compared to other serotypes. The three-dimensional structure of the AAV8 capsid that packages the viral DNA has been determined using various methods. The capsid is composed of 60 copies of three proteins: VP1, VP2, and VP3, of which VP3 contains nine hypervariable regions that are responsible for the sequence and structural variations between AAV serotypes, giving them unique receptor binding, tissue tropism, and antigenicity.