CAG-FLEX-CaMPARI AAV (Serotype 9)

Somatic gene transfer via viral vectors is an indispensable tool in neuroscience, especially for whole animal studies. Adeno-associated virus (AAV) vectors are efficient in delivering genes to neurons and, therefore, have been widely used in basic and clinical research due to their high expression efficiency and relatively minimal immune response. Native AAV is a single-stranded parvovirus with a genome of approximately 4.7 kb. It is a dependent virus in that it requires the helper functions of other viruses to complete its life cycle.

AAV infection is not associated with any known disease. Most people have been exposed to wild-type AAV and are seropositive for AAV antibodies. Recombinant AAV vectors retain only 4% of the viral genome, the inverted terminal repeats, do not express viral genes, and are thought to persist as non-integrated episomes. All of these properties explain why gene transfer using recombinant vectors is relatively well tolerated in a variety of target tissues in different species. AAV has the potential for broad, long-term, and well-tolerated transgene expression. Efficiency has been improved due to a series of improvements in production/purification, promoters, AAV capsid serotypes (subtypes), and delivery methods.