AAV6-Syn-iCre

Adeno-associated virus (AAV) is a small, 25 nm wide, icosahedral, non-enveloped virus with a 4.7 kb single-stranded DNA genome belonging to the Parvoviridae family. AAV is non-pathogenic and replication-defective, relying on co-infection with a helper virus such as adenovirus or herpes simplex virus. AAV was first discovered in the 1960s from adenovirus reservoirs and has recently become an important gene delivery vector for the treatment of disease. Glybera was the first AAV-based gene therapy drug approved by the European Medicines Agency in 2012. The U.S. Food and Drug Administration (FDA) approved two AAV-based gene therapy drugs, Luxturna in 2017 and Zolgesma in 2019.

To date, 13 different serotypes and more than 100 AAV variants have been identified based on phylogenetic analysis. Due to the heterogeneity of the capsid protein, each serotype exhibits different tropism and ability to transduce different cell types. Serotype 6 has a broad range of target cells and has been shown to successfully transduce cells in the central nervous system, human prostate, breast, and liver cancer cells, melanocytes, skeletal muscle, heart, lung, and eye. Recently, AAV serotype 6 (AAV6) has gained popularity for its ability to transduce lymphocytes and for use in generating chimeric antigen receptor T cells.