AAV2-Syn-FLPo

Adeno-associated virus (AAV) is a single-stranded DNA parvovirus with good potential as a gene therapy tool. It belongs to the genus Dependovirus and requires genes from a helper virus (such as adenovirus or herpes virus) for successful replication and assembly. The AAV genome spans approximately 4.7 kilobases (kb) and contains two open reading frames: the rep region and the cap region. The rep region encodes proteins associated with replication, and the cap region encodes three proteins (VP1, VP2, and VP3) that together form the viral capsid. Inverted terminal repeats (ITRs) flank the viral genome and are the only requirement for packaging the DNA into capsids. Therefore, recombinant AAV (rAAV) vectors can be generated by replacing the wild-type coding region with any gene or DNA sequence of interest (up to approximately 5 kb). In addition, vector genomes containing ITRs from one serotype can be packaged into capsids from another serotype, resulting in recombinant pseudotyped viral vectors. Although multiple capsid serotypes have been identified that display a range of tissue tropisms, the vast majority of experiments in AAV virology have been performed with vectors of AAV serotype 2. To transduce cells, AAV vectors must enter cells and deliver their single-stranded DNA genome to the nucleus, where the genome becomes double-stranded before transcription.