AAV2-CAG-Cre

Adeno-associated virus (AAV) is a small (25 nm), non-enveloped virus that packages approximately 4.7 kilobases of linear single-stranded DNA (ssDNA). The ssDNA genome of wild-type AAV contains two 145-nucleotide inverted terminal repeats (ITRs) flanking two open reading frames (ORFs) of structural (cap) and packaging (rep) genes. The rep ORF is essential for viral replication and packaging of the viral genome, while the cap ORF encodes the viral capsid proteins VP1, VP2, and VP3 and the assembly activation protein (AAP), which are essential for viral particle production. During capsid assembly, the positive and negative strands are encapsidated into separate virions at equal frequencies. After being initially discovered as a contaminant of adenoviral preparations, AAV has attracted increasing attention as a human gene therapy vector over the past two decades. This is primarily due to its lack of pathogenicity in humans, high transduction efficiency, low inflammatory response, and broad host cell tropism. Therefore, recombinant AAV (rAAV) has attracted great interest in experimental and clinical gene therapy applications. rAAV vectors have been generated from several AAV serotypes, but to date, recombinant AAV serotype 2 (rAAV2) is the most extensively studied rAAV-based gene transfer vector.