AAV PHP.B-Syn-GFP

Adeno-associated virus (AAV) is a small and simple virus with a 4.7 kb single-stranded DNA genome consisting of only two genes, rep and cap, flanked by two inverted terminal repeats (ITRs) and encapsidated in a capsid. In addition, AAV integrates into the host genome at a low frequency, suggesting that it is safe for use in clinical studies. Another advantage of AAV vectors is their ability to provide stable transgene expression, which can last for more than a year in some in vivo experiments. AAV has been used to treat degenerative diseases of the central nervous system and retina, various types of muscular dystrophy, and heart, lung, and liver diseases. In addition to natural AAV serotypes, recombinant AAV (rAAV) serotypes are also included in pilot and clinical trials for the treatment of different diseases, such as hemophilia B. AAV cannot replicate alone by infecting host cells, but requires co-infection with another virus that provides helper functions, such as adenovirus, to replicate. Through alternative splicing and the use of alternative translation initiation, the two genes produce multiple transcripts and proteins. The large Rep proteins Rep78/68 and the small Rep proteins Rep52/40 are essential for AAV genome replication and packaging, respectively, and are encoded by the rep gene. VP1, VP2, and VP3, encoded by the cap gene, form the capsid in a 1:1:10 ratio. The assembly-activating protein (AAP) and membrane-associated accessory protein (MAAP), encoded by the cap gene, are essential for AAV capsid assembly and secretion, respectively.