Adeno-Associated Virus (AAV) is a nonpathogenic virus species that belongs to the Parvoviridae family. AAV is a single-stranded nonenveloped DNA virus causing a very mild immune response in humans. The virus is classified as small (25nm) and infection with AAV occurs only with the help of other viruses, either herpesvirus or adenovirus (hence adeno-associated virus). There are twelve serotypes of human AAV but the number of nonhuman AAVs exceeds 100. AAV-2 is the only mammalian DNA virus that is known to integrate in a specific site of the genome.
The low immunogenicity and the ability to infect dividing as well as non-dividing cells with stable expression make the adeno-associated virus an attractive vector for the application in gene therapy. AAV has proven successful as a gene therapy vector with the ability to attach and enter the target cell, transfer to the nucleus and express the transgene in a stable manner over a sustained period of time. AAV has been used in several clinical trials (e.g. FIX, CFTR, Parkinsons’s, Canavan disease) showing no serious vector-related adverse effects. Clinically relevant tissues have, however, shown a diversified susceptibility to AAV infection. The gene transduction with AAV-2 vector in muscle, retina, liver and heart resulted in lower gene expression than when using AAV serotypes 1, 5, 8 and 9 for transduction in the respective tissues. The challenge lies, however, in the resistance to transduction by available serotypes exhibited by some tissues.
Methods for the characterisation of AAV preparations currently include titration ELISA, real-time PCR, DNA dot blot, determination of transducing units, infectious center assay, SDS-PAGE or electron microscopy. You may enrich your research on AAV by browsing through Progen's AAV Product portfolio and access the highest quality research tools.