About
Adeno-associated viruses (AAV) are non-pathogenic ssDNA viruses, which are subject of intense studies as viral vectors for gene therapy. The virus transduces a variety of dividing and non-dividing cells showing long-term gene expression with low cellular immune response. AAV has been used in several clinical trials (e.g. FIX, CFTR, Parkinson's, Canavan disease) showing no serious vector-related adverse effects. Methods for the characterization of AAV preparations currently include titration ELISA, qPCR, ddPCR, DNA dot blot, determination of transducing units, infectious center assay, SDS-PAGE or electron microscopy.
