Adeno-associated viruses (AAV) are nonpathogenic ssDNA viruses, which are a subject of intense studies as viral vectors for gene therapy. The virus transduces a variety of dividing and nondividing cells showing long-term gene expression with low cellular immune response. AAV has been used in several clinical trials (e.g. FIX, CFTR, Parkinson&rsquo;s, Canavan disease) showing no serious vector-related adverse effects. AAV9 is especially useful for applications in the CNS.
Methods for the characterization 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.
AAV preparations contain up to 10fold more empty virus capsids than filled, therapeutically active virus particles. Since both full and empty particles can induce an immune reaction, it is crucial for in in vivo appilcations to determine the complete particle titer.
Immunotitration by PROGEN's AAV9 Titration ELISA offers a fast, sensitive and reproducible method for titration of intact AAV9 wt virions, AAV9 recombinant virions or assembled and intact empty AAV9 capsids.