Optimalisation and characterisation of production of recombinantadeno-associated virus (rAAV) as a vector for gene therapy.

The recombinant adeno-associated virus (rAAV) was developed as a candidate for gene therapy over the past few years. The absence of any pathogenicity of the wild type AAV (wtAAV), the simplicity of the AAV capsid and genoom (eicosahedral symmetry, 20nm; genoom of 4.8kb with two genes) and the absence of any wtAAV viral genes in the rAAV vector make of rAAV a unique vector. Additional properties of rAAV are the very broad range of cells that can be transduced, the stability of the rAAV capsid that allows purification and concentration of the rAAV and the ability of the rAAV genoom to integrate stable into the host genoom by the two AAV-inverted terminal repeats (ITR). In order to develop gene therapeutic models based on identified molecular targets in ovarian cancer, we decided to develop the rAAV system for this purpose. At this moment the production methods for rAAV need to be refined up to a level that permits the realization of this goal. Therefor our efforts will be directed in first place at the establishment of rAAV producer cell lines.