ESTABLISHMENT OF AN AAV VECTOR PRODUCTION UNIT (AVU)

This research proposal is aimed at the establishment of a facility for the production and characterization of viral vectors based on the adeno-associated virus (AAV) for gene therapy applications in animal models of human disease. AAV is a small virus presenting several appealing features for gene transfer, since it is widely diffused in the general population and has never been associated to human disease. Once injected in vivo, AAV vectors persist for prolonged periods of time and efficiently express their therapeutic genes. As a gene therapy vehicle, AAV is ideal for clinical applications in which gene transfer is sought in the skeletal muscle, heart, arterial wall, brain and retina. Besides being a clinically relevant gene therapy vector, AAV also represents an appealing tool for biological investigation in the living animal in all cases in which the analysis of the effects of the prolonged expression of a given gene is required.