Among the many viruses currently being developed as vectors for gene transfer in vivo, the adeno-associated virus (AAV) is a most primising candidate. It fulfills most of the requirements of an “ideal” vehicles for gene transfer:

• AAV derived vectors infect both dividing and nondividing cells of multiple tissue origin
• It can persist in the transduced cells as an integrated provirus or in episomal forms resulting in a stable long-time gene expression
• It is nonpathogenic.
• On the other hand, AAV derived vector system has one major limitation: the size limitation of the cDNA tat can be inserted between both ITRs (< 4,5 kb).

AAV derived vectors are developed in Vectalys to generate animals models where the vector is directly administrated to the animal to develop reliable disease models representative of the human situation.