In the last fifty years since its accidental discovery, the non-pathogenic adeno-associated virus (AAV) has emerged as a highly versatile vector system for both research and clinical applications. AAV is a very simple non-enveloped virus with a single-stranded linear DNA genome flanked by inverted terminal repeat sequences (ITRs). This apparent simplicity has enabled the advancement of recombinant AAV (rAAV) into the clinic. I would like to provide a historical perspective on the progression of AAV for gene therapy from discovery to the clinic during this seminar...