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Adeno-associated virus (AAV) has been isolated from numerous vertebrate species since 1966. Besides its wide and promiscuous tropism, AAV infection does not re‐ sult in considerable toxicity or pathogenicity and is capable of achieving adequate and long-term levels of gene transfer, especially following generation of the AAV recombinant variant: rAAV. Due to these properties, rAAV has gained special at‐ tention as a viral vector for gene therapy in the last decade. Currently, there are 130 clinical trials taking place worldwide for several diseases testing the safety and effi‐ cacy profiles of rAAV. During preclinical and clinical studies, several challenges have arisen in terms of reaching the full therapeutic potential of rAAV, such as effi‐ cient delivery of the virus in a targeted and specific manner to a desired tissue. Im‐ portantly, the development of immune responses towards the viral capsids poses an obstacle to rAAV applicability in the clinical setting. Numerous approaches have been developed in order to tailor an optimized therapeutic virus for treating specif‐ ic diseases, including the use of different AAV serotypes or the creation of recombi‐ nant capsid variants with distinctive transduction and immunological profiles. This chapter reviews current information on rAAV clinical trials and the potential for combining rAAV platform with other technologies, such as induced pluripotent cells and gene editing.

AAV Biology, Infectivity and Therapeutic Use from Bench to Clinic