Safety of Adeno-Associated Viral Vectors

Vectors based on recombinant adeno-associated viruses (AAVs) are being extensively explored for gene therapy owing to some of their distinguishing characteristicss such as lack of pathogenicity, wide range of infectivity and ability to provide long-term transgene expression. For many of the same reasons, recombinant AAV (rAAV) vectors have also been used as vaccine carriers to elicit immune responses against their transgene products. Extensive studies of rAAV vectors in animal models and in the clinic have revealed some safety concerns relating to their construction and production, adverse events following delivery, potential integration of the vector’s genome into host cell genomes, and the impairment of rAAV-induced CD8+ T-cell responses, which could have dire consequences for rAAV-treated individuals. Further studies to advance our knowledge of the biology of AAV and rAAV vectors are deemed necessary to allow for their more successful application in the clinic.