Adeno-associated virus-based vectors

Abstract

Viral vectors based on recombinant adeno-associated virus have gained increasing interest over the last two decades as promising delivery vehicles in gene therapy. This enthusiasm is based on their ability to infect a broad range of tissues including proliferative and quiescent cells, to establish long-term expression in vitro and in vivo, combine to an excellent safety profile, as they are replication deficient, poorly immunogenic, and have not been associated to any disease. This chapter provides detailed protocols for small-scale production and purification of adeno-associated vectors and currently used methods for the titration and quality controls of these vectors.