Adeno-associated viral vectors for gene therapy

To date, studies with rAAV have proven very successful in demonstrating the primary requirements for effective gene therapy: This includes a) the ability to efficiently produce vector substrate, b) demonstration of vector transduction in dividing and non-dividing cells, and c) long term gene expression without toxicity or a host immune response. Future generation of more effective AAV vectors are expected as we gain more insight into the biology of AAV life-cycle. This review will focus on accomplishments currently seen with AAV vectors to date, as well as describe new insights into AAV biology that will impact next generation of AAV vectors.