The work summarized in this chapter illustrates the increasingly rapid progress that is now being made in developing therapeutic applications of AAV vectors. The early clinical testing of AAV vectors for the treatment of CF has been extremely important in initiating the regulatory environment for AAV vectors and demonstrating the inherent good safety pro file of AAV vectors. As more groups have extended investigations to additional in vivo models, the potential utility of AAV vectors as therapeutic gene delivery vehicles has gained more widespread interest. The development of more sophisticated production systems for AAV vectors has enhanced both the quantity and quality of vectors that can be produced. Additional work to modify the transduction efficiency by judicious choice of serotype for the capsid or by modification of the capsid structure will expand the use and potency of AAV vectors. In addition, further understanding of the intracellular metabolism of AAV vectors also will increase the sophistication with which these vectors can be deployed. It is likely that in the next few years there will be a significant increase in clinical testing of AAV vectors for additional therapeutic applications.

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