Delivery Systems for Gene Therapy: Adeno‐Associated Virus

The purpose of this chapter is to provide an overview of recombinant adeno-associated virus (rAAV) vectors and the current “state of the art” of their potential utility in gene therapy of hematopoietic disorders. The authors briefly review AAV biology and genetics and suggest more general reviews about AAV. The basic requirements that need to be fulfilled for gene therapy as treatment of hematopoietic disorders are presented.