The third section of this book is ‘Adeno-associated-virus Vector’. Chapter nine by Sun et al. introduces the adeno-associated virus (AAV) mediated β-thalassemia gene therapy. Human hematopoietic stem cells (HSCs) were obtained from β-thalassemia patients, transfected with the recombinant AAV containing β-globin gene. The transfected cells were then transplanted into Nude/SCID mice, and the long term expression of β-globin in vivo was examined. In the tenth chapter, Korecka et al. compare AAV serotypes for gene delivery to dopaminergic neurons in the substantia nigra (SN)
