Overview of adeno-associated viral vectors.

The use of adeno-associated virus (AAV) as a gene transfer vector has been steadily increasing over the past several years. AAV vectors have been particularly useful for applications where sustained gene expression is required. Prolonged in vivo expression following AAV treatment has been seen in the liver (1,2), brain (3,4), skeletal muscle (5,6), lung (7,8), and hematopoietic stem cells (9,10) of animal models. Therapeutic benefit from AAV treatment has been shown in a number of preclinical models of disease, including animal models of coagulopathies (11,12), lysosomal storage diseases (13,14), vision defects (15,16), and amino acid disorders. Clinical trials using AAV for the treatment of hemophilia B have begun, and early reports from these trials have been promising. In this introductory chapter to AAV, we will provide a brief overview of the molecular biology of this virus, an overview of methods of vector production, and a brief summary of the use of alternate AAV serotypes. The following chapters will then focus on specific methods and techniques for AAV transduction of the organs listed previously.