Abstract: The non-pathogenic property of adeno-associated virus(AAV)has made its recombinants one of the most attractive candidate gene therapy vectors for its outstanding feature in safety, which have already been applied in the clinics for the treatments of a variety of diseases. As the basic researches in molecular virology of AAV have progressed rapidly within the past two decades, novel strategies to improve the specificity of AAV infection in host/targeted cells and the efficiency of viral transduction have been attempted, part of which are being subsequently translated for clinical trials. This review will introduce the current understandings of AAV with its basic biological characteristics, and take several successful laboratory researches as examples to discuss the strategies and approaches for developing improved AAV vectors as well as their features for application. The current status and the challenges for AAV-mediated gene therapy are also addressed.

Key words: adeno-associated virus, gene therapy, recombinant vectors, clinical trials