Vectrology of adeno-associated viruses (AAV)

Abstract

In vivo gene replacement is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors are currently among the most frequently used viral vectors for gene therapy and they have shown therapeutic efficacy in a range of animal models. The lack of pathogenicity of the virus, low immunogenicity, its stability, and many available serotypes have increased AAV's potential as a delivery vehicle for gene therapy applications. There are some limitations to the use of rAAV in gene therapy. The first is their size. Due to the small size of the vector, the ability to conduct a therapeutic gene expression cassette is limited. Another limitation is the common occurrence of neutralizing antibodies in human populations. This review will focus on the biology of AAV, its use as a vector for gene therapy and mechanisms of AAV/host cell interaction.