Application of adeno-associated virus (AAV) as a gene therapy vector

Adeno-associated virus (AAV) is a small virus encapsidates a single-stranded DNA genome. AAV have rapidly gained popularity as a vector in genetic engineering and gene therapy applications, due to its lack of pathogencity, wide range of infectivity, and ability to integrate into human genome (19q13.4). Recombinant AAV vectors have been used for treatment of a variety of diseases such as hemophilia, cystic fibrosis, and Parkinson. A challenge with wide application of this vector is the relatively small packaging size of the viral genome to enable the delivery of a large transgene. Therefore it is essential making changes in it to overcome this problem. In this review we will introduce with this virus and the new finding in reginal of gene therapy with this virus.