Published online Aug 27, 2015. doi: 10.5496/wjmg.v5.i3.28
Peer-review started: January 8, 2015
First decision: February 6, 2015
Revised: May 11, 2015
Accepted: May 16, 2015
Article in press: May 18, 2015
Published online: August 27, 2015
Core tip: Adeno-associated virus (AAV) has become the first gene therapy drug approved by the Western world and spurred huge excitement in the gene therapy field. The gene therapy community is forging ahead with cautious optimism despite some challenges down the road. A battery of more than 100 AAV serotypes and variants are available and AAV production and purification technologies have become well established. Several clinical trials with AAV vectors have yielded exciting results. This paper will give you the information needed to understand the current development of the gene therapy field with AAV vectors.