AAV2-CAG-FLuc-2A-GFP

rAAV is a popular viral vector system in clinical trials. The vast majority of past and current clinical trials employ AAV vectors based on serotype 2. Most of these applications focus on the treatment of monogenic disorders such as cystic fibrosis, Duchenne muscular dystrophy, alpha-1-antitrypsin deficiency and haemophilia B, and cancer using local vector application or rAAV modified cells, respectively. In addition, rAAV-2 is routinely used as a gene transfer vector for in vitro transduction and in a large number of pre-clinical animal models.

In vivo AAV serotypes other than rAAV-2 have demonstrated superior transduction efficiencies for a variety of tissues. For example, rAAV-1 is reported to be more effective than rAAV-2 in transducing muscle, arthritic joints, pancreatic islets, heart, vascular endothelium, central nervous system (CNS), and hepatocytes, whereas rAAV-3 appears well suited for transducing cochlear inner hair cells, AAV-4 for transducing the brain, AAV-5 for transducing the CNS, lung, eye, arthritic joints, and hepatocytes, rAAV-6 for transducing muscle, heart, and airway epithelium, rAAV-7 for transducing muscle, and rAAV-8 for transducing muscle, pancreas, heart, and liver.