mRFP AAV (Serotype 6)

The biology of adeno-associated viruses (AAVs) has been extensively studied since the discovery of the first adeno-associated satellite virus in the 1960s. Over the next three decades, the potential application of recombinant AAV as a gene transfer vector was realized. The vast majority of these pioneering studies were conducted with AAV serotype 2, the most common strain in humans. As a result, receptor usage, routes of infection, tissue tropism, antigenicity, immune signatures, and persistence of AAV2 vectors in various animal models are now well understood. During the transition of AAV2 vectors from the bench to the clinic in the last decade, several additional serotypes and novel AAV strains have been isolated. AAV6 can efficiently transduce cells in muscle, liver, lung, and the central nervous system—key targets for many gene therapy strategies. This broad tissue tropism makes AAV6 an attractive vector for the treatment of a variety of genetic diseases, from muscular dystrophy to cystic fibrosis. Coronary infusion of the closely related serotype AAV6 mediates potent and long-term myocardial gene expression. AAV6 has also been shown to be effective in delivering shRNA-encoding vector genomes to the heart in canine models. In addition, a recent report using a novel technique for myocardial gene delivery demonstrated robust cardiac gene transfer using AAV6 vectors in a sheep model. Similarly, high transduction efficiencies have been demonstrated following intramuscular injection of AAV6 vectors in nonhuman primate skeletal muscle and canine muscular dystrophy models.