CAG-ChR2-YFP AAV (Serotype 5)

Adeno-associated virus (AAV) has several properties that make it an attractive viral vector candidate for gene therapy applications. First, AAVs are non-pathogenic viruses, as there are no diseases associated with them. AAV vectors have also demonstrated the ability to stably integrate into the target genome, a property that is essential for gene therapy use, given that high levels of expression can be maintained without long-term effects on target cell function. Furthermore, due to having only ITRs, AAV vectors are also unable to interfere with the regulation of the inserted gene. More specifically, in contrast to all currently available gene editing platforms, a unique property of AAV vectors is the utilization of a homologous recombination pathway that does not involve exogenous nucleases, thus providing a highly precise editing process that maintains genome integrity without increasing the mutational burden at the target site. Due to this unique gene editing property, AAV vectors are currently the leading platform for in vivo gene therapy delivery.

Another attractive feature is the very broad range of cells, tissues, and hosts that AAV can effectively transduce in vivo and in vitro, including dividing and non-dividing cells in humans, as well as murine, canine, NHPs, feline, and various other models. In addition to genetic properties, the unique physical properties of AAVs represent additional reasons why they should be used for gene therapy, such as ease of manipulation, resistance to pH changes, thermal changes, and detergents.