AAV1-hSyn-NULL

AAV has several properties that make it an attractive viral vector candidate for gene therapy applications. First, AAV is a non-pathogenic virus, as no disease has been associated with it. AAV vectors have also been shown to stably integrate into the target genome, a property that is critical for gene therapy applications because it maintains high levels of expression without long-term effects on target cell function. In addition, since AAV vectors only have endonuclease transcriptional regulatory regions (ITRs), they do not interfere with the regulation of the inserted gene. More specifically, unlike all currently available gene editing platforms, AAV vectors are unique in that they utilize a homologous recombination pathway that does not involve exogenous nucleases, thereby providing a highly precise editing process that maintains genome integrity without increasing the mutation burden at the target site. With this unique gene editing property, AAV vectors have now become the leading platform for in vivo gene therapy delivery.

Another attractive property is that AAV can effectively transduce a wide range of cells, tissues, and hosts in vivo and in vitro, including dividing and non-dividing cells in humans, non-human primates, mice, dogs, cats, and various other models. In addition to its genetic properties, the unique physical properties of AAV provide additional reasons for its use in gene therapy, such as ease of handling, resistance to pH changes, heat resistance, and detergent resistance.