scAAV DJ-Syn-GFP

Adeno-associated virus (AAV) is a small, non-pathogenic virus that can be engineered into recombinant adeno-associated virus (rAAV) vectors for the delivery of specific genetic material to target cells. rAAV has become a popular tool for genetic engineering and gene therapy due to its non-pathogenicity and ability to efficiently transduce a variety of cell types, including actively dividing and post-mitotic cells. The use of rAAV for clinical gene delivery has the potential to treat or even cure thousands of genetic diseases, as highlighted by the recent market approval of multiple gene therapies using rAAV. In addition, more than 130 clinical trials using AAV are currently underway, highlighting rAAV as a key transformational gene delivery tool.

With regard to rAAV serotypes, there are currently 12 known naturally occurring AAV serotypes. In addition to these naturally occurring AAV serotypes, artificial AAV serotypes have been created to increase infectivity and/or enhance their ability to evade neutralization by pre-existing AAV antibodies in circulation. In this regard, the recombinant AAV-DJ pseudo-serotype vector has been shown to outperform eight naturally occurring AAV serotypes in transducing a variety of different cell types in vitro and infecting liver tissue in vivo. The AAV-DJ pseudo-serotype was developed using the "adaptive DNA family shuffling" technique, which aims to create a complex mixed capsid library from eight different wild-type viruses. An important additional feature of this AAV-DJ capsid is that it was selected in the presence of mixed human antisera. Therefore, this capsid is likely to be able to evade neutralization by existing AAV antibodies that may be present in patients. These advantages could make this pseudo-serotype more attractive than other AAV serotypes and more effective as a gene therapy vector for clinical use in humans.