scAAV DJ/8-Syn-GFP

Adeno-associated virus (AAV) is one of the most promising vectors for human gene therapy because of its persistence, high efficiency, and nonpathogenicity in gene delivery. To date, about 12 serotypes of AAV vectors have been reported, each with its own outer capsid protein. Tissue tropism is determined by the serotype of the AAV vector and is of therapeutic importance. Vector systems based on other AAV serotypes are being investigated that have more efficient gene transduction and different cell and tissue specificities.

In addition to the naturally occurring AAV lines, novel recombinant AAV capsid variants with high efficiency gene transfer and tropism have recently been generated by rational design or directed evolution. Continuous evaluation of novel AAV vectors for human gene therapy and its basic research is important, because cell type-specific gene introduction tailored for each disease will make the treatment more effective and minimize immunological sequelae, while improvements in packaging capacity expand the range of therapeutic genes to be selected. AAV-DJ (type 2/type 8/type 9 chimera) is a recombinant of 8 different wild-type viruses and is widely used for gene transduction due to its ability to efficiently transfer genes into a variety of cell types. For example, AAV-DJ vectors were used to knock out genes in porcine fibroblasts with a higher targeting frequency than other natural serotypes. AAV-DJ far outperformed all other serotypes in terms of transduction efficiency in human keratinocytes.