CAG-mCherry AAV (Serotype BR1)

Gene therapy using viral vectors is a valuable strategy. Commonly used vectors include modified lentiviruses, adenoviruses, retroviruses, or adeno-associated viruses (rAAV). These vectors are chosen for their ability to deliver genes to specific tissues or organs with high efficiency, persistent expression of the introduced gene, and overall safety and tolerability. rAAV in particular meets these criteria. The success of rAAV in gene therapy is evident from the large number of clinical trials conducted. There have been more than three hundred clinical trials involving rAAV, accounting for 9.4% of trials in the gene therapy field. Successful trials have led to the development of gene medicines using AAV as a transgene vector. AAV has been recognized in the scientific community since Bob Atkinson discovered small, antigenically distinct "impurities" while studying adenovirus (Ad) in 1965. It was noted that replication of these particles occurred only in the presence of Ad. Subsequent studies confirmed this relationship and showed that co-infection with other viruses, such as herpes simplex virus (HSV) and cytomegalovirus (CMV), is necessary for the synthesis of AAV antigens. This characteristic is also reflected in the taxonomy of AAV, which is included in the Dependovirus genus of the Parvoviridae family. AAV can infect both dividing and non-dividing cells. These small viruses have no envelope, are about 26 nm in size, and have a single-stranded genetic material consisting of 4.7 kb. The AAV genome includes parts such as the promoter, polyA, replication response gene (Rep), capsid structural gene (Cap), and assembly activation protein (AAP) gene. At both ends of the genome, there are terminal repeat sequences consisting of 125 nucleotides, called inverted terminal repeats (ITRs).