AAV9-CAG-RLuc

Thanks to the rapid development of biomedical technology, many new high-tech treatments for human diseases have been developed in large numbers today. However, despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatments. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAV). AAV is effectively used to treat many inherited and acquired human diseases, more specifically, monogenic inherited diseases, i.e. diseases caused by mutations in one gene. Today, many AAV-based gene therapy drugs are being studied in preclinical and clinical trials, and many new drugs are available on the market for the treatment of many human diseases, including those that were previously considered untreatable. Adeno-associated virus (AAV) is a single-stranded virus belonging to the Parvoviridae family that is safe, has low toxicity, and is able to express stably with very low immunogenicity. Therefore, AAV is considered an interesting candidate vector for gene delivery. AAV can infect both dividing and non-dividing cells and persists outside the chromosome without integrating into the host cell's genome. There are more than 10 serotypes of AAV. Compared with other serotypes, AAV9 has the additional advantages of being able to deliver large amounts of transgenes into host cells and being able to infect a wide variety of different animal tissues.