AAV8-CAG-FLuc

Compared with other viral vectors, AAV requires a lower viral load, so it can be used to treat certain rare diseases. This vector is often used for non-dividing target cells, such as liver, nervous system, eye, and skeletal muscle cells. The advantage of using this vector is that once delivered, the DNA it carries can exist in a free form in the patient's body for a long time, even a lifetime, and will not cause potential insertion mutations within the host cell genome, thereby improving the safety of gene therapy. In addition, different AAV serotypes have specific affinities for different tissues, so they can be used to design treatments for different target organs. AAV8 has a wide range of tissue tropism, including brain, liver, heart, retina, lung, and muscle cells. AAV2, AAV5, AAV8, AAV9 and other serotypes are more commonly used for transfecting hepatocytes. Studies have shown that AAV8 and AAV9 have strong affinity for hepatocytes, among which AAV8 has the strongest affinity for the liver. In mice, dogs or primates, rAAV8 can efficiently and stably transfect hepatocytes by peripheral vein, portal vein or intraperitoneal injection. Studies have shown that in the liver, AAV8-mediated target gene expression is nearly 10 to 100 times higher than other serotypes. AAV8 vectors can also transfect cells in other tissues, including muscle, heart, pancreas, and brain. In newborn dogs, a single jugular vein injection of AAV8 can show extensive and sustained systemic transduction in skeletal and cardiac muscles, and this expression can persist in the heart for at least one year.