单克隆 抗腺病毒 腺相关病毒9型 抗体, 小鼠免疫球蛋白G1

AAV9 (Adeno-Associated Virus Serotype 9) is a highly efficient and safe gene therapy vector belonging to the Parvoviridae family. It is characterized by broad tissue tropism, enabling efficient transduction of the heart, liver, skeletal muscle, central nervous system (with the ability to cross the blood-brain barrier), and retina, among others. Compared to other serotypes, AAV9 exhibits stronger systemic diffusion capacity and lower immunogenicity, reducing the risk of host immune rejection. Its single-stranded DNA genome (~4.7 kb) allows long-term stable expression of transgenes but has a limited packaging capacity (≤5 kb). AAV9 has been utilized in clinical trials for various diseases, such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial hypercholesterolemia.