AAV8-CMV-saCas9-U6-sgRNA

Adeno-associated virus (AAV) is a non-enveloped, single-stranded DNA virus belonging to the Parvoviridae family (genus Dependoparvovirus). Structurally, virions assemble into icosahedral particles with a diameter of approximately 250 Å. Full permissive infection requires the presence of adenovirus or herpes simplex virus. Because AAV is non-pathogenic to humans, AAV vectors are often used in gene therapies that are administered directly to patients by infusion or topical administration (in vivo). As of April 2024, the U.S. Food and Drug Administration (FDA) has approved six AAV vector-based gene therapy drugs.

Thirteen human/non-human primate AAV serotypes (and more than 100 variants) have been identified, which not only exhibit different cell tropisms, but also different seroprevalence and geographic distribution within the population. AAV serotypes have been extensively studied as gene therapy tools because serotypes influence tissue tropism. Therefore, choosing the correct AAV serotype for gene delivery is critical. The AAV8 serotype has a high affinity for hepatocytes, with hepatocyte transduction rates as high as 95% depending on the dose, making it highly effective for liver-targeted gene therapy.