AAV8

AAV8 is an adeno-associated virus serotype 8, a non-pathogenic, non-enveloped parvovirus that is widely used as a **gene therapy vector** due to its strong tissue tropism, especially for the liver. Compared to other AAV serotypes, AAV8 offers exceptionally high transduction efficiency in hepatocytes, as well as notable capability to transduce muscle, pancreas, kidney, and retinal cells with systemic or local delivery. AAV8 utilizes the laminin receptor (LamR) as its primary cell entry receptor and is advantageous for clinical gene delivery because it is relatively less recognized by pre-existing human neutralizing antibodies, increasing the effectiveness of gene transfer. Preclinical and clinical development has focused on genetic diseases such as hemophilia A and B, Wilson’s disease, familial hypercholesterolemia, Crigler-Najjar syndrome, and other inherited liver, muscle, cardiac, and retinal disorders. rAAV8 (recombinant AAV8) is typically engineered to package therapeutic DNA for in vivo delivery. Improvement in gene therapy success with AAV8 relates to high vector yield, efficient tissue targeting, and comparatively low immunogenicity[1][2][3][4].

Other names

adeno-associated virus serotype 8AAV serotype 8rAAV8rAAV-8rAAV 8recombinant adeno-associated virus 8