rAAV8

Recombinant adeno-associated virus serotype 8 (rAAV8) is a viral vector platform extensively utilized in gene therapy for the delivery of therapeutic genetic material to target tissues. Derived from the parvovirus family, rAAV8 is particularly noted for its strong tropism for the liver (hepatotropism) and skeletal muscle, making it a leading candidate for treating hepatic-based genetic disorders such as hemophilia and various metabolic deficiencies. rAAV8 vectors are engineered to be replication-deficient, typically replacing viral genes with a transgene expression cassette flanked by inverted terminal repeats (ITRs). While highly effective for in vivo gene transfer, the clinical application of rAAV8 faces challenges including preexisting humoral and cellular immunity in the human population. Memory T cells or neutralizing antibodies against the AAV8 capsid can trigger inflammatory responses, such as liver inflammation, or lead to the loss of transgene expression, particularly at high systemic doses.

Other names

recombinant adeno-associated virus 8AAV8 vectorAAV-8 vectorAAV 8 vectorAAV serotype 8