Browsing by keyword "rAAV7"
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Liver targeting with rAAV7: balancing tropism with immune profilesThe value of intravenous (IV) recombinant adeno-associated virus (rAAV) as a platform for delivery of transgenes to the liver for gene therapy has been well established. The most notable example is the use of a self-complementary rAAV9 vector expressing a high specific activity Factor IX gene in patients with hemophilia B. Since the liver is the primary site for expression of numerous other serum proteins and biochemical pathways, this platform has the potential for very broad utility in a wide variety of genetic diseases. Thus, the optimization of the delivery of rAAV to hepatocytes is of central importance in the field of human gene therapy.
