Fig. 3
From: Gene therapy for ultrarare diseases: a geneticist’s perspective
Mechanism of systemic AAV vector delivery to the liver. After intravenous infusion, the majority of AAV vectors are taken up by the liver. The transduced hepatocytes restore their functions, such as by conducting an enzyme reaction. If the transgene encodes a secretory protein, the protein can be released into the circulation to treat systemic diseases. The secretary protein, if it contains a brain-targeting epitope, can enter the brain