Figure 4.
Immune response in treated offspring. Late gestation IUGT with scAAV2/8-LP1-hFIXco resulted in a robust immune response against the viral capsid 8-002 which gradually declined over time. In comparison IUGT with scAAV2/5-LP1-hFIXco resulted in more robust and prolonged immune responses. AAV, adeno-associated viral vector; hFIX, human Factor IX; IUGT, intrauterine gene transfer.