Figure 3.
Increase of hFIX-vector DNA in liver following IA. Levels of hSEAP- and hFIX-vector DNA were monitored by qPCR in the liver tissue at euthanization. NHPs received a primary administration of rAAV5-hSEAP (day 0) and a second administration of rAAV5-hFIX (day 49) with or without pretreatment with IA (IA). Per animal, 30 different liver samples were analyzed. The hSEAP- (A) and hFIX-vector (B) DNA levels are plotted per animal with indication of the medians. (C-D) Medians and range per group are reported. Although animal groups with or without an IA procedure had similar levels of hSEAP-vector DNA, an increase of 27.86 times in genome copy number of hFIX-vector DNA was measured in the animal group that underwent the IA. The levels of hFIX-vector DNA measured in the control group, primary administered with rAAV5-hFIX at the same dose of 3 × 1013 gc/kg, were 4.62 times higher than the group that underwent IA.