Figure 4.

AAV-mediated expression of codon-optimized hFVII in monkeys. (A) Human FVII levels over time in male (N = 3, dotted line) and female (N = 3, solid line) adult monkeys following a single peripheral vein administration of 4 × 10¹¹ vg/kg scAAV5-LP1-hFVII-coop. (B) Southern blot of DNA extracted from male and female rhesus monkey liver at 4 weeks after administration of scAAV5-LP1-hFVII-coop following digestion with BsRDI (double cutter) and probed with a hFVII-coop probe. As shown in the schematic, the probe is designed to detect the 1377- and 942-bp fragments expected from digest of head-to-tail concatemers.