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. Author manuscript; available in PMC: 2012 Jun 22.
Published in final edited form as: N Engl J Med. 2011 Dec 10;365(25):2357–2365. doi: 10.1056/NEJMoa1108046

Figure 1. Factor IX (FIX) Activity after Peripheral-Vein Infusion of the Adenovirus-Associated Virus (AAV) Vector in the Six Study Participants.

Figure 1

A one-stage clotting assay was used to determine FIX coagulation activity at prespecified time points (arrows) after the administration of the AAV vector (scAAV2/8-LP1-hFIXco). Panels A and B show FIX levels in Participants 1 and 2, respectively, who received the low dose of vector (2×1011 vector genomes [vg] per kilogram of body weight). Panels C and D show FIX levels in Participants 3 and 4, respectively, who received the intermediate dose of vector (6×1011 vg per kilogram). Panels E and F show both FIX levels and alanine aminotransferase (ALT) levels in Participants 5 and 6, respectively, who received the high dose of vector (2×1012 vg per kilogram). The duration of treatment with prednisolone (Pred), which was initiated at a dose of 60 mg per day and then gradually tapered, is also shown.