Figure 4
Figure 4. Transduction of rhesus macaques following peripheral vein administration of scAAV2/5. (A) Human FIX concentration in rhesus plasma was determined at the indicated time points after peripheral vein administration of 1 × 1012 vg/kg scAAV vector pseudotyped with serotype 5 capsid (M3-sc, ⋄; M8-sc, □; M9-sc, ○) into 3 rhesus macaques with moderate to high titers of anti-AAV8 antibodies. (B) Human FIX expression in rhesus plasma was determined at the indicated time points after mesenteric vein administration of 1 × 1012 vg/kg scAAV vector pseudotyped with serotype 5 capsid (328, ▿) of a macaque with high titers of anti-AAV8 antibody. Extended expression profile of a macaque previously transduced with scAAV2/5-LP1-hFIXco (M4-sc, □) is shown for comparison. Each sample was independently evaluated on at least 3 separate occasions, and the results are depicted as an average together with the standard error of the mean.

Transduction of rhesus macaques following peripheral vein administration of scAAV2/5. (A) Human FIX concentration in rhesus plasma was determined at the indicated time points after peripheral vein administration of 1 × 1012 vg/kg scAAV vector pseudotyped with serotype 5 capsid (M3-sc, ⋄; M8-sc, □; M9-sc, ○) into 3 rhesus macaques with moderate to high titers of anti-AAV8 antibodies. (B) Human FIX expression in rhesus plasma was determined at the indicated time points after mesenteric vein administration of 1 × 1012 vg/kg scAAV vector pseudotyped with serotype 5 capsid (328, ▿) of a macaque with high titers of anti-AAV8 antibody. Extended expression profile of a macaque previously transduced with scAAV2/5-LP1-hFIXco (M4-sc, □) is shown for comparison. Each sample was independently evaluated on at least 3 separate occasions, and the results are depicted as an average together with the standard error of the mean.

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