Fig. 2.
Fig. 2. Levels of cFIX expression and anti-cFIX IgG antibody after transduction of normal BALB/c mice. / (A) Levels of cFIX in mice transduced as neonates. Neonatal normal BALB/c mice were injected intravenously with a single dose of 1 × 1010 TU/kg hAAT-cFIX–WPRE at 2 or 3 days after birth. Plasma cFIX levels were determined by immunoassay at the indicated time after transduction, and the average at each point ± standard error of the mean (SEM) is shown. The number of animals evaluated (N) is shown. The normal range of cFIX (3 to 8 μg/mL) is indicated by a gray bar. (B) Levels of cFIX in mice transduced as adults with preceding HGF. Adult BALB/c mice were injected intravenously with RV after administration of HGF as diagrammed in Figure 1B, and plasma cFIX antigen levels were determined. Values for individual mice are shown. (C) Levels of cFIX in mice transduced as adults without preceding HGF. Adult BALB/c mice were injected with RV after administration of PBS as diagrammed in Figure 1B, and plasma cFIX antigen levels were determined. Values for individual mice are shown. (D) Anti-cFIX IgG antibody levels in mice transduced as neonates. Plasma from the mice whose cFIX levels are shown in panel A was tested for anti-cFIX–specific antibodies at the indicated times after transduction as detailed in “Materials and methods.” The antibody levels are presented as the specific OD observed for a sample that was captured with cFIX minus the nonspecific signal for that sample, times the dilution of the plasma sample that was used. None of the animals had detectable antibodies. (E) Anti-cFIX IgG antibodies in mice transduced as adults after preceding HGF. Plasma from the mice whose cFIX levels are shown in panel B was tested for anti-cFIX–specific antibodies. Individual animals have the same symbol in both panels. (F) Anti-cFIX IgG antibodies in mice transduced as adults without preceding HGF. Plasma from the mice whose cFIX levels are shown in panel C was tested for anti-cFIX–specific antibodies. Individual animals have the same symbol in both panels.

Levels of cFIX expression and anti-cFIX IgG antibody after transduction of normal BALB/c mice.

(A) Levels of cFIX in mice transduced as neonates. Neonatal normal BALB/c mice were injected intravenously with a single dose of 1 × 1010 TU/kg hAAT-cFIX–WPRE at 2 or 3 days after birth. Plasma cFIX levels were determined by immunoassay at the indicated time after transduction, and the average at each point ± standard error of the mean (SEM) is shown. The number of animals evaluated (N) is shown. The normal range of cFIX (3 to 8 μg/mL) is indicated by a gray bar. (B) Levels of cFIX in mice transduced as adults with preceding HGF. Adult BALB/c mice were injected intravenously with RV after administration of HGF as diagrammed in Figure 1B, and plasma cFIX antigen levels were determined. Values for individual mice are shown. (C) Levels of cFIX in mice transduced as adults without preceding HGF. Adult BALB/c mice were injected with RV after administration of PBS as diagrammed in Figure 1B, and plasma cFIX antigen levels were determined. Values for individual mice are shown. (D) Anti-cFIX IgG antibody levels in mice transduced as neonates. Plasma from the mice whose cFIX levels are shown in panel A was tested for anti-cFIX–specific antibodies at the indicated times after transduction as detailed in “Materials and methods.” The antibody levels are presented as the specific OD observed for a sample that was captured with cFIX minus the nonspecific signal for that sample, times the dilution of the plasma sample that was used. None of the animals had detectable antibodies. (E) Anti-cFIX IgG antibodies in mice transduced as adults after preceding HGF. Plasma from the mice whose cFIX levels are shown in panel B was tested for anti-cFIX–specific antibodies. Individual animals have the same symbol in both panels. (F) Anti-cFIX IgG antibodies in mice transduced as adults without preceding HGF. Plasma from the mice whose cFIX levels are shown in panel C was tested for anti-cFIX–specific antibodies. Individual animals have the same symbol in both panels.

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