Figure 3
Figure 3. FVIII activity and inhibitor titers after intraperitoneal or subcutaneous administration of lentiviral vectors to neonatal hemophilia A mice. Neonatal BALB/c hemophilia A mice were treated with 2 × 107 IU of Lenti-HCR/hAAT-cFVIII intraperitoneally (A-B) or subcutaneously (C-D). (A,C) Plasma levels of FVIII in mice treated via these delivery routes and (B,D) levels of neutralizing anti-cFVIII antibodies as measured by a Bethesda assay (note the different inhibitor axes). Five of the mice that were injected subcutaneously with Lenti-HCR/hAAT-cFVIII and which developed neutralizing anti-cFVIII antibodies that ranged between 23 and 95 BU were used as positive controls for inhibitor development.

FVIII activity and inhibitor titers after intraperitoneal or subcutaneous administration of lentiviral vectors to neonatal hemophilia A mice. Neonatal BALB/c hemophilia A mice were treated with 2 × 107 IU of Lenti-HCR/hAAT-cFVIII intraperitoneally (A-B) or subcutaneously (C-D). (A,C) Plasma levels of FVIII in mice treated via these delivery routes and (B,D) levels of neutralizing anti-cFVIII antibodies as measured by a Bethesda assay (note the different inhibitor axes). Five of the mice that were injected subcutaneously with Lenti-HCR/hAAT-cFVIII and which developed neutralizing anti-cFVIII antibodies that ranged between 23 and 95 BU were used as positive controls for inhibitor development.

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