Figure 7.
Figure 7. A FVIII B-domain variant and a FVIII A1-domain/B-domain variant hybrid are expressed more efficiently in vivo in FVIII-/- mice. A FVIII B-domain variant and a FVIII A1-domain/B-domain variant hybrid were transiently expressed in vivo following a hydrodynamic tail-vein injection method into FVIII-/- mice. Plasmid DNA (100 μg) containing the FVIII variants diluted in 2.5 mL lactated Ringer solution was injected as a bolus over 10 seconds into the tail vein. Expression of the FVIII variants was analyzed from mouse plasma harvested by retro-orbital blood sampling at 24 hours (□) and 48 hours (▪) after injection. FVIII activity was determined by a 2-stage chromogenic assay and compared with mice injected similarly with BDD-FVIII as a control. Average expression of BDD-FVIII was 0.62 U/mL at 24 hours and 0.62 U/mL at 48 hours. Data presented are the mean of several independent experiments (n ≥ 3), and the error bars represent the standard deviation.

A FVIII B-domain variant and a FVIII A1-domain/B-domain variant hybrid are expressed more efficiently in vivo in FVIII-/- mice. A FVIII B-domain variant and a FVIII A1-domain/B-domain variant hybrid were transiently expressed in vivo following a hydrodynamic tail-vein injection method into FVIII-/- mice. Plasmid DNA (100 μg) containing the FVIII variants diluted in 2.5 mL lactated Ringer solution was injected as a bolus over 10 seconds into the tail vein. Expression of the FVIII variants was analyzed from mouse plasma harvested by retro-orbital blood sampling at 24 hours (□) and 48 hours (▪) after injection. FVIII activity was determined by a 2-stage chromogenic assay and compared with mice injected similarly with BDD-FVIII as a control. Average expression of BDD-FVIII was 0.62 U/mL at 24 hours and 0.62 U/mL at 48 hours. Data presented are the mean of several independent experiments (n ≥ 3), and the error bars represent the standard deviation.

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