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Figure 2. Phenotypic correction of hemophilia A mice by platelet-targeting gene delivery. A) Blood from FVIII-deficient mice transplanted with KSL cells transduced with SIV-GPIb
-hFVIII was stimulated without or with 50 µg/ml of collagen and 1 µM PMA for 15 min. After centrifugation, platelet-poor plasma was obtained, and hFVIII antigen levels were measured by ELISA. Columns and error bars are mean ± SD (n=4 per group). B) Mortality rate within 24 h after tail clipping in mice transplanted with KSL cells transduced with control or SIV-GPIb
-hFVIII (n=10 for control; n=8 for GPIb
). Mortality rate was statistically evaluated by a
2 test.