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Fig. 3 | Thrombosis Journal

Fig. 3

From: Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors

Fig. 3

hFVIII expression in platelets of G-F8-LV treated inhibitor HemA mice corrected their hemophilia A phenotype. Inhibitor HemA mice were established by repeated intraperitoneal injection (3×/week for 2 weeks) of 3U rhFVIII into 10- to 12-week-old HemA mice. These inhibitor HemA mice were then intraosseously infused with G-F8-LV (2.2 × 107 ifu/animal) or PBS (20 μl/animal, mock) on day 0. a Platelets were isolated from peripheral blood and marked with CD42d+, and their GFP expression levels at 5 months post infusion. b Platelets from LV-treated (n = 5) and mock (n = 3) mice and lysed. The resulting lysate was examined for hFVIII expression level by ELISA on day 27 post infusion. c The phenotypic correction of G-F8-LV treated HemA inhibitor mice (n = 7) was examined by tail clip assays on day 160 post infusion. The average blood loss of untreated HemA (n = 10) mice was set as 100 %. Wild-type C57BL/6 mice (n = 8) were used as positive controls. * P < 0.05, ** P < 0.005. This figure is reproduced from Ref [10]

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