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Fig. 2 | Thrombosis Journal

Fig. 2

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

Fig. 2

Comparison of hFVIII levels in plasma and/or platelets after a single IO infusion of E-F8-LVs and G-F8-LVs. a HemA mice were intraosseously infused with E-F8-LV (5 × 107 ifu/animal, n = 4) or PBS (20 μl/animal, mock, n = 3) on day 0. Plasma samples were collected and hFVIII activity and anti-FVIII antibodies were measured by aPTT and Bethesda assay, respectively. No FVIII activity or anti-FVIII antibody was detected in the PBS treated control mice (data not shown). b-d HemA mice were given IO infusion of G-F8-LV (2.2 × 107 ifu/animal or 2.2 × 106 ifu/animal) or PBS (20 μl/animal, mock) on day 0. b Platelets were isolated from peripheral blood of high (n = 8) or low (n = 5) titer G-F8-LV treated or mock (n = 3) mice. hFVIII expression levels in CD42d+ platelets were evaluated by flow cytometry on day 27, 62, 84, 112 and 160. c HemA phenotype correction of G-F8-LV treated mice was monitored by tail clip assay on day 35, 118, and 160 (n = 4–7/group). The average blood loss of untreated HemA mice was set as 100 %. Wild-type C57BL/6 mice were used as positive controls. * P < 0.05. d Plasma samples were collected from high titer G-F8-LV treated (n = 10) or mock (n = 3) mice, and hFVIII activity and anti-FVIII antibodies were measured by aPTT and Bethesda assay, respectively. This figure is reproduced from Ref [10]

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