Figure 2.

Intraperitoneal implantation of BOECs with Cytodex microcarrier beads

(A) Kinetics of the percentage of FVIII activity measured by aPTT assay in the plasma of transplanted NSG-HA mice. BOECs used were transduced only with LV-VEC.GFP or with both LV-VEC.hBDD-FVIII and LV-VEC.GFP. Data are expressed as mean ± SD and are representative of two independent experiments using BOECs from two healthy donors (n = 7 mice), and four independent experiments using HA BOECs from four patients (n = 23 mice). (B) Blood loss evaluation on NSG-HA mice between weeks 7 and 10 (n = 4) after cell transplantation. (C) FVIII concentration in plasma of mice transplanted with transduced or non-transduced BOECs at week 16. Data are expressed as mean ± SD (∗∗∗p < 0.0001, ∗∗p < 0.001). (D) Representative immunofluorescence on beads showing cells co-expressing GFP and CD31.