Figure 3.

Large-scale expansion of HA patient-derived BOECs

(A) Light microscope pictures of transduced HA BOECs pre- and post-expansion. (B) Cell size, cell density, culture time, and population doubling level during pre- and post-large-scale expansion. (C) Endothelial marker expression pre- and post-large-scale expansion expressed as stained cells versus cells with secondary isotype controls. (D) Tubulogenic assay to assess the functionality of transduced HA BOECs after pre- and post-large-scale expansion. (E) Kinetics of the percentage of FVIII activity measured by aPTT assay in plasma of transplanted NSG-HA mice. Data are expressed as mean ± SD and are representative of two independent experiments (n = 7).