Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice
J. Clin. Invest. Antonia Follenzi, et al. 118:935 doi:10.1172/JCI32748 [
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Figure 6Therapeutic efficacy of transplanted LSECs in NOD/SCID hemophilia A mice. (
A) RT-PCR analysis of total liver RNA showing FVIII mRNA in virtually all treated mice (lanes 2–13). The samples were analyzed for β-actin in parallel to verify RNA integrity. Lane 1, negative control with PCR mix alone; lane 14, healthy mouse liver RNA as positive control. (
B) Immunostaining of liver showing transplanted FVB/N-Tie2–GFP LSECs in NOD/SCID hemophilia A mouse (green) with coexpression of CD31 endothelial marker (red in native cells; yellow in transplanted cells). (
C) GFP-positive transplanted cells (green) distinct from F4/80-expressing Kupffer cells (red). (
D) Plasma FVIII activity in NOD/SCID hemophilia A mice 1 month after transplantation of FVB/N-Tie2–GFP LSECs with therapeutic correction requiring more than 10% plasma FVIII. Original magnification, ×200 (
B and
C).
