Figure 1.

Gene delivery to humanized chimeric mouse livers

(A) Livers from liver-humanized FRG mice were stained to show human (red, anti-human albumin [hAlb]) and mouse (green, anti-mouse albumin [mAlb]) hepatocytes. (B) GFP expression in humanized livers of FRG mice 14 days after retro-orbital delivery of scAAVLK03-smCBA-GFP vector at a dose of 5 × 10¹⁰ vector genomes/mouse. Livers were stained using an anti-GFP antibody. (C) In situ GFP expression in whole livers at necropsy detected using an AMG EVOS fluorescence microscope 14 days after retro-orbital administration of a low (5 × 10¹⁰ vector genomes) or high (2 × 10¹¹ vector genomes) dose of scAAVLK03-smCBA-GFP. (D) Co-labeling with anti-GFP (green) and anti-human albumin (red) antibodies in humanized livers from a FRG mouse treated with 2 × 10¹¹ vector genomes of scAAVLK03-smCBA-GFP. (E) Liver gene delivery in liver-humanized Alb-uPA/scid mice. Liver tissue was harvested from Alb-uPA/scid mice 14 days after intravenous retro-orbital delivery of scAAV-LK03-smCBA-GFP vector at a dose of 5 × 10¹⁰ vector genomes/mouse. Images show human albumin (red, hAlb) and GFP (green, anti-GFP).