Figure 5. Genome editing with promoterless mini-ATP7B reduces copper levels and improves liver disease in Atp7b^tx-J mice.

Atp7b^tx-J mice (4 weeks old) and WT or heterozygous (Het) healthy control mice were injected with 1 × 10¹⁴ vg/kg AAV-DJ-Alb-ATP7BΔ1-4 (ATP7BΔ1-4) or vehicle and sacrificed at the age of 42 weeks (n = 4 per group). (A) Representative photographs of livers at sacrifice. (B) Representative images from H&E and human ATP7B histochemical staining. Scale bars: 200 μm and 100 μm (high-magnification insets). (C) Serum ALT levels. Copper content by ICP-MS in (D) liver and (E) urine. For 1-way ANOVA plus Tukey’s post hoc test, *P < 0.05; **P < 0.01; ****P < 0.001.