Genome Editing Improves Liver Phenotype
Z-AAT serum levels are decreased, as shown in (A) by ELISA, normalized to the average of the age-matched control group and presented as percentage of silencing. *Statistically significant differences between treated and control groups at a given time point (unpaired t test); **statistically significant differences between treatment groups (two-way ANOVA). Liver phenotype was further analyzed at 6 and 8 months post-treatment in the P1-injected group, by (B) PASD immunohistochemistry at 6 and 8 months, quantified by threshold analysis. ****Statistically significant differences between groups (two-way ANOVA). (C) Picro Sirius immunohistochemistry at 8 months, quantified by threshold analysis; (D and E) representative animals for the PASD staining (D, control; E, treated); (F and G) representative animals for the PSR staining (F, control; G, treated). For all graphs, data are represented as mean ± SEM.