Fig. 4: Phenotypic analysis of 301H-dosed huR83C mice at age 8 weeks.

Newborn (NB) and 3-week-old (3W) huR83C mice, non-fasted, were treated with 301H (BEAM-301 at 1.5 mg/kg) and the phenotype of the resulting NB-301H-8W and 3W-301H-8W mice was analyzed at age 8 weeks. The sex-matched mR83-8W and mR83/huR83C-8W littermates displaying a wild-type phenotype were used as the controls. a Liver and kidney microsomal G6Pase-α activity in mR83-8W (n = 8), mR83/huR83C-8W (n = 9), NB-301H-8W (n = 8), and 3W-301H-8W (n = 9) mice. b Restoration of hepatic G6Pase-α activity as a function of base editing efficiency along with on-target and bystander values of liver base editing. NB-301H-8W (n = 8); 3W-301H-8W (n = 9). c Fasting blood glucose levels in control (n = 23), NB-301H-8W (n = 8), and 3W-301H-8W (n = 9) mice. d BW, LW/BW, and KW/BW values of control (n = 23), NB-301H-8W (n = 8), and 3W-301H-8W (n = 9) mice. e Restoration of hepatic G6Pase-α activity as a function of LW/BW values and hepatic levels of glycogen and G6P in the 301H-8W mice (n = 17), including NB-301H-8W (n = 8) and 3W-301H-8W (N = 9) mice. f Blood glucose and serum cholesterol, triglyceride, lactate, and uric acid levels in control (n = 17), NB-301H-8W (n = 8), and 3W-301H-8W (n = 9) mice. g Liver glucose, glycogen, triglyceride, lactate, and G6P levels in control (n = 17), NB-301H-8W (n = 8), and 3W-301H-8W (n = 9) mice. Statistics were performed using a two-tailed unpaired T test. Data are presented as Mean values ± SEM, and individual data points for each animal are displayed. * denotes p < 0.05, ** denotes p value < 0.005.