Fig. 5: Phenotypic analysis of 301L-dosed huR83C mice at age 8 weeks. | Nature Communications

Fig. 5: Phenotypic analysis of 301L-dosed huR83C mice at age 8 weeks.

From: Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia

Fig. 5

Newborn (NB) and 3-week-old (3W) huR83C mice, non-fasted, were treated with a low dose of BEAM-301 (301L) at 0.75 mg/kg and the phenotype of the resulting NB-301L-8W and 3W-301L-8W mice was analyzed at age 8 weeks. The sex-matched mR83 and mR83/hR83C littermates displaying a wild-type phenotype were used as the controls. a Liver microsomal G6Pase-α activity in control (n = 17), NB-301L-8W (n = 9), and 3W-301L-8W (n = 8) 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 in NB-301L-8W (n = 9) and 3W-301L-8W (n = 8) mice. c Fasting blood glucose levels in control (n = 17), NB-301L-8W (n = 9), and 3W-301L-8W (n = 8) mice. d BW, LW/BW, and KW/BW values of control (n = 23), NB-301L-8W (n = 9), and 3W-301L-8W (n = 8) mice. e Restoration of hepatic G6Pase-α activity as a function of LW/BW values and hepatic levels of glycogen and G6P in the edited 301L-8W (n = 17) mice, including NB-301L-8W (n = 9) and 3W-301L-8W (n = 8) mice. f Blood glucose and serum cholesterol, triglyceride, lactate, and uric acid levels in control (n = 17), NB-301L-8W (n = 9), and 3W-301L-8W (n = 8) mice. g Liver glucose, glycogen, triglyceride, lactate, and G6P levels in control (n = 17), NB-301L-8W (n = 9), and 3W-301L-8W (n = 8) 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.

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