Fig. 6: Phenotypic analysis of NB-301H-dosed huR83C mice at age 53 weeks. | Nature Communications

Fig. 6: Phenotypic analysis of NB-301H-dosed huR83C mice at age 53 weeks.

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

Fig. 6

Newborn (NB) huR83C mice, non-fasted, were treated with 301H (BEAM-301 at 1.5 mg/kg) and the phenotype of the NB-301H-dosed mice (NB-301H-53W, n = 19) was evaluated at 53 weeks of age using sex-matched wild-type littermates (mR83-53W, n = 16) as the controls. a Liver microsomal G6Pase-α activity. 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. c Fasting blood glucose levels. d BW, LW/BW, and KW/BW values. e Restoration of hepatic G6Pase-α activity as a function of LW/BW values and hepatic levels of glycogen and G6P in the edited mice. f Blood glucose and serum cholesterol, triglyceride, lactate, and uric acid levels. g Liver glucose, glycogen, triglyceride, lactate, and G6P levels. 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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