Fig. 2: Phenotype of 3-week-old untreated and NB-301H-dosed huR83C mice.

Newborn (NB) huR83C mice, non-fasted, were treated with a high dose of BEAM-301 (301H) at 1.5 mg/kg. At age 3 weeks, the phenotype of the resulting NB-301H mice (NB-301H-3W) was evaluated and compared to age-matched unedited huR83C (huR83C-3W), wild-type (mR83-3W), and heterozygote (mR83/huR83C-3W) mice. a Liver and kidney microsomal G6Pase-α activity. Liver (mR83-3W, n = 5; mR83/huR83C-3W, n = 11; huR83C-3W, n = 8; NB-301H-3W, n = 6) and kidney (mR83-3W, n = 6; mR83/huR83C-3W, n = 10; huR83C-3W, n = 8; NB-301H-3W, n = 6). b Restoration of hepatic G6Pase-α activity as a function of base editing efficiency in NB-301H-3W mice (n = 6) along with on-target and bystander values of liver base editing. c Fasting blood glucose levels in control (mR83-3W and mR83/huR83C-3W; n = 5) and NB-301H-3W (n = 5) mice. d Histochemical analysis of liver and kidney G6Pase-α activity in control (mR83-3W and mR83/huR83C-3W, n = 6), untreated (huR83C-3W, n = 6), and NB-301H-3W (n = 6) mice. Each image represents an individual mouse. The arrow indicates the kidney cortex. Scale bar = 100 µm. The numbers represent hepatic G6Pase-α activity expressed in the mice. e Body weight (BW), liver weight (LW)/BW, and kidney weight (KW)/BW values of control (mR83-3W and mR83/huR83C-3W, n = 25), untreated (huR83C-3W, n = 17), and NB-301H-3W (n = 6) mice. f Size comparison of mR83 and huR83C mice, showing growth retardation of the huR83C mice at age 3 weeks. g Restoration of hepatic G6Pase-α activity as a function of LW/BW values and hepatic levels of glycogen, triglyceride, and G6P in the NB-301H-3W mice (n = 6). 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.