Fig. 3: Biochemistry 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, biochemical phenotype of the edited mice was analyzed and compared to the age matched unedited huR83C. The 3-week-old mR83 and mR83/huR83C littermates displaying a wild-type phenotype were used as the controls. a Blood glucose levels (control-3W, n = 24; huR83C-3W, n = 16; NB-301H-3W, n = 6), serum cholesterol levels (control-3W, n = 16; huR83C-3W, n = 7; NB-301H-3W, n = 6), serum triglyceride levels (control-3W, n = 24; huR83C-3W, n = 16; NB-301H-3W, n = 6), serum lactate and uric acid levels (control-3W, n = 16; huR83C-3W, n = 8; NB-301H-3W, n = 6). b Liver glucose, triglyceride, lactate, glycogen, and G6P levels in control (n = 16), huR83C-3W (n = 8), and NB-301H-3W (n = 6) mice. c Hematoxylin and eosin (H&E)-staining of liver and kidney sections in mR83/huR83C-3W (n = 6), huR83C-3W (n = 6), and NB-301H-3W (n = 6) mice. The liver and kidney in all experimental animals were examined. A single image from an individual mouse, representative of the results in all mice, is shown to illustrate the results. Scale bar = 20 µm. The numbers represent hepatic G6Pase-α activity expressed in the mice. d Oil Red O staining of liver and kidney sections in mR83/huR83C-3W (n = 6), huR83C-3W (n = 6), and NB-301H-3W (n = 6) mice. The liver and kidney in all experimental animals were examined. A single image from an individual mouse, representative of the results in all mice, is shown to illustrate the results. Scale bar = 20 μm. The numbers represent hepatic G6Pase-α activity expressed in the 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.