Extended Data Fig. 7: No gross toxicity was observed in mice treated with rAAV9.2xsup-tRNATyr.
From: AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
a, Representative H&E staining images of liver, brain, spleen, and kidney sections in untreated (n = 3) or sup-tRNATyr-treated (n = 4) male KI/+ mice. Mice were treated at 6 weeks old, and euthanized at 16 weeks old. Scale bar = 200 µm. b, Blind pathological assessment of the liver, brain, spleen, and kidney H&E slides as described in (a). c, Summary of endpoint serum clinical biochemistry of untreated (n = 3) or sup-tRNATyr-treated (n = 4) KI/+ mice. Data are mean ± s.d. of biological replicates. Statistical analysis was performed by two-sided Student t-test. *p < 0.05, **p < 0.01. Note that for the endpoints showing statistical significance, values of both groups are within the normal ranges (https://phenome.jax.org/projects/CGDpheno2).
