Fig. 3: A single injection of Cbh-ABE9-dual-AAV9 has long-term effect in Pkd1RC/RC mice.
From: In vivo base editing rescues ADPKD in a humanized mouse model
a Representative histological images of six-month old kidneys from Pkd1RC/RC mice treated with 1 × 1011 or 1 × 1012 vg of Cbh-ABE9-dual-AAV9 at PN14. Scale bar, 1 mm. b Percent cystic area relative to total kidney area of kidneys from Pkd1RC/RC mice treated with 1 × 1011 or 1 × 1012 vg of Cbh-ABE9-dual-AAV9 at PN14 (n = 10 independent replicates). c, d Treatment with 1 × 1011 or 1 × 1012 vg of Cbh-ABE9-dual-AAV9 at PN14 decreased KW/BW ratios (c) and BUN levels (d) in six-month old Pkd1RC/RC mice. (n = 10, equal numbers of male and female mice, the colors represent female (pink) and male mice (blue)). e Representative images of hearts from six-month old Pkd1RC/RC mice injected with 1 × 1011 or 1 × 1012 of Cbh-ABE9-dual-AAV9 at PN14. Scale bar, 1 mm. f Treatment with 1 × 1012 vg of Cbh-ABE9-dual-AAV9 at PN14 decreased HW/BW ratios in six-month-old Pkd1RC/RC mice compared to vehicle-treated controls (n = 10 independent replicates). g, h Western blot analysis of PC1 protein in six-month-old wide type and age-matched Pkd1RC/RC kidneys and heart treated with 1 × 1012 vg of Cbh-ABE9-dual-AAV9 or vehicle at PN14 (n = 3 independent replicates). i the editing efficiency (%) of the targeted pathogenic variant R3277C in kidneys, hearts and livers from three and six-month-old Pkd1RC/RC mice injected with a single dose of dual-AAV9-ABE9 (1 × 1012 vg) at PN14 (n = 3 independent replicates). Values and error bars reflect mean ± SD from indicated counts of independent experiments, ns: p > 0.05, *p  <  0.05, **p  <  0.01, ***p  <  0.001, and ****p  <  0.0001 or as indicated in the figure, using two-tailed t-test. Source data are provided as a Source Data file.
