Fig. 3.

Therapeutic efficacy of CRISPR/Cas9-mediated STR in PH1 animals. a Schematic experimental procedure, where 8–10-week-old PH1 animals were intravenously treated with saline (n = 7), Cas9 (n = 6), Hao1-g1 (n = 6) and Hao1-g2 (n = 5). A 7-days EG challenge was performed 4 months after vector administration, and 24 h urine samples were collected before and on days 3 and 7 of challenge. b Quantification of basal urine levels of oxalate and glycolate (µmol/24 h) 4 months after treatment. c, d Quantification of urine oxalate (c) and glycolate (d) levels (µmol/24 h) before and on days 3 and 7 of EG challenge. Data are presented as mean ± SEM and Kruskal–Wallis statistical test was used to compare the groups in each day. e Weight change of the animals during a one-week EG challenge. f Representative histological analysis of CaOx accumulation in the kidneys of PH1 animals from control or treatment groups after a 10-days EG challenge. Scale bar: 200 μm. *p < 0.05; **p < 0.01; ***p < 0.001