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. 2023 Jul 22;31(9):2767–2782. doi: 10.1016/j.ymthe.2023.07.013

Figure 2.

Figure 2

ICV Delivery of scAAV9.P546.MECP2 does not affect survival or behavioral phenotypes in WT mice

(A) Survival in WT mice treated with any vector dose is not significantly different from survival in untreated WT mice (p = 0.1525) (log-rank/Mantel Cox test). (B) Severity score of untreated WT- and vector-treated WT mice shows that treatment overwhelmingly does not affect score. (C) At 60 days, vector-treated WT mice do not have statistically different severity scores vs. untreated WT. p values: untreated KO, p < 0.0001; WT 1.50 × 109, p > 0.9999; WT 3.75 × 109, p = 0.9992; WT 7.50 × 109, p > 0.9999; WT 1.50 × 1010, p = 0.9512; WT 3.00 × 1010, p = 0.9876; WT 6.00 × 1010, p > 0.9999. (D) At 90 days, vector-treated WT mice do not have statistically different severity scores vs. WT, except 3.00 × 1010 vg. p values: untreated KO, p < 0.0001; 1.50 × 109, p > 0.9999; 3.75 × 109, p = 0.9911; 7.50 × 109, p > 0.8146; 1.50 × 1010, p = 0.9983; 3.00 × 1010, p = 0.0442; 6.00 × 1010, p > 0.4566. (E and F) Open field assay for distance and velocity was performed at 49–63 days. (E) Vector-treated WT mice do not have a significantly different open field velocity compared with untreated WT mice. p values vs. untreated WT: untreated KO, p < 0.0001; 1.50 × 109, p > 0.9999; 3.75 × 109, p > 0.9999; 7.50 × 109, p = 0.9959; 1.50 × 1010, p = 0.9991; 3.00 × 1010, p > 9999; 6.00 × 1010, p > 0.9999. (F) Vector-treated WT mice do not have a significantly different open field distance compared with untreated WT mice. p values vs. untreated WT: untreated KO, p = 0.0037; 1.5 × 109, p > 0.9999; 3.75 × 109, p > 0.9999; 7.5 × 109, p = 0.4199; 1.5 × 1010, p = 0.9998; 3.0 × 1010, p = 9976; 6.0 × 1010, p = 0.7980. 60 and 90-day average severity scores were taken ±2–4 days to account for slight time point variability in biweekly scoring intervals. WT untreated, n = 40; KO untreated, n = 43; WT- 1.50 × 109, n = 11; WT- 3.75 × 109, n = 32; WT-7.50 × 109, n = 16; WT- 1.50 × 1010, n = 36; WT- 3.00 × 1010, n = 20; WT- 6.00 × 1010, = 18. Statistical significance was determined via ANOVA with Tukey’s test. Significance is in relation to untreated WT mice. ∗p < 0.05; ∗∗∗p < 0.001; ∗∗∗∗p < 0.000. ns, not significant.