Figure 6.

Systemic Treatment with AAV9-Bic Vector: Maximum Effective Treatment Age and Minimum Effective Dose

(A) SD mice were treated with 1 × 10¹² vg AAV9-Bic at 8 (n = 10), 10 (n = 9), and 12 weeks (n = 8) of age and survival followed until the humane endpoint. (B) Dose escalation study in 4-week-old SD mice treated at doses of 1 × 10¹¹ (n = 11), 3 × 10¹¹ (n = 8), and 1 × 10¹² vg (n = 4). Kaplan-Meier survival curves are shown for treatment groups compared to a common group of untreated (UT) SD mice (n = 6). Log-rank (Mantel-Cox) test was used to compare survival of treatment groups to that of untreated SD mice: ∗∗p < 0.01; ∗∗∗∗p < 0.0001.