Fig. 5.

Whole-body efficacies after systemic miniagrin gene delivery. (a) Growth curves of the untreated dy^w/dy^w mice (•), AAV-treated dy^w/dy^w mice (gray triangle), and WT littermate control mice (gray diamond). Most of the untreated dy^w/dy^w mice died by 9 weeks. (b) Whole-body photograph of the 1 ^½-month-old WT control (Top), the AAV-treated (Middle), and untreated (Bottom) dy^w/dy^w mice. (c) Rotarod assays performed with 4-week-old mice. Animals were first trained three times to allow for accommodation to the task. After resting for at least 1 h, animals were tested on the machine. Each data point represents mean ± SD from four trials of each mouse (n = 4). (d) Survival rate of the mice. Fifty percent survival rate of the untreated dy^w/dy^w mice is 4 weeks (n = 18, •), the AAV-treated dy^w/dy^w mice is 17 weeks (n = 7, ▴), and the normal life span of the WT mice is >1 year (▪).