Figure 4.

AAV9-U1-FD rescues FD symptoms in FD mice

(A) The righting reflex test evaluates the latency to right at PND-6, n > 17 pups per group.

(B) Clasping on tail suspension gives the mean clasping score achieved by each genotype on a scale from 0 to 4 at PND-45, where 0 refers to wild-type behavior, linearly increasing to 4 which refers to highly impaired behavior, n > 10 mice per group.

(C) The gait assay test gives the paw angle variation between fore- and hind-paws during the walk at PND-90. The graph plots the final average of all tested animals in each group. n = 13 control, n = 12 FD, and n = 7 FD treated.

(D) Ejection fraction percentage (modified Simpson’s method) at PND-45 (n = 6 control, n = 3 FD, and n = 6 FD treated) and PND-90 (n = 8 control, n = 5 FD affected, and n = 8 FD treated).

(E) Creatinine levels measured in serum of PND-90 mice using an enzymatic assay (n = 10 mice per group). Statistical analysis was performed using two-way ANOVA with Bonferroni correction. Bars are SEM; ns, not significant; ^∗p < 0.05; ^∗∗p < 0.01; ^∗∗∗p < 0.001; ^∗∗∗∗p < 0.0001. The tests were performed on littermate controls (Ikbkap^flox/⁺; h-TgFD9/+) and FD mice (Ikbkap^Δ20/Ikbkap^flox; h-TgFD9/+) treated with one intracerebral ventricular injection (i.c.v.) at PND-0 and a second intraperitoneal injection (i.p.) at PND-2 of AAV9-U1-FD (1.6 × 10¹¹ VG/mouse) or saline only. Littermate controls + saline are in black; FD mice + saline in orange; FD mice treated with AAV9-U1-FD in blue.