(A) Schematic of AAV-shSGCE-GFP construct. (B) Images of the whole brain (i) and sagittal cerebellar section (ii) from an Sgce KD CB mouse. (C) Quantification of qRT-PCR confirms that Sgce RNA is reduced in vivo. (Mann-Whitney Test, WT vs. NT CB: p=0.7922; NT CB vs. Sgce KD CB 1: p=0.0079; NT CB vs. Sgce KD CB 2: p=0.0159; NWT = 6, NNT CB = 5, NSgce KD CB 1 = 5, NSgce KD CB 2 = 4). (D,i) Injection of Sgce KD 1 and 2 into the cerebellum produced dystonia, while injection of NT did not. (Sgce KD CB 1: N = 39; Sgce KD CB 2: N = 40; NT CB: N = 16). Dystonia was measured on a previously published dystonia scale by four scorers blind to the condition of the animal. A score greater than or equal to two indicates dystonia. The dystonia scores for Sgce KD CB 1 and Sgce KD CB 2 mice for time points of 2 weeks or more after injection were significantly different from the dystonia scores of the same animals at <1 week (Wilcoxon matched-pairs signed rank test, p<0.01). The dystonia scores of Sgce KD CB 1 and Sgce KD CB 2 mice at <1 week after injection were not significantly different from NT CB mice at the same time point (t-test, Holm-Sidak method, p=0.81 and p=0.97, respectively). (ii) Example dystonic postures exhibited by Sgce KD CB mice. (E) Scatter plot of RNA levels normalized to the mean of WT, determined by qRT-PCR, plotted against the Dystonia Score observed in a subset of animals injected with varying concentrations of shRNA (WT: N = 5, NT: N = 5, Sgce KD CB 1: N = 13, Sgce KD CB 2: N = 7).