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. 2019 Dec 18;19:562–571. doi: 10.1016/j.omtn.2019.12.012

Figure 2.

Figure 2

Analysis of Efficacy and Allele Selectivity of CAG-Targeting shRNAs in Cellular Models of DRPLA, SCA3, and SCA7

(A) Characterization of polyQ models used in this study. (B–D) Western blot analysis of atrophin-1 levels in DRPLA patient-derived fibroblasts (16/68Q) (B), ataxin-3 levels in SCA3 patient-derived fibroblasts (18/69Q) (C), and ataxin-7 levels in SCA7 patient-derived fibroblasts (8/62Q) (D). Protein was isolated at 7 days posttransduction with two concentrations of LV (MOI of 1 and 10) containing shA2R, shA2R1, shG2, and shG4 expression cassettes. The shLuc construct (Luc) was used as a negative control. Signal intensities of the protein level were normalized to vinculin protein level and compared using a one-sample t test. The graph bars represent the mean value of protein levels ± SEM. The p values are indicated by asterisks (*p < 0.05, **p < 0.01, ***p < 0.001). All experiments were repeated at least three times.