Allele-specific silencing of mutant ataxin-3 reduces the levels of mutant aggregated protein.

<p>A) Western blot analysis of cerebellar lysates stained with 1C2 antibody (for the polyglutamine expansion). Differences in levels of high molecular weight protein species were detected between transgenic mice injected with shAtx3 (<i>n</i> = 3) and animals injected with shGFP (<i>n</i> = 3). Normalization of protein levels was made with β-actin protein endogenous levels, and with β-gal marker (reporter gene of shRNAs lentiviral vector). B, D). The quantification of aggregated protein levels (in the stacking gel) revealed significant differences between transgenic mice injected with shAtx3 and control animals injected with shGFP (<i>n</i> = 3; *<i>P</i><0.05; Unpaired Student's <i>t</i>-test). C,E). Oligomerized species were also significantly reduced in animals injected with shAtx3 compared to control animals (<i>n</i> = 3; *<i>P</i><0.05; Unpaired Student's <i>t</i>-test).