Figure 5.

Allele-specific reduction of mHTT protein in the mouse adult striatum. (A) Protein sample analysis of HTT and mHTT protein from the striatum of HD model mice with the Cas9, BAC97, and YAC18 transgenes. Each animal was bilaterally injected with AAV expressing the control (Rosa26) or Ex50Tg1 sgRNAs. Protein was isolated from striatal tissue. Vinculin serves as a total protein control. Signal is visualized as a virtual blot format. (B) Quantitative WES signals for mHTT and wt HTT were normalized to vinculin. Each pair of control and Ex50T-treated samples were further adjusted such that the control has a value of 1. At both 4- and 6-weeks after injection with AAV expressing sgRNA Ex50T, mHTT was reduced by >50% while wt HTT was not significantly changed. n = 3 mice and error bars represent the SEM. p-values <0.05 were considered significant. Exact p-values are reported in Supplementary File 3. WES, Simple Western protein analysis.