Figure 4. Potent and selective inhibition of mutant ataxin-3.
All data show analysis of ataxin-3 expression in GM06151 fibroblast cells. Examples of Western gels used for quantitation are shown in Supplementary Figure 10 online. (a) Schematic of target sites for PNAs within ataxin-3 mRNA, Inhibition of ataxin-3 expression by (b) PNA conjugate REP19, (c) PNA conjugate 3J/ATX, (d) PNA conjugate 5J/ATX, and (e) siRNA/REP.