Table 1.
IC50 Values for Inhibition of Ataxin-3 and Huntingtin Expression for Single-Stranded Silencing RNAs Examined in this Study
| Effect on HTT | Effect on ATX-3 | ||||||
|---|---|---|---|---|---|---|---|
| No. | Mismatch position | mut IC50 (nM) | wt IC50 (nM) | Selectivity (fold) | mut IC50 (nM) | wt IC50 (nM) | Selectivity (fold) |
| 537775 | 9 | 3.5±0.3 | >100 | >29 | 3.6±1.4 | 20±2.5 | 6 |
| 537787 | None | 8.0±1.7 | >100 | >13 | 28±6.4 | 52±12 | 2 |
| 553822 | 9 | 4.9±0.8 | 90±9.7 | 18 | 8.4±2.5 | 99±28 | 12 |
| 557409 | 9, 10 | 6.3±0.5 | >100 | >16 | 16±3.5 | 62±16 | 4 |
| 557426 | 9, 10, 11 | 3.3±0.5 | >100 | >30 | 30±6.5 | >100 | 3 |
| 581440 | 9 | 3.0±0.6 | 20±7.5 | 7 | 12±1.2 | 75±18 | 6 |
| 618385 | 9, 14 | 6.4±1.5 | >100 | >16 | 1.4±0.2 | >50 | >35 |
| 641385 | 9, 18 | 5.8±0.3 | >100 | >17 | 2.1±0.3 | >50 | >23 |
| 618202 | 9 | 2.7±0.7 | 44±4.9 | 16 | 7.9±1.4 | >50 | >6 |
| 618204 | 9 | 7.2±1.2 | >100 | >14 | 3.1±0.8 | >50 | >16 |
ATX-3, ataxin-3; HTT, huntingtin; IC50, half maximal inhibitory concentration; Mut, mutant; ss-siRNAs, single-stranded silencing RNA; WT, wild-type.