Table 1. Inhibition of Expression of ATN-1, ATXN-3, and HTT by Duplex RNAs and ss-siRNAs.
| effect
on HTT |
effect on ATX-3 |
effect
on ATN-1 |
|||||||
|---|---|---|---|---|---|---|---|---|---|
| name | mutant IC50 (nM) | wt IC50 (nM) | selectivity (x-fold) | mutant IC50 (nM) | wt IC50 (nM) | selectivity (x-fold) | mutant IC50 (nM) | wt IC50 (nM) | selectivity (x-fold) |
| dsRNAs | |||||||||
| P9 | 3.2 ± 0.3 | >100 | >31 | 4 | 50 | 11 | 0.7 ± 0.2 | 8.5 ± 1.6 | 12 |
| PM4 | 4.8 ± 1.6 | >100 | >21 | 6.5 ± 2.8 | 50 | 8 | 1.4 ± 0.3 | >25 | >18 |
| AB8 | 3.3 ± 0.6 | >100 | >30 | 3.3 ± 0.6 | >100 | >30 | 0.9 ± 0.2 | 3.5 ± 0.5 | 3.9 |
| P9U13 | 22 ± 0.4 | >100 | >43 | 13 ± 2.8 | 43 ± 7.9 | 3.2 | 3.9 ± 0.8 | 21 ± 7.8 | 5.3 |
| ss-siRNAs | |||||||||
| 537775 | 3.5 ± 0.3 | >100 | >29 | 2.9 ± 0.3 | 24 ± 6 | 8 | 1.1 ± 0.2 | >25 | >23 |
| 557426 | 3.3 ± 0.5 | >100 | >30 | 28 ± 2.2 | 67 ± 11 | 2.4 | 1.7 ± 0.3 | >25 | >15 |
| 553822 | 4.9 ± 0.8 | 90 ± 9.7 | 18 | 8.4 ± 2.5 | 99 ± 28 | 12 | 1.9 ± 0.3 | >25 | >13 |
| 618385 | 3.6 ± 0.4 | 53 | 14 | 1.4 ± 0.2 | >50 | >37 | 3.0 ± 0.4 | 8.5 ± 1.7 | 2.8 |
| 641384 | 5.1 ± 0.4 | >100 | >19 | 5.1 ± 0.6 | 17 ± 2.9 | 3.4 | 1.2 ± 0.1 | >25 | >21 |
| 618204 | 7.2 ± 1.2 | >100 | >14 | 3.1 ± 0.7 | 71 ± 26 | 23 | 1.1 ± 0.1 | 14 ± 6.1 | 13 |