Table 2.
Screening of Notch3 mutant ASOs for activity in vitro.
| ASO | Notch3 Downregulation | Notch36691TAATGA Downregulation |
|---|---|---|
| 07 | 0% | 0% |
| 08 | 0% | 0% |
| 10 | 0% | 0% |
| 11 | 0% | 0% |
| 13 | 0% | 0% |
| 14 | ~15% | 100% |
| 16 | ~40% | 100% |
| 17 | 0% | 70% |
| 18 | 0% | 60% |
| 19 | ~40% | 0% |
| 20 | ~25% | 0% |
| 21 | 100% | 100% |
Twelve ASOs considered safe were tested at 20 μM for their effect on Notch3 wild type or Notch36691TAAGTAmutant transcripts in osteoblast-enriched cells from Notch3em1Ecan mice. Notch3 mutant ASOs that were not safe or borderline safe in vivo were not tested in vitro.