Table 1.
Gapmer ASOs used in the first screening
| ASO ID | Sequencea | Target | References |
|---|---|---|---|
| C1 | CCTATaggactatccAGGAA | – | Jiang et al.21 |
| 1 | GGGCTcatcgttctcATCTT | exon 2 | Ayala et al.12, Iguchi et al.13, Aulas et al.29 |
| 2 | TTCAAtgggctcatcGTTCT | exon 2 | Ayala et al.12, Iguchi et al.13, Aulas et al.29 |
| 3 | CAGTCttaagatcttTCTTG | exon 4 | Koyama et al.30 |
| 4 | CAAAGgctcatcttgGCTTT | exon 5 | Aulas et al.29, McDonald et al.31, De Conti et al.32 |
| 5 | TTAATgatcaagtccTCTCC | exon 6 | Stoica et al.33 |
| 6 | ATATAtgaacgctgaTTCCT | exon 6 | Swarup et al.34 |
| 7 | GTGCTtaggttaggcATTGG | exon 6 | Iguchi et al.13 |
| 8 | ATCCAtgcttgagccAAAGC | exon 6 | Yu et al.35 |
| 9 | AAGGCttcatattgtACTTT | 3′ UTR | Lagier-Tourenne et al.36 |
| 10 | AATATccattatgcaCCACC | 3′ UTR | Yu et al.35 |
a
Capital letter, ENA; small letter, DNA.