Table 1.
ASOs used in this study
| ASO name | IONIS # | Length | Sequence 5'-3' | Chemistry |
|---|---|---|---|---|
| ASO1-MOE | 5644676 | 18 nt | CGGCGGAGTTCCTCAAAT | Uniform MOE |
| ASO1-cEt/DNA | 742114 | 18 nt | CGGCGGAGTTCCTCAAAT | kkddkddkddkddkddkk |
| ASO1-MOE/DNA | 759514 | 18 nt | CGGCGGAGTTCCTCAAAT | eeddeddeddeddeddee |
| ASO2-MOE | 564472 | 18 nt | AGGCGGCGGAGTTCCTCA | Uniform MOE |
| ASO2-cEt/DNA | 872245 | 18 nt | AGGCGGCGGAGTTCCTCA | kkddkddkddkddkddkk |
| GN3-ASO2-MOE | 1057288 | 18 nt | AGGCGGCGGAGTTCCTCA | 5'-GalNAc3-Uniform MOE |
| GN3-ASO2-cEt/DNA | 950663 | 18 nt | AGGCGGCGGAGTTCCTCA | 5'-GalNAc3-kkddkddkddkddkddkk |
| GN3-SMN-MOE | 699819 | 20 nt | ATTCACTTTCATAATGCTGG | 5'-GalNAc3-Uniform MOE |
| Ctrl. ASO-MOE (M4) | 759518 | 18 nt | CGGATGAGTGCCTGCAAT | Uniform MOE |
| Ctrl. ASO-cEt/DNA (M4) | 872243 | 18 nt | CGGATGAGTGCCTGCAAT | kkddkddkddkddkddkk |
| Scr. Ctrl ASO | 742116 | 18 nt | GCTGATCCGAGTAAGCTC | Uniform MOE |
| 12mer MOE | 759513 | 12 nt | CGGCGGAGTTCC | Uniform MOE |
| I9-MOE | 461378 | 15 nt | TACCTGCCCTTAGGG | Uniform MOE |
e: 2'-O-methoxyethyl (MOE) base modification; k: constrained ethyl (cEt) base modification; d: DNA base; GN3: tri-antennary N-acetyl galactosamine; all ASOs have a uniform PS backbone and 5-methyl-C modifications.