Table 1.
Variables associated with the splicing screen score (S3) for diamidine analog screen
|
INSR ex11 |
FLNB ex31 |
SYNE1 ex65 |
IC50 | S3∗ | ||||
|---|---|---|---|---|---|---|---|---|
| EC50 | Emax | EC50 | Emax | EC50 | Emax | |||
| DM1-B | ||||||||
| Furam | 3 ± 0.4 | 75 ± 7 | 3 ± 0.4 | 83 ± 4 | – | – | 18 ± 0.4 | 1.00 |
| DB1210 | – | – | 25 ± 4 | 25 ± 7 | – | – | 88 ± 3.6 | 0.09 |
| DB1242 | – | – | 2 ± 1 | 97 ± 9 | – | – | 68 ± 4.7 | 3.18 |
| DB829 | 27 ± 5 | 41 ± 15 | 2 ± 1 | 89 ± 15 | – | – | 73 ± 1.1 | 3.23 |
| DB1246 | 22 ± 4 | 22 ± 9 | 3 ± 2 | 74 ± 13 | – | – | 15 ± 0.5 | 0.42 |
| DB1247 | 0.6 ± 0.1 | 77 ± 12 | 0.5 ± 0.3 | 70 ± 4 | 1.7 ± 1.3 | 36 ± 11 | 1 ± 0.2 | 0.38 |
| DB1273 | 0.8 ± 0.3 | 45 ± 8 | 0.1 ± 0.1 | 109 ± 3 | – | – | 2 ± 0.1 | 1.64 |
| DB270 | 186 ± 26 | 13 ± 3 | 1.6 ± 1 | 57 ± 13 | – | – | 69 ± 1.3 | 2.46 |
| Cmpd7 | 0.1 ± 0.1 | 42 ± 10 | 0.4 ± 0.3 | 68 ± 5 | 20 ± 2 | 28 ± 11 | 69 ± 0.5 | 35.48 |
| DM2-A | ||||||||
| Furam | 2 ± 0.6 | 77 ± 10 | 2 ± 0.7 | 74 ± 4 | 5 ± 1 | 63 ± 12 | 19 ± 0.8 | 1.00 |
| DB1210 | 23 ± 8 | 38 ± 8 | 24 ± 6 | 34 ± 8 | – | – | 93 ± 5.1 | 0.16 |
| DB1242 | 24 ± 5 | 51 ± 6 | 71 ± 14 | 36 ± 6 | 185 ± 2 | 23 ± 16 | 75 ± 6.1 | 0.12 |
| DB829 | 10 ± 2 | 74 ± 1 | 22 ± 5 | 53 ± 4 | 129 ± 3 | 25 ± 14 | 74 ± 4.8 | 0.43 |
| DB1246 | 8 ± 3 | 57 ± 7 | 7 ± 3 | 51 ± 1 | 16 ± 2 | 28 ± 13 | 15 ± 0.9 | 0.15 |
| DB1247 | 0.9 ± 0.5 | 86 ± 11 | 0.9 ± 0.4 | 49 ± 4 | 0.9 ± 1.1 | 51 ± 7 | 2 ± 0.4 | 0.19 |
| DB1273 | 0.9 ± 0.4 | 58 ± 2 | 0.2 ± 0.3 | 77 ± 4 | 1.8 ± 1.4 | 34 ± 11 | 2 ± 0.3 | 0.43 |
| DB270 | 23 ± 4 | 43 ± 9 | 33 ± 9 | 21 ± 3 | 16 ± 2 | 25 ± 13 | 67 ± 1.1 | 0.15 |
| Cmpd7 | 0.7 ± 0.3 | 48 ± 11 | 0.8 ± 1 | 36 ± 7 | 15 ± 2 | 19 ± 14 | 68 ± 2.1 | 4.46 |
EC50 = Concentration required to reach 50% rescue; Emax = maximum percent rescue; IC50 = concentration required to reach 50% viability; S3∗ = Average of [(Emax/EC50) x IC50] normalized to score of furamidine for INSR exon11, FLNB exon31, and SYNE1 exon65 alternative exon events. DM1-B cell line values under blue column headings and DM2-B cell line values are under green column headings.Bolded entry indicate compound with the highest S3 score.