Table 2.
The summary of cell-based reporter assays used to test ataluren readthrough activity.
| Cell line | Construct | Nonsense Mutation and Its Position | Read-Out | Ataluren Concentration (μg/mL) |
|---|---|---|---|---|
| ADXC8 | Fluc2P (stable) | TGA-G at 223 | luciferase | 0.0000028–28.4 |
| AD293 | β-galactosidase (stable) | TGA-G at 320 | β-gal | 0.028–28.4 |
| Rluc (transient) | TGA-G at 21 | luciferase | 0.00028–28.4 | |
| AD293 | FLAG-tagged collagen VII (transient) | TAG-T (Q251X) | ELISA | 0.028–28.4 |
| FLAG-tagged collagen VII (transient) | TGA-G (R578X) | ELISA | 0.028–28.4 | |
| AD293 | Keratin 6a-YFP fusion (transient) | TGA/TAG/TAA at 533 | Western blot | 0.85 |