Table 3.
Comparison of the PTC-readthrough efficiency for AAG/PTC combinations tested ex vivo in the HEK293FT cells transfected with pDluc constructs.
| Normalized PTC-readthrough efficiency (%) in the presence of AAG |
|||
|---|---|---|---|
| Location of PTC (gene_exon) | Paromomycin 2000 µg/ml | Gentamicin 2000 µg/ml | G418 400 µg/ml |
| DNAH5_32 | 2.3 ± 0.3 | 2.1 ± 0.4 | 2.5 ± 0.5 |
| DNAH5_49 | 1.9 ± 0.6 | 1.9 ± 0.3 | 4.4 ± 1.1 |
| DNAH11_70 | 2.6 ± 0.2 | 3.2 ± 0.1 | 6.1 ± 1.0 |
| RSPH4A_3(a) | 1.5 ± 0.0 | 2.0 ± 0.0 | 3.2 ± 0.5 |
| DNAH5_20 | 1.2 ± 0.2 | 1.0 ± 0.3 | 2.3 ± 0.1 |
| SPAG1_16 | 0.8 ± 0.0 | 1.1± 0.0 | 1.3 ± 0.0 |
The PTC-readthrough efficiency is expressed as the ratio of Rluc to Fluc activities in the AAG-treated cells transfected with pDluc-PTC constructs, normalized by the ratio in the untreated cells transfected with the corresponding wild-typeconstruct (set as 100%). Only the data for the highest concentration of each AAG are shown.