Molecule | Structure | Mechanism of action | Side effects |
---|---|---|---|
Aminoglycosides [71] |
| PTC readthrough | Toxic at clinical active concentrations |
Caffeine [72] |
| SMG1 inhibition | Toxicity, highly aspecific by targeting several kinases |
| Stabilization of the phosphorylated form of UPF1 | No side effects in mice | |
5-azacytidine [75] | Non identified, but c-MYC-mediated | Already approved for the treatment of diverse malignancies | |
Amlexanox [76] |
| Inhibits NMD at a late stage | Clinically approved for aphthous ulcers |
NMDI14 [77] |
| Disrupts the SMG7–UPF1 complex | Negligible toxicity in cell-based assays |