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Table 1 Structure, mechanism of action and side effects of NMD pathway inhibitors

From: Deciphering the nonsense-mediated mRNA decay pathway to identify cancer cell vulnerabilities for effective cancer therapy

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
NMDI-1 [73, 74] 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