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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



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]

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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