Figure 1. NMD inhibition therapy has the potential to improve the clinical outcome of a subset of human genetic diseases.

In cases where transcripts harboring a premature termination codon (PTC) produce a protein detrimental to the cell, NMD reduces the dominant-negative or toxic effects by targeting these transcripts for degradation. In transcripts in which the position of the PTC allows for the generation of a still functional protein, NMD is detrimental because it degrades the useful transcript. By reversing this decay, NMD inhibition therapy could improve disease symptoms.