Skip to main content
. Author manuscript; available in PMC: 2015 Oct 1.
Published in final edited form as: Mutat Res Rev Mutat Res. 2014 May 28;0:52–64. doi: 10.1016/j.mrrev.2014.05.001

Figure 4. Combining NMD Inhibition with Nonsense Suppression to Enhance Therapeutic Outcome.

Figure 4

Nonsense-mediated decay targets PTC-bearing transcripts in the cytoplasm, leading to a decrease in the pool of mRNA, which produces little to no full-length protein. The goal of nonsense suppression therapy is to produce full-length functional protein to alleviate disease pathology. However, nonsense-mediated degradation of PTC-bearing transcripts decreases the abundance of mRNA, limiting the efficacy of read-through therapy and decreasing the chance of surpassing the therapeutic threshold. By combining NMD inhibition with nonsense suppression there is an increased abundance of PTC-bearing mRNA with which read-through drugs can act on, leading to an increase in the production of full-length protein.