Exploring the therapeutic potential of modulating nonsense-mediated mRNA decay

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FIGURE 4.
FIGURE 4.

Proposed role of NMD in FXS and potential benefit of NMD inhibitor treatment in FXS. Schema highlighting the reported roles of NMD misregulation in FXS (left). Dampening NMD (either genetically or pharmacologically) leads to NMD target stabilization and rescue of transcriptome misregulation in FXS (right). The development of NMD inhibitors to dampen NMD hyperactivity in FXS is an attractive area of further therapeutic development.

This Article

  1. RNA 31: 333-348