Therapeutic Targeting of RNA Splicing in Myelodysplasia

RNA splicing factors are exquisitely dependent on the presence of the remaining wild-type (WT) allele to maintain residual normal splicing for cell survival. The high frequency of these mutations in MDS, combined with their mutual exclusivity and noteworthy dependence on the WT allele, make targeting RNA splicing attractive in MDS. To this end, two promising therapeutic approaches targeting RNA splicing are being tested clinically currently. According to Cancer Research UK, the risk varies from five in every 100 cases (5%) to 65 out of every 100 cases (65%), depending on the type of MDS you have.

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