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RNA-binding protein with multiple splicing (RBPMS) is a member of the RNA recognition motif (RRM) family that acts as a master regulator of alternative splicing, particularly in smooth muscle cells and cardiomyocytes. It functions by binding to specific RNA sequences, such as tandem CAC motifs, and interacting with other RNA-binding protein partners like MBNL1, RBFOX2, and RBM4 to form stable regulatory complexes that govern cell-specific splicing patterns (Farazi et al., 2014; GeneCards). Beyond splicing, RBPMS is involved in mRNA transport, stability, and the modulation of SMAD signaling through direct interactions with SMAD2/3/4 (Wikipedia; Piri et al., 2006). In disease, RBPMS is linked to dilated cardiomyopathy, where its loss leads to abnormal splicing of sarcomeric genes, and various cancers where it may act as a tumor suppressor or regulator of proliferation (NIH; ResearchGate). Recent research has identified RBPMS as a critical driver of acute myeloid leukemia (AML) progression by stabilizing FOXO1 mRNA, leading to the development of the specific small-molecule inhibitor RB98 (Liu et al., 2026). RB98 competitively disrupts the RBPMS-RNA interaction, inhibiting leukemic cell growth and promoting apoptosis while sparing normal hematopoiesis (ProbeChem; ecancer.org). Additionally, RBPMS serves as a definitive biomarker for identifying and tracking retinal ganglion cells in neurodegeneration and glaucoma research (arvojournals.org).
Competitive inhibition of RNA-protein binding
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