Target intelligence / Profile preview

RNA-binding protein with multiple splicing (RBPMS) (RBPMS)

Target
RBPMS
Molecular classification
RNA-binding protein, RRM family, Splicing factor, Transcription coactivator
01

Overview

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).

Other names
HERMESRNA binding protein mRNA processing factorRBP-MSHeart and RRM expressed sequence
02

Mechanism of action

Competitive inhibition of RNA-protein binding

03

Biological functions

Alternative splicing regulationmRNA transportmRNA stabilitySMAD signalingCardiomyocyte contractilityRetinal ganglion cell survivalGlycolysis regulation
04

Disease associations

Acute myeloid leukemiaDilated cardiomyopathyBladder cancerProstate cancerOvarian cancerRetinal degenerationPolycystic ovary syndrome
05

Safety considerations

Potential cardiotoxicity due to role in cardiomyocyte contractilityPotential ocular toxicity due to role in retinal ganglion cell survivalBroad off-target effects from systemic splicing modulation
06

Interacting drugs

RB98
07

Biomarkers

RBPMS (Retinal ganglion cell marker)FOXO1 expression levels

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