RNA-binding protein RBM47 in health and disease: molecular mechanisms, preclinical evidence, and translational challenges.

RNA-binding motif protein 47 (RBM47) is an evolutionarily conserved multifunctional RNA-binding protein. It mediates post-transcriptional regulation through C-to-U RNA editing, alternative splicing, and messenger RNA (mRNA) stability control. Preclinical evidence indicates that RBM47 exhibits context-dependent, dual effects in human disease. In breast cancer (BC), colorectal cancer (CRC), renal cell carcinoma (RCC), papillary thyroid carcinoma (PTC), and non-small cell lung cancer (NSCLC), experimental studies suggest that RBM47 exerts tumor-suppressive activity through the inhibition of the Wnt/β-catenin, phosphoinositide 3-kinase (PI3K)-AKT, and nuclear factor erythroid 2-related factor 2 (Nrf2) pathways. Conversely, in glioma and pancreatic cancer (PC), cell and animal models indicate that RBM47 exerts oncogenic activity by promoting M2 macrophage polarization and immune evasion. Furthermore, aberrant RBM47 expression has been implicated in postoperative cognitive dysfunction (POCD), inflammatory bowel disease (IBD), and antiviral innate immune regulation in preclinical models. This review summarizes the molecular characteristics and pathological mechanisms of RBM47, discusses the preclinical rationale for its potential utility as a biomarker and therapeutic node, and critically analyzes current research limitations, conflicting evidence, and translational bottlenecks. All biomarker and therapeutic applications discussed remain at the preclinical or retrospective-correlative stage; no RBM47-targeted intervention has entered clinical trials.