RNA-binding protein, Post-transcriptional regulator, Splicing factor, Member of ELAV-like RBP family
01
Overview
CELF1 is a pivotal post-transcriptional regulator with three RNA recognition motifs that bind GU-rich elements within target mRNAs. It orchestrates alternative splicing, translation, and mRNA decay, thus modulating key processes such as cell proliferation, apoptosis, differentiation, and migration. CELF1’s dysregulation contributes to multiple pathologies—including myotonic dystrophy and diverse cancers (where it promotes tumor growth, EMT, and metastasis). As a central node in post-transcriptional gene regulation, CELF1 is considered a promising but complex therapeutic target. There are significant challenges to its clinical modulation, given its broad essential functions for normal cellular homeostasis.
Other names
CUG-binding protein 1CUGBP1BRUNOL2NAB50EDEN-BP homologRNA-binding protein BRUNOL-2Bruno-like protein 2CUG triplet repeat RNA-binding protein 1CUG-BP- and ETR-3-like factor 1Deadenylation factor CUG-BPhNab50NAPOR50 kDa nuclear polyadenylated RNA-binding proteinEmbryo deadenylation element-binding protein homolog
02
Mechanism of action
Proposed for research molecules: modulation of RNA-binding activity to alter splicing, translation, or stability of disease-associated mRNAs
03
Biological functions
Regulation of alternative splicingRegulation of mRNA stability and decayControl of mRNA translation initiation and repressionCell cycle regulationRegulation of cell proliferationApoptosis modulationEpithelial-mesenchymal transition (EMT) and metastasis
04
Disease associations
Myotonic dystrophy type 1 (DM1)Cancer (notably breast, lung, head & neck squamous cell carcinoma)Neurodegenerative disease (e.g., fragile-X tremor/ataxia syndrome)Liver dysfunction
05
Safety considerations
Targeting CELF1 carries potential risks of off-target effects due to its widespread physiological RNA regulatory functions (e.g., cell cycle, apoptosis, tissue development). Knockout models exhibit embryonic lethality.Challenge in specifically modulating CELF1 activity without disturbing normal post-transcriptional regulation in vital tissues.
06
Interacting drugs
No approved drugs directly targeting CELF1 are currently described in the literature. Research is ongoing into RNA therapeutics and small molecules indirectly affecting CELF1-related pathways.
07
Biomarkers
CELF1 protein overexpression in tumor tissue (prognostic marker in head & neck, breast, and lung cancers)CELF1 levels in DM1 muscle tissuemRNAs regulated by CELF1 (e.g., MYC, IL24, MMP1)
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