Cytoplasmic polyadenylation element-binding protein 1 (CPEB1) is a highly conserved, sequence-specific RNA-binding protein that regulates translation and stability of mRNAs by binding to uridine-rich cytoplasmic polyadenylation elements (CPEs) in the 3' UTR of target transcripts. CPEB1 controls poly(A) tail elongation (to enable translation) and represses or activates translation in a phosphorylation-dependent manner, thereby regulating essential biological processes such as cell cycle progression, oocyte maturation, stem cell activation, synaptic plasticity, and formation of stress granules. Dysfunction or altered regulation of CPEB1 is associated with cancer, neurodegenerative diseases, and infertility, making it a potential therapeutic target.
Other names
CPEB1CPEBCPE-BP1CPE-binding protein 1h-CPEBhCPEB-1FLJ13203
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Mechanism of action
Drugs or small molecules targeting CPEB1 would be expected to modulate its RNA-binding activity or its phosphorylation state, thereby altering translation of CPE-containing mRNAs and affecting cellular proliferation, differentiation, or plasticity.
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Biological functions
Regulation of mRNA translation via cytoplasmic polyadenylationmRNA processing and stabilityControl of cell cycle progression (notably G1/prophase entry)Regulation of oocyte maturation and early developmental processesModulation of synaptic plasticity and memory in neuronsFormation and regulation of stress granulesPost-transcriptional control during muscle stem cell activation and regeneration
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Disease associations
Cancer: CPEB1 modulates proliferation and tumorigenesis, affects glioblastoma, hepatocellular carcinoma, and is implicated in epithelial–mesenchymal transitionNeurodegenerative disease: Associated with synaptic dysfunction and memory formation pathwaysPrimary ovarian insufficiencyOthers: roles in aging/senescence (TP53 translation)
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Safety considerations
Broad post-transcriptional regulation creates risk for widespread “off-target” effects if modulated therapeutically.Potential impact on essential cell processes (cell cycle, stem cell activation, neuronal plasticity) raises safety concerns during drug modulation.Lack of highly specific inhibitors or modulators limits clinical translation.
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Interacting drugs
No clinical drugs are directly listed as targeting CPEB1 in current major databases or literature searches. Experimental compounds may modulate its phosphorylation or RNA-binding activity, mainly in research settings.
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Biomarkers
CPEB1 expression levels (as measured by mRNA or protein staining)Phosphorylation state of CPEB1mRNA/poly(A) tail length or presence of CPEs in the 3′ UTR (as surrogates for CPEB1 activity)Upstream/downstream targets, e.g., Myod1, TP53, Cyclin B1, p27Kip1, Sirt1
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