Target intelligence / Profile preview

CCAAT enhancer-binding protein alpha (CEBPA) (CEBPA)

Target
CEBPA
Molecular classification
Transcription factor, Basic leucine zipper (bZIP) protein family
01

Overview

CCAAT enhancer-binding protein alpha (CEBPA) is a critical transcription factor belonging to the basic leucine zipper (bZIP) family, primarily known for its role in coordinating the balance between cell proliferation and terminal differentiation (UniProt P49715). It is a master regulator of myelopoiesis, essential for the development of granulocytes, and also plays a vital role in the metabolic regulation of hepatocytes and adipocytes (PubMed: 15133543). In clinical oncology, CEBPA is frequently associated with acute myeloid leukemia (AML), where mutations or epigenetic silencing lead to a block in myeloid maturation and the expansion of leukemic blasts (NCBI Gene: 1050). Therapeutic strategies targeting CEBPA include the use of small activating RNAs (saRNAs), such as MTL-CEBPA, which are designed to upregulate its expression and restore normal cellular differentiation (PubMed: 30104334). Beyond cancer, CEBPA is investigated for its potential in treating chronic liver diseases and metabolic disorders due to its influence on lipid and glucose homeostasis.

Other names
C/EBP alphaCEBPACCAAT/enhancer-binding protein alphaC/EBP-alphaTranscription factor C/EBP alpha
02

Mechanism of action

Upregulation of CEBPA expression through small activating RNA (saRNA) technology to promote myeloid differentiation and inhibit tumor growth, or indirect modulation via epigenetic modifiers to reverse gene silencing.

03

Biological functions

Cell differentiationMyelopoiesisAdipogenesisHepatocyte differentiationCell cycle regulationGlucose metabolismLipid metabolism
04

Disease associations

Acute myeloid leukemia (AML)Non-alcoholic steatohepatitis (NASH)Hepatocellular carcinomaObesityDiabetes mellitus
05

Safety considerations

Potential for systemic inflammatory responseOff-target effects of oligonucleotide deliveryLiver enzyme elevationsLineage-specific toxicity in hematopoiesis
06

Interacting drugs

MTL-CEBPA

3 more in the full profile.

07

Biomarkers

CEBPA mutation statusCEBPA mRNA expression levelsCD11b expressionCD14 expression

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