Target intelligence / Profile preview

MYCN proto-oncogene protein (MYCN)

Target
MYCN
Molecular classification
Transcription factor, Oncogene, DNA-binding protein
01

Overview

The MYCN proto-oncogene protein (frequently known as N-Myc, encoded by the MYCN gene) is a transcription factor belonging to the MYC family. It binds DNA at E-box sequences and regulates the expression of genes involved in fundamental processes such as cell proliferation, differentiation, metabolism, cell cycle progression, apoptosis, and DNA repair[1][3][4][5][7][8]. MYCN has tightly controlled spatial and temporal expression in development, peaking during embryogenesis and organogenesis, and is critical for the normal development of tissues including the nervous system, heart, kidney, and lung[2][3][6]. Pathologically, MYCN amplification or overexpression is a well-established driver of multiple pediatric and adult cancers, most notably neuroblastoma, where it is the strongest genetic biomarker of poor prognosis and aggressive disease[1][2][6][7][8]. Targeting MYCN, either directly or indirectly through pathways regulating its stability and activity, is an area of active therapeutic development, though clinical application is challenged by its role in normal development and the lack of a well-defined druggable pocket[1][6][7][8].

Other names
N-MycMYCN geneMYCN protein
02

Mechanism of action

Inhibition of MYCN transcriptional activity, Destabilization of MYCN protein, Disruption of MYCN/MAX dimerization, Suppression of MYCN gene expression or translation

03

Biological functions

Regulation of cell proliferationRegulation of cell differentiationEmbryonic developmentCell cycle controlApoptosisMetabolic regulationDNA repair
04

Disease associations

CancerNeuroblastomaMedulloblastomaRhabdomyosarcomaWilms tumorProstate cancerLung cancer
05

Safety considerations

On-target toxicity in normal embryonic and stem cells (given MYCN’s essential roles in development)Off-target effects on other MYC family proteinsPotential disruption of normal tissue regeneration and differentiation
06

Interacting drugs

BET inhibitors (e.g., JQ1)

4 more in the full profile.

07

Biomarkers

MYCN amplification (marker of poor prognosis in neuroblastoma)N-Myc protein overexpression

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