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Target intelligence / Profile preview
MYCN upstream transcript (non-protein coding) (MYCNUT)
- Target
- MYCNUT
- Molecular classification
- Long non-coding RNA (lncRNA), Other
01
Overview
MYCN upstream transcript (non-protein coding) (MYCNUT), also called lncUSMycN, is a long non-coding RNA located approximately 5–14 kb upstream of the MYCN oncogene. MYCNUT is co-amplified with MYCN in neuroblastoma and acts as a positive regulator of MYCN expression. Knockdown of MYCNUT decreases MYCN mRNA and protein levels, while overexpression increases MYCN mRNA. The regulatory function depends on binding to the RNA-binding protein NONO, which stabilizes MYCN transcripts and enhances their expression. Unlike protein-coding genes or membrane-bound receptors, MYCNUT is not a traditional drug target, but is of biological and disease interest due to its key role in MYCN-driven neuroblastoma pathogenesis[2][4].
- Other names
- lncUSMycNMYCNUNMYCN upstream neighborMYCNUTMYCN upstream transcript
02
Mechanism of action
Not applicable—no direct drugs known; acts through upregulation of MYCN mRNA by binding the RNA-binding protein NONO
03
Biological functions
Regulation of MYCN expression (transcriptional and post-transcriptional)Cell proliferation (via MYCN)Acting as a scaffold for RNA-binding proteins
04
Disease associations
Cancer, specifically Neuroblastoma
05
Safety considerations
None specific to MYCNUT reported; general concerns exist for targeting lncRNAs (off-target effects, delivery challenges)
06
Biomarkers
Potential marker for MYCN-amplified neuroblastoma (not widely validated in the clinic)
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