NUPR1

Chr 16

nuclear protein 1, transcriptional regulator

Also known as: COM1, P8

The protein encoded by this gene is a transcription regulator that converts cellular stress signals into gene expression programs, controlling cell cycle progression, apoptosis, autophagy, and DNA repair responses. Mutations in NUPR1 cause autosomal recessive neurodevelopmental disorder with microcephaly and structural brain anomalies, typically presenting in early infancy with developmental delays and neurological features. The gene shows tolerance to loss-of-function variants (pLI 0.004, LOEUF 1.91), consistent with the recessive inheritance pattern observed in affected patients.

ResearchSummary from RefSeq, UniProt
MultiplemechanismLOEUF 1.91
Clinical SummaryNUPR1
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
71 unique Pathogenic / Likely Pathogenic· 46 VUS of 123 total submissions
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.91LOEUF
pLI 0.004
Z-score -0.41
OE 1.29 (0.521.91)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.12Z-score
OE missense 1.04 (0.861.27)
68 obs / 65.3 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE1.29 (0.521.91)
00.351.4
Missense OE1.04 (0.861.27)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 3 / 2.3Missense obs/exp: 68 / 65.3Syn Z: 0.02
DN
0.6936th %ile
GOF
0.6442th %ile
LOF
0.3552th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median
GOFprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

123 submitted variants in ClinVar

Classification Summary

Pathogenic66
Likely Pathogenic5
VUS46
Likely Benign1
66
Pathogenic
5
Likely Pathogenic
46
VUS
1
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
66
0
66
Likely Pathogenic
0
0
5
0
5
VUS
0
25
21
0
46
Likely Benign
0
0
1
0
1
Benign
0
0
0
0
0
Total025930118

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

NUPR1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
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