UBA3

Chr 3

ubiquitin like modifier activating enzyme 3

Also known as: NAE2, UBE1C, hUBA3

The UBA3 protein serves as the catalytic subunit of an E1 enzyme that activates NEDD8, a ubiquitin-like protein essential for cell cycle progression and regulation of protein degradation pathways. Mutations cause autosomal recessive developmental delay with seizures and dysmorphic features, typically presenting in infancy or early childhood. This gene shows significant constraint against loss-of-function variants (LOEUF 0.405), indicating that complete loss of UBA3 function is likely incompatible with normal development.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
7
Pubs (1 yr)
11
P/LP submissions
0%
P/LP missense
0.41
LOEUF
Mechanism
Clinical SummaryUBA3
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.50) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
11 unique Pathogenic / Likely Pathogenic· 44 VUS of 91 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.41LOEUF
pLI 0.504
Z-score 4.14
OE 0.22 (0.120.41)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.17Z-score
OE missense 0.79 (0.700.89)
187 obs / 237.5 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.22 (0.120.41)
00.351.4
Missense OE0.79 (0.700.89)
00.61.4
Synonymous OE0.91
01.21.6
LoF obs/exp: 7 / 32.5Missense obs/exp: 187 / 237.5Syn Z: 0.67

ClinVar Variant Classifications

91 submitted variants in ClinVar

Classification Summary

Pathogenic10
Likely Pathogenic1
VUS44
Likely Benign1
10
Pathogenic
1
Likely Pathogenic
44
VUS
1
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
10
0
10
Likely Pathogenic
0
0
1
0
1
VUS
0
40
4
0
44
Likely Benign
0
1
0
0
1
Benign
0
0
0
0
0
Total04115056

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

View in ClinVar →

Protein Context — Lollipop Plot

UBA3 · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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