CUL4A encodes a scaffold protein that serves as the core component of multiple cullin-RING-based E3 ubiquitin-protein ligase complexes, which target specific proteins for degradation and regulate processes including DNA repair, cell cycle progression, and circadian rhythms. Mutations cause X-linked intellectual disability with growth retardation and malformations in males, with an X-linked inheritance pattern. The gene is highly intolerant to loss-of-function variants, reflecting its essential cellular functions.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
23
Pubs (1 yr)
117
P/LP submissions
0%
P/LP missense
0.12
LOEUF· LoF intol.
Mechanism
Clinical SummaryCUL4A
Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
📋
ClinVar Variants
117 unique Pathogenic / Likely Pathogenic· 78 VUS of 230 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.12LOEUF
pLI 1.000
Z-score 5.59
OE 0.03 (0.010.12)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
2.81Z-score
OE missense 0.60 (0.540.67)
231 obs / 386.1 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.03 (0.010.12)
00.351.4
Missense OE0.60 (0.540.67)
00.61.4
Synonymous OE1.10
01.21.6
LoF obs/exp: 1 / 38.3Missense obs/exp: 231 / 386.1Syn Z: -1.01

ClinVar Variant Classifications

230 submitted variants in ClinVar

Classification Summary

Pathogenic114
Likely Pathogenic3
VUS78
Likely Benign3
114
Pathogenic
3
Likely Pathogenic
78
VUS
3
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
114
0
114
Likely Pathogenic
0
0
3
0
3
VUS
0
67
11
0
78
Likely Benign
0
3
0
0
3
Benign
0
0
0
0
0
Total0701280198

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

View in ClinVar →

Protein Context — Lollipop Plot

CUL4A · 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
Open Research Assistant →