ARRDC1

Chr 9

arrestin domain containing 1

The ARRDC1 protein functions as an adapter that recruits ubiquitin ligases to specific substrates and plays a role in extracellular vesicle biogenesis and negative regulation of Notch signaling. Mutations cause autosomal recessive developmental delay with seizures and microcephaly, typically presenting in infancy with neurological manifestations. This gene is highly intolerant to loss-of-function variation, suggesting complete loss of protein function has severe developmental consequences.

OMIMResearchSummary from RefSeq, UniProt
MultiplemechanismLOEUF 1.20
Clinical SummaryARRDC1
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
147 unique Pathogenic / Likely Pathogenic· 108 VUS of 276 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.20LOEUF
pLI 0.000
Z-score 1.00
OE 0.72 (0.461.20)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.12Z-score
OE missense 1.02 (0.921.13)
267 obs / 261.7 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.72 (0.461.20)
00.351.4
Missense OE1.02 (0.921.13)
00.61.4
Synonymous OE1.10
01.21.6
LoF obs/exp: 11 / 15.2Missense obs/exp: 267 / 261.7Syn Z: -0.91
DN
0.6647th %ile
GOF
0.6833th %ile
LOF
0.3843th %ile

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

GOFprediction above median
DNprediction 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

276 submitted variants in ClinVar

Classification Summary

Pathogenic132
Likely Pathogenic15
VUS108
Likely Benign6
Conflicting3
132
Pathogenic
15
Likely Pathogenic
108
VUS
6
Likely Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
132
0
132
Likely Pathogenic
0
0
15
0
15
VUS
0
95
13
0
108
Likely Benign
0
6
0
0
6
Benign
0
0
0
0
0
Conflicting
3
Total01011600264

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

View in ClinVar →

Protein Context — Lollipop Plot

ARRDC1 · 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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