AGFG1

Chr 2

ArfGAP with FG repeats 1

Also known as: HRB, RAB, RIP

The protein encoded by AGFG1 is required for vesicle docking or fusion during acrosome biogenesis and plays a role in RNA trafficking from the nucleus to cytoplasm. Mutations cause autosomal recessive developmental and epileptic encephalopathy with microcephaly, typically presenting in infancy with seizures and severe developmental delays. This gene is highly constrained against loss-of-function variants, indicating that complete loss of protein function is likely incompatible with normal development.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
0
Pubs (1 yr)
23
P/LP submissions
0%
P/LP missense
0.28
LOEUF· LoF intol.
LOF
Mechanism· predicted
Clinical SummaryAGFG1
Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.99). One damaged copy is likely sufficient to cause disease.
📋
ClinVar Variants
23 unique Pathogenic / Likely Pathogenic· 50 VUS of 108 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.28LOEUF
pLI 0.993
Z-score 4.37
OE 0.11 (0.050.28)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
1.66Z-score
OE missense 0.73 (0.660.82)
225 obs / 306.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.11 (0.050.28)
00.351.4
Missense OE0.73 (0.660.82)
00.61.4
Synonymous OE1.04
01.21.6
LoF obs/exp: 3 / 27.9Missense obs/exp: 225 / 306.9Syn Z: -0.35
DN
0.5280th %ile
GOF
0.4184th %ile
LOF
0.68top 10%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · LOEUF 0.28

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

108 submitted variants in ClinVar

Classification Summary

Pathogenic22
Likely Pathogenic1
VUS50
Likely Benign7
22
Pathogenic
1
Likely Pathogenic
50
VUS
7
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
22
0
22
Likely Pathogenic
0
0
1
0
1
VUS
0
46
4
0
50
Likely Benign
0
4
0
3
7
Benign
0
0
0
0
0
Total05027380

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

View in ClinVar →

Protein Context — Lollipop Plot

AGFG1 · 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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