AGAP1

Chr 2

ArfGAP with GTPase domain, ankyrin repeat and PH domain 1

Also known as: AGAP-1, CENTG2, GGAP1, cnt-g2

This gene encodes a GTPase-activating protein that regulates ARF1 and ARF5, and directly controls adapter protein 3 (AP-3)-dependent trafficking of proteins in the endosomal-lysosomal system. Mutations cause autosomal recessive spastic paraplegia with intellectual disability and thin corpus callosum. The gene is highly constrained against loss-of-function variants (pLI 0.99, LOEUF 0.23), indicating that biallelic mutations are required for disease manifestation.

Summary from RefSeq, UniProt
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0
Active trials
6
Pubs (1 yr)
58
P/LP submissions
0%
P/LP missense
0.23
LOEUF· LoF intol.
LOF
Mechanism· predicted
Clinical SummaryAGAP1
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
58 unique Pathogenic / Likely Pathogenic· 180 VUS of 404 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.23LOEUF
pLI 1.000
Z-score 5.86
OE 0.12 (0.060.23)
Highly constrained

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

Missense Constraint
1.29Z-score
OE missense 0.85 (0.790.91)
481 obs / 567.3 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.12 (0.060.23)
00.351.4
Missense OE0.85 (0.790.91)
00.61.4
Synonymous OE1.13
01.21.6
LoF obs/exp: 6 / 51.3Missense obs/exp: 481 / 567.3Syn Z: -1.64
DN
0.5772th %ile
GOF
0.5759th %ile
LOF
0.65top 25%

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

LOFprediction above median · LOEUF 0.23

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

404 submitted variants in ClinVar

Classification Summary

Pathogenic56
Likely Pathogenic2
VUS180
Likely Benign108
Benign30
Conflicting2
56
Pathogenic
2
Likely Pathogenic
180
VUS
108
Likely Benign
30
Benign
2
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
56
0
56
Likely Pathogenic
0
0
2
0
2
VUS
1
145
34
0
180
Likely Benign
0
5
44
59
108
Benign
1
3
8
18
30
Conflicting
2
Total215314477378

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

View in ClinVar →

Protein Context — Lollipop Plot

AGAP1 · 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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