AP2S1

Chr 19AD

adaptor related protein complex 2 subunit sigma 1

Also known as: AP17, CLAPS2, FBH3, FBHOk, HHC3

The AP2S1 protein is a component of the adaptor protein complex 2 (AP-2) that functions in clathrin-dependent endocytosis, selectively sorting membrane proteins and forming clathrin-coated vesicles for transport to early endosomes. Mutations cause hypocalciuric hypercalcemia type III, an autosomal dominant disorder affecting calcium homeostasis. The gene shows high constraint against loss-of-function variants (LOEUF 0.502), indicating intolerance to protein-truncating mutations.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

Hypocalciuric hypercalcemia, type IIIMIM #600740
AD
1
Active trials
13
Pubs (1 yr)
40
P/LP submissions
23%
P/LP missense
0.50
LOEUF
GOF
Mechanism· predicted
Clinical SummaryAP2S1
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.79) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
13 unique Pathogenic / Likely Pathogenic· 44 VUS of 159 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
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GeneReview available — AP2S1
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.50LOEUF
pLI 0.785
Z-score 2.55
OE 0.11 (0.040.50)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.86Z-score
OE missense 0.16 (0.110.25)
15 obs / 92.4 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.11 (0.040.50)
00.351.4
Missense OE0.16 (0.110.25)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 1 / 9.4Missense obs/exp: 15 / 92.4Syn Z: 0.06
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
moderateAP2S1-related developmental disorderOTHERAD
DN
0.5575th %ile
GOF
0.5366th %ile
LOF
0.4627th %ile

The Badonyi & Marsh model scores dominant-negative highest, but genomic evidence most strongly supports gain-of-function as the primary mechanism.

GOF1 literature citation

Literature Evidence

GOFLoss-of-function mutations of adaptor protein-2 sigma subunit (AP2o 2), encoded by AP2S1, cause FHH3, and we therefore sought for gain-of-function AP2S1 mutations that may cause an additional form of ADH, which we designated ADH3.PMID:24708097

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

159 submitted variants in ClinVar

Classification Summary

Pathogenic10
Likely Pathogenic3
VUS44
Likely Benign81
Benign10
Conflicting4
10
Pathogenic
3
Likely Pathogenic
44
VUS
81
Likely Benign
10
Benign
4
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
2
8
0
10
Likely Pathogenic
0
1
2
0
3
VUS
0
29
15
0
44
Likely Benign
0
4
42
35
81
Benign
0
1
9
0
10
Conflicting
4
Total0377635152

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

View in ClinVar →

Protein Context — Lollipop Plot

AP2S1 · protein map & ClinVar variants

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

Clinical Literature
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