AIRE

Chr 21ADAR

autoimmune regulator

Also known as: AIRE1, APECED, APS1, APSI, PGA1

The protein encoded by this gene is a transcriptional regulator that promotes immune self-tolerance by controlling the expression of tissue-restricted antigens in the thymus, allowing developing T-cells to recognize and eliminate autoreactive cells. Mutations cause autoimmune polyendocrinopathy syndrome type I (APECED), a rare autosomal recessive disorder characterized by chronic mucocutaneous candidiasis, hypoparathyroidism, and adrenal insufficiency, typically presenting in childhood. The gene is extremely intolerant to loss-of-function variants, reflecting its critical role in preventing autoimmunity.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Autoimmune polyendocrinopathy syndrome , type I, with or without reversible metaphyseal dysplasiaMIM #240300
ADAR
2
Active trials
110
Pubs (1 yr)
72
P/LP submissions
7%
P/LP missense
1.11
LOEUF
LOF
Mechanism· G2P
Clinical SummaryAIRE
🧬
Gene-Disease Validity (ClinGen)
autoimmune polyendocrine syndrome type 1 · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
68 unique Pathogenic / Likely Pathogenic· 199 VUS of 500 total submissions
💊
Clinical Trials
2 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — AIRE
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.11LOEUF
pLI 0.000
Z-score 1.09
OE 0.77 (0.551.11)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.00Z-score
OE missense 1.00 (0.921.09)
351 obs / 350.9 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.77 (0.551.11)
00.351.4
Missense OE1.00 (0.921.09)
00.61.4
Synonymous OE1.01
01.21.6
LoF obs/exp: 21 / 27.1Missense obs/exp: 351 / 350.9Syn Z: -0.13
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveAIRE-related autoimmune polyendocrinopathy syndromeLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.4686th %ile
GOF
0.5758th %ile
LOF
0.4332th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Literature Evidence

DNIlmarinen et al. (2005) concluded that the G228W protein acts with a dominant-negative effect by binding to wildtype AIRE, preventing the protein from forming the complexes needed for transactivation.PMID:16114041

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

500 submitted variants in ClinVar

Classification Summary

Pathogenic44
Likely Pathogenic24
VUS199
Likely Benign221
Benign4
Conflicting5
44
Pathogenic
24
Likely Pathogenic
199
VUS
221
Likely Benign
4
Benign
5
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
16
1
27
0
44
Likely Pathogenic
16
4
4
0
24
VUS
1
164
26
8
199
Likely Benign
0
1
120
100
221
Benign
0
0
4
0
4
Conflicting
5
Total33170181108497

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

View in ClinVar →

Protein Context — Lollipop Plot

AIRE · protein map & ClinVar variants

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

Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗