The EMX2 protein is a homeobox transcription factor that patterns the developing neocortex into functional areas and regulates stereociliary bundle organization in the inner ear. Mutations cause schizencephaly, a rare brain malformation characterized by clefts extending from the brain surface to the ventricles, with inheritance typically following an autosomal dominant pattern. This gene is highly constrained against loss-of-function variants (pLI 0.95), indicating that such mutations are likely to cause significant developmental abnormalities.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismLOEUF 0.321 OMIM phenotype
Clinical SummaryEMX2
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Gene-Disease Validity (ClinGen)
schizencephaly · ADLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.95). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
33 unique Pathogenic / Likely Pathogenic· 37 VUS of 90 total submissions
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GeneReview available — EMX2
Authoritative clinical overview · Recommended first read
Open GeneReview ↗
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.32LOEUF
pLI 0.951
Z-score 2.85
OE 0.00 (0.000.32)
Highly constrained

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

Missense Constraint
1.57Z-score
OE missense 0.64 (0.540.76)
94 obs / 147.6 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.00 (0.000.32)
00.351.4
Missense OE0.64 (0.540.76)
00.61.4
Synonymous OE0.92
01.21.6
LoF obs/exp: 0 / 9.5Missense obs/exp: 94 / 147.6Syn Z: 0.50
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedEMX2-related familial schizencephalyLOFAD
DN
0.4586th %ile
GOF
0.2497th %ile
LOF
0.84top 5%

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

LOFprediction above median · LOEUF 0.32
DN1 literature citation

Literature Evidence

DNA novel nonsense mutation p.E142X was detected in one woman with a didelphic uterus (1 of 517, 0.19%). The results of Western blot analysis confirmed that the mutation caused a truncated protein as predicted, and functional studies proved that it resulted in a dominant negative effect.PMID:25577462

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

90 submitted variants in ClinVar

Classification Summary

Pathogenic29
Likely Pathogenic4
VUS37
Likely Benign15
Benign2
Conflicting2
29
Pathogenic
4
Likely Pathogenic
37
VUS
15
Likely Benign
2
Benign
2
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
1
25
0
29
Likely Pathogenic
0
1
3
0
4
VUS
0
30
5
2
37
Likely Benign
0
9
1
5
15
Benign
0
0
1
1
2
Conflicting
2
Total34135889

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

View in ClinVar →

Protein Context — Lollipop Plot

EMX2 · 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.

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Clinical Literature
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