GSX2

Chr 4AR

GS homeobox 2

Also known as: DMJDS2, GSH2

GSX2 encodes a transcription factor that binds specific DNA sequences and regulates genes critical for brain development, particularly in specifying neuronal fates in the ventral telencephalon and striatal projection neurons. Autosomal recessive mutations cause diencephalic-mesencephalic junction dysplasia syndrome 2, a neurodevelopmental disorder affecting brain structures at the junction between the diencephalon and mesencephalon. This gene is essential for proper ventralization of brain progenitor cells and development of structures including the striatum and olfactory bulb interneurons.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismARLOEUF 0.831 OMIM phenotype
Clinical SummaryGSX2
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Gene-Disease Validity (ClinGen)
diencephalic-mesencephalic junction dysplasia syndrome 2 · ARLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.26) despite low pLI — interpret in context.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.83LOEUF
pLI 0.257
Z-score 1.89
OE 0.26 (0.110.83)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.59Z-score
OE missense 0.87 (0.761.00)
143 obs / 164.3 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.26 (0.110.83)
00.351.4
Missense OE0.87 (0.761.00)
00.61.4
Synonymous OE1.02
01.21.6
LoF obs/exp: 2 / 7.6Missense obs/exp: 143 / 164.3Syn Z: -0.16
DN
0.6551th %ile
GOF
0.4183th %ile
LOF
0.69top 10%

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
DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

GSX2 · 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
Open Research Assistant →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC