GLI3

Chr 7AD

GLI family zinc finger 3

Also known as: ACLS, GCPS, GLI3-190, GLI3FL, PAP-A, PAPA, PAPA1, PAPB

This gene encodes GLI3, a zinc finger transcription factor that acts as both an activator and repressor in the sonic hedgehog signaling pathway and is essential for proper limb development and digit formation. Mutations cause autosomal dominant conditions including Greig cephalopolysyndactyly syndrome, Pallister-Hall syndrome, and various forms of polydactyly, all characterized by limb malformations and often craniofacial abnormalities. The gene is highly constrained against loss-of-function variants in the general population, reflecting its critical role in development.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.204 OMIM phenotypes
Clinical SummaryGLI3
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.
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ClinVar Variants
8 unique Pathogenic / Likely Pathogenic· 71 VUS of 100 total submissions
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GeneReview available — GLI3
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.20LOEUF
pLI 1.000
Z-score 6.17
OE 0.09 (0.050.20)
Highly constrained

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

Missense Constraint
0.52Z-score
OE missense 0.95 (0.901.01)
932 obs / 978.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.09 (0.050.20)
00.351.4
Missense OE0.95 (0.901.01)
00.61.4
Synonymous OE1.12
01.21.6
LoF obs/exp: 5 / 53.8Missense obs/exp: 932 / 978.0Syn Z: -2.02
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveGLI3-related Pallister-Hall syndromeDNAD
definitiveGLI3-related Greig cephalopolysyndactyly syndromeLOFAD
DN
0.3892th %ile
GOF
0.2198th %ile
LOF
0.83top 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 · 1 literature citation · 75% of P/LP variants are LoF · LOEUF 0.20
DN1 literature citation

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.

Literature Evidence

DNGenotype/phenotype correlations have led to fine mapping of GLI3 and the recognition that PHS is caused by dominant negative mutations in the middle third of the gene.PMID:25424727
LOFGCPS is known to be caused by mutations in the transcription factor GLI3 gene (7p13) which results in functional haploinsufficiency of this gene.PMID:25606469

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

100 submitted variants in ClinVar

Classification Summary

Pathogenic4
Likely Pathogenic4
VUS71
Likely Benign7
Conflicting3
4
Pathogenic
4
Likely Pathogenic
71
VUS
7
Likely Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
4
0
0
0
4
Likely Pathogenic
2
2
0
0
4
VUS
1
69
1
0
71
Likely Benign
0
4
0
3
7
Benign
0
0
0
0
0
Conflicting
3
Total7751389

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

View in ClinVar →

Protein Context — Lollipop Plot

GLI3 · 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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