FOXH1

Chr 8

forkhead box H1

Also known as: FAST-1, FAST1

The FOXH1 protein is a transcriptional activator that binds specific DNA sequences and forms complexes with SMAD2/SMAD4 to regulate TGF-beta and activin signaling pathways, particularly in activating the goosecoid promoter. Mutations in FOXH1 cause holoprosencephaly-12, an autosomal dominant disorder affecting forebrain development. The gene shows tolerance to loss-of-function variants (pLI 0.04, LOEUF 0.84), suggesting that complete loss of function may not be the primary disease mechanism in affected individuals.

ResearchSummary from RefSeq, UniProt
MultiplemechanismLOEUF 0.84
Clinical SummaryFOXH1
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Gene-Disease Validity (ClinGen)
congenital heart disease · ADLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Low constraint (pLI 0.04) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
62 unique Pathogenic / Likely Pathogenic· 209 VUS of 358 total submissions
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 503

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.84LOEUF
pLI 0.042
Z-score 1.93
OE 0.37 (0.180.84)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
-2.55Z-score
OE missense 1.53 (1.391.69)
278 obs / 181.5 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.37 (0.180.84)
00.351.4
Missense OE1.53 (1.391.69)
00.61.4
Synonymous OE1.37
01.21.6
LoF obs/exp: 4 / 10.9Missense obs/exp: 278 / 181.5Syn Z: -2.59
DN
0.6841th %ile
GOF
0.6345th %ile
LOF
0.4038th %ile

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

DNprediction above median
GOFprediction 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

358 submitted variants in ClinVar

Classification Summary

Pathogenic51
Likely Pathogenic11
VUS209
Likely Benign54
Benign22
Conflicting9
51
Pathogenic
11
Likely Pathogenic
209
VUS
54
Likely Benign
22
Benign
9
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
51
0
51
Likely Pathogenic
0
6
5
0
11
VUS
3
163
41
2
209
Likely Benign
0
7
5
42
54
Benign
0
1
14
7
22
Conflicting
9
Total317711651356

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

View in ClinVar →

Protein Context — Lollipop Plot

FOXH1 · 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.

Search ClinicalTrials.gov →
Clinical Literature
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