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.

Summary from RefSeq, UniProt
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0
Active trials
6
Pubs (1 yr)
0
P/LP submissions
P/LP missense
0.84
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryFOXH1
Population Constraint (gnomAD)
Low constraint (pLI 0.04) — loss-of-function variants are relatively tolerated in the population.

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

0 submitted variants 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.

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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