FTH1

Chr 11AD

ferritin heavy chain 1

Also known as: FHC, FTH, FTHL6, HFE5, NBIA9, PIG15, PLIF

The ferritin heavy chain stores iron in a soluble, non-toxic form and has ferroxidase activity that oxidizes ferrous iron to ferric iron for cellular iron homeostasis. Mutations cause neurodegeneration with brain iron accumulation 9 and hemochromatosis type 5, both involving abnormal iron metabolism affecting the brain and other organs. Inheritance is autosomal dominant.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.782 OMIM phenotypes
Clinical SummaryFTH1
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Gene-Disease Validity (ClinGen)
hemochromatosis type 5 · ADLimited

Limited evidence — not for standalone diagnostic reporting

2 total gene-disease associations curated

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.30) despite low pLI — interpret in context.
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ClinVar Variants
19 unique Pathogenic / Likely Pathogenic· 46 VUS of 102 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.78LOEUF
pLI 0.140
Z-score 2.05
OE 0.30 (0.140.78)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.86Z-score
OE missense 0.75 (0.620.91)
72 obs / 95.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.30 (0.140.78)
00.351.4
Missense OE0.75 (0.620.91)
00.61.4
Synonymous OE1.17
01.21.6
LoF obs/exp: 3 / 10.0Missense obs/exp: 72 / 95.8Syn Z: -0.88
DN
0.80top 10%
GOF
0.6638th %ile
LOF
0.2582th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). The Badonyi & Marsh model scores dominant-negative highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports gain-of-function. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median · 1 literature citation
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.

Literature Evidence

GOFFTH1 pathogenic variants appear to act by a dominant, toxic gain-of-function mechanism.PMID:36778397

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

ClinVar Variant Classifications

102 submitted variants in ClinVar

Classification Summary

Pathogenic10
Likely Pathogenic9
VUS46
Likely Benign13
Benign14
Conflicting3
10
Pathogenic
9
Likely Pathogenic
46
VUS
13
Likely Benign
14
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
0
7
0
10
Likely Pathogenic
0
5
4
0
9
VUS
3
28
14
1
46
Likely Benign
0
2
3
8
13
Benign
0
0
11
3
14
Conflicting
3
Total635391295

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

View in ClinVar →

Protein Context — Lollipop Plot

FTH1 · 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
Open Research Assistant →