FAM83H

Chr 8AD

scaffolding CK1 anchoring protein H

Also known as: AI3, AI3A, FAM83H

The protein encoded by this gene plays a major role in the structural organization and calcification of developing tooth enamel and may regulate epithelial cell migration through keratin cytoskeleton disassembly. Mutations cause amelogenesis imperfecta type IIIA, a dental disorder affecting enamel formation, with autosomal dominant inheritance. The gene is highly constrained against loss-of-function variants (pLI 0.89, LOEUF 0.36), indicating intolerance to protein disruption.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

Amelogenesis imperfecta, type IIIAMIM #130900
AD
Amelogenesis imperfecta, type IIIAMIM #130900
AD
0
Active trials
17
Pubs (1 yr)
50
P/LP submissions
0%
P/LP missense
0.36
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryFAM83H
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.89) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
49 unique Pathogenic / Likely Pathogenic· 279 VUS of 405 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.36LOEUF
pLI 0.892
Z-score 4.16
OE 0.17 (0.090.36)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
-0.42Z-score
OE missense 1.04 (0.981.10)
824 obs / 791.0 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.17 (0.090.36)
00.351.4
Missense OE1.04 (0.981.10)
00.61.4
Synonymous OE1.14
01.21.6
LoF obs/exp: 5 / 29.3Missense obs/exp: 824 / 791.0Syn Z: -2.10
DN
0.4587th %ile
GOF
0.5661th %ile
LOF
0.67top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function, gain-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 · LOEUF 0.36
GOF1 literature citation
DN1 literature citation

Literature Evidence

DNHart et al. (2009) noted that all of the FAM83H mutations identified in AI3A thus far were nonsense mutations that clustered within a 380-amino acid region, supporting a dominant-negative effect.PMID:19220331
GOFADHCAI is likely caused by gain-of-function effects mediated by truncated FAM83H, which potentially mislocalizes CK1 as part of its pathological mechanism.PMID:26788537

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

405 submitted variants in ClinVar

Classification Summary

Pathogenic41
Likely Pathogenic8
VUS279
Likely Benign33
Benign29
Conflicting1
41
Pathogenic
8
Likely Pathogenic
279
VUS
33
Likely Benign
29
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
41
0
41
Likely Pathogenic
3
0
5
0
8
VUS
2
273
4
0
279
Likely Benign
0
14
5
14
33
Benign
0
14
6
9
29
Conflicting
1
Total53016123391

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

View in ClinVar →

Protein Context — Lollipop Plot

FAM83H · 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.

Search ClinicalTrials.gov →
Clinical Literature
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