This gene encodes OTOP1, a proton-selective ion channel that is essential for the formation of otoconia, the calcium carbonate structures in the inner ear that detect gravity and linear acceleration. Mutations cause autosomal recessive benign paroxysmal positional vertigo type 2, characterized by episodic vertigo and balance problems due to absent or malformed otoconia. The gene shows minimal constraint against loss-of-function variants, consistent with its recessive inheritance pattern.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
17
Pubs (1 yr)
81
P/LP submissions
0%
P/LP missense
1.88
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryOTOP1
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
81 unique Pathogenic / Likely Pathogenic· 110 VUS of 200 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.88LOEUF
pLI 0.000
Z-score -1.95
OE 1.47 (1.091.88)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.66Z-score
OE missense 1.10 (1.011.21)
356 obs / 322.4 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE1.47 (1.091.88)
00.351.4
Missense OE1.10 (1.011.21)
00.61.4
Synonymous OE1.03
01.21.6
LoF obs/exp: 30 / 20.5Missense obs/exp: 356 / 322.4Syn Z: -0.31
DN
0.6453th %ile
GOF
0.77top 25%
LOF
0.4135th %ile

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

GOFprediction above median
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.

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

ClinVar Variant Classifications

200 submitted variants in ClinVar

Classification Summary

Pathogenic77
Likely Pathogenic4
VUS110
Likely Benign5
Benign2
77
Pathogenic
4
Likely Pathogenic
110
VUS
5
Likely Benign
2
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
77
0
77
Likely Pathogenic
0
0
4
0
4
VUS
0
110
0
0
110
Likely Benign
0
5
0
0
5
Benign
0
0
2
0
2
Total0115830198

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

View in ClinVar →

Protein Context — Lollipop Plot

OTOP1 · 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
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 1 results · since 2015Search PubMed ↗