KCTD1

Chr 18AD

potassium channel tetramerization domain containing 1

Also known as: C18orf5, DNRD, RMDA

The protein negatively regulates AP-2 transcription factors and the Wnt signaling pathway by enhancing ubiquitination and degradation of beta-catenin. Mutations cause scalp-ear-nipple syndrome and dental radicular dysplasia with autosomal dominant inheritance. The gene is highly constrained against loss-of-function variants (pLI 0.86, LOEUF 0.44), indicating intolerance to protein-disrupting mutations.

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

Dental radicular dysplasiaMIM #621434
AD
Scalp-ear-nipple syndromeMIM #181270
AD
0
Active trials
8
Pubs (1 yr)
64
P/LP submissions
25%
P/LP missense
0.44
LOEUF
GOF*
Mechanism· G2P
Clinical SummaryKCTD1
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.86) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
53 unique Pathogenic / Likely Pathogenic· 155 VUS of 269 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.44LOEUF
pLI 0.855
Z-score 2.75
OE 0.09 (0.030.44)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.82Z-score
OE missense 0.59 (0.500.70)
95 obs / 159.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.09 (0.030.44)
00.351.4
Missense OE0.59 (0.500.70)
00.61.4
Synonymous OE0.81
01.21.6
LoF obs/exp: 1 / 10.7Missense obs/exp: 95 / 159.9Syn Z: 1.19
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedKCTD1-related scalp-ear-nipple syndromeGOFAD
DN
0.4785th %ile
GOF
0.3986th %ile
LOF
0.65top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-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 0.44
DN1 literature citation

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

DNKCTD1 mutations associated with SEN syndrome abrogate KCTD1 transcriptional repression activity on transcription factor AP-2a (13). These missense mutations result in the loss of KCTD1 function in a dominant-negative manner.PMID:33000225

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

269 submitted variants in ClinVar

Classification Summary

Pathogenic49
Likely Pathogenic4
VUS155
Likely Benign32
Benign19
Conflicting5
49
Pathogenic
4
Likely Pathogenic
155
VUS
32
Likely Benign
19
Benign
5
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
10
39
0
49
Likely Pathogenic
0
3
1
0
4
VUS
1
138
15
1
155
Likely Benign
0
4
7
21
32
Benign
0
1
15
3
19
Conflicting
5
Total11567725264

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

View in ClinVar →

Protein Context — Lollipop Plot

KCTD1 · 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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Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗