KLHL20

Chr 1AD

kelch like family member 20

The protein functions as a substrate-specific adapter of a BCR E3 ubiquitin ligase complex that regulates protein degradation, Golgi to endosome transport, and neurite outgrowth. Mutations cause a neurodevelopmental disorder with early-onset seizures, facial dysmorphism, and behavioral abnormalities, inherited in an autosomal dominant pattern. The gene shows moderate constraint against loss-of-function variants (LOEUF 0.511), consistent with its role in early neurodevelopment.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.511 OMIM phenotype
Clinical SummaryKLHL20
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Gene-Disease Validity (ClinGen)
complex neurodevelopmental disorder · ADModerate

Moderate evidence — consider for supplementary testing

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.30) despite low pLI — interpret in context.
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Missense constrained — critical functional residues
LoF Constraint
0.51LOEUF
pLI 0.009
Z-score 3.73
OE 0.30 (0.180.51)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
3.61Z-score
OE missense 0.47 (0.410.53)
168 obs / 361.2 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.30 (0.180.51)
00.351.4
Missense OE0.47 (0.410.53)
00.61.4
Synonymous OE1.00
01.21.6
LoF obs/exp: 10 / 33.2Missense obs/exp: 168 / 361.2Syn Z: -0.02
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
moderateKLHL20-related developmental disorder with seizuresOTHERAD
DN
0.7230th %ile
GOF
0.6639th %ile
LOF
0.3550th %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

KLHL20 · 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.

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Clinical Literature
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