HECW2

Chr 2AD

HECT, C2 and WW domain containing E3 ubiquitin protein ligase 2

Also known as: NDHSAL, NEDL2

The protein functions as an E3 ubiquitin ligase that regulates neural crest cell proliferation, migration and differentiation through GDNF/Ret signaling, and also stabilizes endothelial cell junctions via angiomotin-like 1 regulation. Loss-of-function mutations cause autosomal dominant neurodevelopmental disorder with hypotonia, seizures, and absent language. The protein is extremely intolerant to loss-of-function variants, indicating haploinsufficiency as the likely pathogenic mechanism.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
MultiplemechanismADLOEUF 0.261 OMIM phenotype
Clinical SummaryHECW2
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Gene-Disease Validity (ClinGen)
complex neurodevelopmental disorder · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.26LOEUF
pLI 1.000
Z-score 6.98
OE 0.16 (0.100.26)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
3.31Z-score
OE missense 0.69 (0.650.74)
625 obs / 904.8 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.16 (0.100.26)
00.351.4
Missense OE0.69 (0.650.74)
00.61.4
Synonymous OE0.95
01.21.6
LoF obs/exp: 13 / 80.6Missense obs/exp: 625 / 904.8Syn Z: 0.78
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedHECW2-related neurodevelopmental disorder (biallelic)OTHERAR
definitiveHECW2-related neurodevelopmental disorder (monoallelic)OTHERAD
DN
0.3694th %ile
GOF
0.4579th %ile
LOF
0.64top 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 0.26
DN1 literature citation

Literature Evidence

DNThese findings suggest that the dominant negative effects of missense variants around the HECT domain may be the mechanism underlying HECW2-related disorder.PMID:34047014

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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

HECW2 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold
Clinical Literature
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