NEDD4L

Chr 18AD

NEDD4 like E3 ubiquitin protein ligase

Also known as: NEDD4-2, NEDD4.2, PVNH7, RSP5, hNEDD4-2

The protein is a HECT domain E3 ubiquitin ligase that transfers ubiquitin to target proteins for lysosomal degradation and critically regulates epithelial sodium channel (ENaC) cell surface expression to control epithelial sodium transport. Mutations cause periventricular nodular heterotopia 7, inherited in an autosomal dominant pattern. The pathogenic mechanism involves loss of function, as evidenced by the gene's extreme intolerance to loss-of-function variants.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.201 OMIM phenotype
Clinical SummaryNEDD4L
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Gene-Disease Validity (ClinGen)
periventricular nodular heterotopia 7 · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

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.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.20LOEUF
pLI 1.000
Z-score 6.75
OE 0.11 (0.060.20)
Highly constrained

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

Missense Constraint
3.73Z-score
OE missense 0.55 (0.510.61)
308 obs / 555.0 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.11 (0.060.20)
00.351.4
Missense OE0.55 (0.510.61)
00.61.4
Synonymous OE0.94
01.21.6
LoF obs/exp: 7 / 66.3Missense obs/exp: 308 / 555.0Syn Z: 0.69
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongNEDD4L-related periventricular nodular heterotopia with intellectual disability, cleft palate, and 2-3 toe syndactylyOTHERAD
DN
0.5082th %ile
GOF
0.6248th %ile
LOF
0.58top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function and dominant-negative). The Badonyi & Marsh model scores gain-of-function highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports loss-of-function (haploinsufficiency). Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOFLOEUF 0.20
DN1 literature citation

Literature Evidence

DNWe show that a recombinant KIAA0439 WW domain protein acts as a dominant negative mutant that can interfere with the Na(+)-dependent feedback inhibition of ENaC in whole-cell patch clamp experiments.PMID:11244092

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

NEDD4L · 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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