Ectodermal dysplasia-syndactyly syndrome 1

disease
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Also known as ectodermal dysplasia-syndactyly syndrome caused by mutation in NECTIN4EDSS1NECTIN4 ectodermal dysplasia-syndactyly syndrome

Summary

Ectodermal dysplasia-syndactyly syndrome 1 (MONDO:0024565) is a disease caused by NECTIN4 (GenCC Definitive), with 1 cohort gene.

At a glance

  • Causal gene: NECTIN4 (GenCC Definitive)
  • Cohort genes: 1
  • ClinVar variants: 14

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nameectodermal dysplasia-syndactyly syndrome 1
Mondo IDMONDO:0024565
OMIM613573
UMLSC3150807
MedGen462157
GARD0025433
Is cancer (heuristic)no

Also known as: ectodermal dysplasia-syndactyly syndrome 1 · ectodermal dysplasia-syndactyly syndrome caused by mutation in NECTIN4 · EDSS1 · NECTIN4 ectodermal dysplasia-syndactyly syndrome

Data availability: 14 ClinVar variants · 3 GenCC gene-disease records.

Disease family

Classification path: disease › human disease › disease by body system or component › syndromic diseaseectodermal dysplasia syndromeectodermal dysplasia-syndactyly syndromeectodermal dysplasia-syndactyly syndrome 1

Related subtypes (1): ectodermal dysplasia-cutaneous syndactyly syndrome

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

14 retrieved; paginated sample, class counts are floors:

8 pathogenic, 4 uncertain significance, 1 likely pathogenic, 1 conflicting classifications of pathogenicity

ClinVarVariant (HGVS)GeneClassificationReview
1600NM_030916.3(NECTIN4):c.851G>A (p.Arg284Gln)NECTIN4Pathogenicno assertion criteria provided
1601NM_030916.3(NECTIN4):c.554C>T (p.Thr185Met)NECTIN4Pathogenicno assertion criteria provided
1602NM_030916.3(NECTIN4):c.906del (p.Pro304fs)NECTIN4Pathogenicno assertion criteria provided
30794NM_030916.3(NECTIN4):c.635C>G (p.Pro212Arg)NECTIN4Pathogenicno assertion criteria provided
425551NM_030916.3(NECTIN4):c.724G>A (p.Val242Met)NECTIN4Pathogenicno assertion criteria provided
425552NM_030916.3(NECTIN4):c.181C>T (p.Gln61Ter)NECTIN4Pathogenicno assertion criteria provided
807665NM_030916.3(NECTIN4):c.229C>T (p.Gln77Ter)NECTIN4Pathogeniccriteria provided, single submitter
996828NM_030916.3(NECTIN4):c.880C>T (p.Arg294Ter)NECTIN4Pathogeniccriteria provided, single submitter
3393124NM_030916.3(NECTIN4):c.1516C>T (p.Arg506Trp)NECTIN4Likely pathogeniccriteria provided, single submitter
418440NM_030916.3(NECTIN4):c.1327C>T (p.Arg443Cys)NECTIN4Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
1029983NM_030916.3(NECTIN4):c.1478G>C (p.Arg493Pro)NECTIN4Uncertain significancecriteria provided, single submitter
1032240NM_030916.3(NECTIN4):c.1387C>G (p.Pro463Ala)NECTIN4Uncertain significancecriteria provided, multiple submitters, no conflicts
2434319NM_030916.3(NECTIN4):c.1490C>T (p.Thr497Met)NECTIN4Uncertain significancecriteria provided, multiple submitters, no conflicts
3774316NM_030916.3(NECTIN4):c.374A>C (p.Tyr125Ser)NECTIN4Uncertain significancecriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 4 · Orphanet: 1 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

GeneClassificationInheritanceDiseaseRecords
NECTIN4DefinitiveAutosomal recessiveectodermal dysplasia-syndactyly syndrome 14

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
NECTIN4Orphanet:247820Ectodermal dysplasia-pili torti-cutaneous syndactyly syndrome

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
NECTIN4HGNC:19688ENSG00000143217Q96NY8Nectin-4gencc,clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
NECTIN4Nectin-4Seems to be involved in cell adhesion through trans-homophilic and -heterophilic interactions, the latter including specifically interactions with NECTIN1.

Protein-family classification

Druggable: 1 · Difficult: 0 · Unknown: 0 · Druggable fraction: 1.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Antibody/Immunoglobulin129.2×0.034

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
NECTIN4Antibody/ImmunoglobulinyesIg_sub2, Ig_sub, Ig-like_dom

Expression context

Cohort genes with no expression data: 0.

1 cohort gene are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)1
unknown0

Top tissues across cohort

TissueCohort genes
lower esophagus mucosa1
skin of abdomen1
skin of leg1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
NECTIN4160broadmarkerlower esophagus mucosa, skin of abdomen, skin of leg

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
NECTIN4871

Structural data

PDB: 1 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
NECTIN4Q96NY85

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 5. Enrichment computed across 1 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Nectin/Necl trans heterodimerization11427.5×0.004NECTIN4
Adherens junctions interactions1248.3×0.007NECTIN4
Cell-cell junction organization1248.3×0.007NECTIN4
Cell junction organization1187.2×0.007NECTIN4
Cell-Cell communication1137.6×0.007NECTIN4

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
negative regulation of natural killer cell mediated cytotoxicity1887.0×0.003NECTIN4
heterophilic cell-cell adhesion1337.0×0.004NECTIN4
homophilic cell-cell adhesion1140.4×0.007NECTIN4

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 1

Druggability breadth: 1 of 1 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
NECTIN400

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
NECTIN44Binding:4

Pharmacogenomics

Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)0
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug1NECTIN4
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug0

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
NECTIN44

Clinical trials & evidence

Clinical trials

Clinical trials: 0.