Epilepsy, juvenile absence, susceptibility to, 1

disease
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Also known as EJA1epilepsy, juvenile absence, susceptibility to, type 1JAE1susceptibility to juvenile absence epilepsy 1

Summary

Epilepsy, juvenile absence, susceptibility to, 1 (MONDO:0020772) is a disease with 1 cohort gene.

At a glance

  • Cohort genes: 1
  • ClinVar variants: 4

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nameepilepsy, juvenile absence, susceptibility to, 1
Mondo IDMONDO:0020772
OMIM607631
DOIDDOID:0111324
UMLSC2750892
MedGen413426
Is cancer (heuristic)no

Also known as: EJA1 · EPILEPSY, JUVENILE ABSENCE, SUSCEPTIBILITY TO, 1 · epilepsy, juvenile absence, susceptibility to, type 1 · JAE1 · susceptibility to juvenile absence epilepsy 1

Data availability: 4 ClinVar variants.

Disease family

Classification path: disease susceptibility › inherited disease susceptibility › epilepsy, juvenile absence, susceptibility to › epilepsy, juvenile absence, susceptibility to, 1

Related subtypes (1): epilepsy, idiopathic generalized, susceptibility to, 11

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

4 retrieved; paginated sample, class counts are floors:

3 uncertain significance, 1 conflicting classifications of pathogenicity

ClinVarVariant (HGVS)GeneClassificationReview
205403NM_018100.4(EFHC1):c.1114C>T (p.Arg372Trp)EFHC1Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
3891408NM_018100.4(EFHC1):c.1406_1431dup (p.Thr478fs)EFHC1Uncertain significancecriteria provided, single submitter
4533280NM_018100.4(EFHC1):c.1307G>A (p.Arg436His)EFHC1Uncertain significancecriteria provided, single submitter
837595NM_018100.4(EFHC1):c.742A>G (p.Ile248Val)EFHC1Uncertain significancecriteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 2 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
EFHC1Orphanet:1941Juvenile absence epilepsy
EFHC1Orphanet:307Juvenile myoclonic epilepsy

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
EFHC1HGNC:16406ENSG00000096093Q5JVL4EF-hand domain-containing protein 1clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
EFHC1EF-hand domain-containing protein 1Microtubule inner protein (MIP) part of the dynein-decorated doublet microtubules (DMTs) in cilia axoneme, which is required for motile cilia beating.

Protein-family classification

Druggable: 0 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.0

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
Other/Unknown11.8×0.558

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
EFHC1Other/UnknownnoEF_hand_dom, DM10_dom, EF-hand-dom_pair

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
bronchial epithelial cell1
bronchus1
epithelium of bronchus1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
EFHC1272ubiquitousmarkerbronchial epithelial cell, epithelium of bronchus, bronchus

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
EFHC12,470

Structural data

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

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
EFHC1Q5JVL42

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 0. Enrichment computed across 1 evidence-associated genes (0 with Reactome annotation).

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
cerebral cortex cell migration11532.0×0.002EFHC1
cilium-dependent cell motility11404.3×0.002EFHC1
regulation of cell division1766.0×0.003EFHC1
mitotic spindle organization1271.8×0.005EFHC1
mitotic cytokinesis1259.3×0.005EFHC1
flagellated sperm motility1117.0×0.009EFHC1

Therapeutics

Drug target analysis

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

Druggability breadth: 0 of 1 evidence-associated genes (0%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
EFHC100

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

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 drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug1EFHC1

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
EFHC10

Clinical trials & evidence

Clinical trials

Clinical trials: 0.