Recessive dystrophic epidermolysis bullosa-generalized other

disease
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Also known as autosomal recessive dystrophic epidermolysis bullosa generalisata mitisautosomal recessive dystrophic epidermolysis bullosa, generalised otherautosomal recessive dystrophic epidermolysis bullosa, generalized othergeneralised mitis RDEBgeneralized mitis RDEBRDEB generalisata mitisRDEB, generalised intermediateRDEB, generalized intermediateRDEB, non-Hallopeau-Siemens typeRDEB-generalized otherRDEB-Orecessive dystrophic epidermolysis bullosa, generalised intermediaterecessive dystrophic epidermolysis bullosa, non-Hallopeau-Siemens type

Summary

Recessive dystrophic epidermolysis bullosa-generalized other (MONDO:0019522) is a disease with 1 cohort gene.

At a glance

  • Prevalence: Unknown (Worldwide)
  • Cohort genes: 1
  • Phenotypes (HPO): 46

Clinical features

Signs & symptoms

Clinical features (HPO)

46 HPO clinical features (Orphanet curated; top 46 by frequency):

HPO IDTermFrequency
HP:0000987Atypical scarring of skinVery frequent (80-99%)
HP:0001030Fragile skinVery frequent (80-99%)
HP:0001510Growth delayVery frequent (80-99%)
HP:0001903AnemiaVery frequent (80-99%)
HP:0004325Decreased body weightVery frequent (80-99%)
HP:0008066Abnormal blistering of the skinVery frequent (80-99%)
HP:0031903Abnormal circulating selenium concentrationVery frequent (80-99%)
HP:0000670Carious teethFrequent (30-79%)
HP:0001056MiliaFrequent (30-79%)
HP:0001508Failure to thriveFrequent (30-79%)
HP:0001581Recurrent skin infectionsFrequent (30-79%)
HP:0002015DysphagiaFrequent (30-79%)
HP:0002019ConstipationFrequent (30-79%)
HP:0002020Gastroesophageal refluxFrequent (30-79%)
HP:0002031Abnormal esophagus morphologyFrequent (30-79%)
HP:0002043Esophageal strictureFrequent (30-79%)
HP:0003073HypoalbuminemiaFrequent (30-79%)
HP:0003202Skeletal muscle atrophyFrequent (30-79%)
HP:0003234Decreased circulating carnitine concentrationFrequent (30-79%)
HP:0004395MalnutritionFrequent (30-79%)
HP:0007400Irregular hyperpigmentationFrequent (30-79%)
HP:0010450Esophageal stenosisFrequent (30-79%)
HP:0011968Feeding difficultiesFrequent (30-79%)
HP:0031831Decreased serum zincFrequent (30-79%)
HP:0032676Chronic cutaneous woundFrequent (30-79%)
HP:0040303Decreased serum ironFrequent (30-79%)
HP:0100512Low levels of vitamin DFrequent (30-79%)
HP:0200041Skin erosionFrequent (30-79%)
HP:0000160Narrow mouthOccasional (5-29%)
HP:0000572Visual lossOccasional (5-29%)
HP:0000716DepressionOccasional (5-29%)
HP:0000739AnxietyOccasional (5-29%)
HP:0000823Delayed pubertyOccasional (5-29%)
HP:0001057Aplasia cutis congenitaOccasional (5-29%)
HP:0001371Flexion contractureOccasional (5-29%)
HP:0001644Dilated cardiomyopathyOccasional (5-29%)
HP:0001798AnonychiaOccasional (5-29%)
HP:0001802Absent toenailOccasional (5-29%)
HP:0002164Nail dysplasiaOccasional (5-29%)
HP:0003764NevusOccasional (5-29%)
HP:0004057Mitten deformityOccasional (5-29%)
HP:0010296AnkyloglossiaOccasional (5-29%)
HP:0011471Gastrostomy tube feeding in infancyOccasional (5-29%)
HP:0012390Anal fissureOccasional (5-29%)
HP:0200020Corneal erosionOccasional (5-29%)
HP:0002860Squamous cell carcinomaVery rare (<1-4%)

Identifiers

Disease identifiers

FieldValue
Canonical namerecessive dystrophic epidermolysis bullosa-generalized other
Mondo IDMONDO:0019522
Orphanet89842
UMLSC4511044
MedGen1392226
GARD0012794
Is cancer (heuristic)no

Also known as: autosomal recessive dystrophic epidermolysis bullosa generalisata mitis · autosomal recessive dystrophic epidermolysis bullosa, generalised other · autosomal recessive dystrophic epidermolysis bullosa, generalized other · generalised mitis RDEB · generalized mitis RDEB · RDEB generalisata mitis · RDEB, generalised intermediate · RDEB, generalized intermediate · RDEB, non-Hallopeau-Siemens type · RDEB-generalized other · RDEB-O · recessive dystrophic epidermolysis bullosa, generalised intermediate · recessive dystrophic epidermolysis bullosa, non-Hallopeau-Siemens type

Data availability: 1 GenCC gene-disease record.

Disease family

Classification path: disease › human disease › disease by body system or component › integumentary system disorder › skin disordervesiculobullous skin diseaseepidermolysis bullosainherited epidermolysis bullosaepidermolysis bullosa dystrophicarecessive dystrophic epidermolysis bullosa-generalized other

Related subtypes (11): transient bullous dermolysis of the newborn, generalized dominant dystrophic epidermolysis bullosa, pretibial dystrophic epidermolysis bullosa, epidermolysis bullosa dystrophica Neurotrophica, recessive dystrophic epidermolysis bullosa, dystrophic epidermolysis bullosa pruriginosa, acral dystrophic epidermolysis bullosa, dystrophic epidermolysis bullosa, nails only, centripetalis recessive dystrophic epidermolysis bullosa, localized dystrophic epidermolysis bullosa, epidermolysis bullosa dystrophica with subcorneal cleavage

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

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 26 · Orphanet: 9 · 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
COL7A1DefinitiveAutosomal recessiverecessive dystrophic epidermolysis bullosa26

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
COL7A1Orphanet:158673Localized dystrophic epidermolysis bullosa, acral form
COL7A1Orphanet:158676Localized dystrophic epidermolysis bullosa, nails only
COL7A1Orphanet:231568Autosomal dominant generalized dystrophic epidermolysis bullosa
COL7A1Orphanet:79408Autosomal recessive generalized dystrophic epidermolysis bullosa, severe form
COL7A1Orphanet:79409Recessive dystrophic epidermolysis bullosa inversa
COL7A1Orphanet:79410Localized dystrophic epidermolysis bullosa, pretibial form
COL7A1Orphanet:79411Self-improving dystrophic epidermolysis bullosa
COL7A1Orphanet:89842Autosomal recessive generalized dystrophic epidermolysis bullosa, intermediate form
COL7A1Orphanet:89843Dystrophic epidermolysis bullosa pruriginosa

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
COL7A1HGNC:2214ENSG00000114270Q02388Collagen alpha-1(VII) chaingencc

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
COL7A1Collagen alpha-1(VII) chainStratified squamous epithelial basement membrane protein that forms anchoring fibrils which may contribute to epithelial basement membrane organization and adherence by interacting with extracellular matrix (ECM) proteins such as type IV c…

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)
COL7A1Antibody/ImmunoglobulinyesVWF_A, Kunitz_BPTI, FN3_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
skin of abdomen1
skin of leg1
stromal cell of endometrium1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
COL7A1267ubiquitousmarkerstromal cell of endometrium, skin of abdomen, skin of leg

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
COL7A11,767

Structural data

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

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
COL7A1Q02388

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 10. 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
Anchoring fibril formation1761.3×0.007COL7A1
Fibronectin matrix formation1571.0×0.007COL7A1
Laminin interactions1380.7×0.007COL7A1
Cargo concentration in the ER1335.9×0.007COL7A1
Collagen chain trimerization1259.6×0.007COL7A1
Assembly of collagen fibrils and other multimeric structures1200.3×0.007COL7A1
Collagen degradation1175.7×0.007COL7A1
Collagen biosynthesis and modifying enzymes1170.4×0.007COL7A1
COPII-mediated vesicle transport1163.1×0.007COL7A1
Integrin cell surface interactions1134.3×0.007COL7A1

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
endodermal cell differentiation1495.6×0.006COL7A1
epidermis development1210.7×0.007COL7A1
cell adhesion137.5×0.027COL7A1

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
COL7A100

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 drug1COL7A1
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)
COL7A10

Clinical trials & evidence

Clinical trials

Clinical trials: 0.