Spondyloepiphyseal dysplasia with congenital joint dislocations

disease
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Also known as bifurcation of distal humerus with oligoectro-syndactylychondrodysplasia with congenital joint dislocations, CHST3 typechondrodysplasia with multiple dislocationsCHST3-related skeletal dysplasiaGollop Coates syndromeHumerospinal dysostosisSDCD, CHST3 typeSEDCJDspondyloepiphyseal dysplasiaspondyloepiphyseal dysplasia with congenital joint dyslocations, CHST3 type

Summary

Spondyloepiphyseal dysplasia with congenital joint dislocations (MONDO:0007738) is a disease caused by CHST3 (GenCC Definitive), with 2 cohort genes.

At a glance

  • Prevalence: <1 / 1 000 000 (Worldwide) [Orphanet-validated]
  • Causal gene: CHST3 (GenCC Definitive)
  • Cohort genes: 2
  • ClinVar variants: 387
  • Phenotypes (HPO): 26

Clinical features

Epidemiology

Prevalence records

2 prevalence record(s), Orphanet:

TypeClassValueGeographyValidation
Cases/families2WorldwideValidated
Point prevalence<1 / 1 000 000WorldwideValidated

Signs & symptoms

Clinical features (HPO)

26 HPO clinical features (Orphanet curated; top 26 by frequency):

HPO IDTermFrequency
HP:0000316HypertelorismVery frequent (80-99%)
HP:0001156BrachydactylyVery frequent (80-99%)
HP:0001371Flexion contractureVery frequent (80-99%)
HP:0001552Barrel-shaped chestVery frequent (80-99%)
HP:0002515Waddling gaitVery frequent (80-99%)
HP:0002650ScoliosisVery frequent (80-99%)
HP:0002829ArthralgiaVery frequent (80-99%)
HP:0002857Genu valgumVery frequent (80-99%)
HP:0002945Intervertebral space narrowingVery frequent (80-99%)
HP:0002967Cubitus valgusVery frequent (80-99%)
HP:0003037Enlarged jointsVery frequent (80-99%)
HP:0003312Abnormal form of the vertebral bodiesVery frequent (80-99%)
HP:0003521Disproportionate short-trunk short statureVery frequent (80-99%)
HP:0008905RhizomeliaVery frequent (80-99%)
HP:0009811Abnormality of the elbowVery frequent (80-99%)
HP:0010582Irregular epiphysesVery frequent (80-99%)
HP:0010585Small epiphysesVery frequent (80-99%)
HP:0045075Sparse eyebrowVery frequent (80-99%)
HP:0000337Broad foreheadFrequent (30-79%)
HP:0000343Long philtrumFrequent (30-79%)
HP:0000684Delayed eruption of teethFrequent (30-79%)
HP:0001270Motor delayFrequent (30-79%)
HP:0002553Highly arched eyebrowFrequent (30-79%)
HP:0002751KyphoscoliosisFrequent (30-79%)
HP:0010049Short metacarpalFrequent (30-79%)
HP:0030680Abnormal cardiovascular system morphologyFrequent (30-79%)

Identifiers

Disease identifiers

FieldValue
Canonical namespondyloepiphyseal dysplasia with congenital joint dislocations
Mondo IDMONDO:0007738
MeSHC537283
OMIM143095
Orphanet263463
DOIDDOID:0050813
SNOMED CT702400006
UMLSC1837657
MedGen373381
GARD0013169
Is cancer (heuristic)no

Also known as: bifurcation of distal humerus with oligoectro-syndactyly · chondrodysplasia with congenital joint dislocations, CHST3 type · chondrodysplasia with multiple dislocations · CHST3-related skeletal dysplasia · Gollop Coates syndrome · Humerospinal dysostosis · SDCD, CHST3 type · SEDCJD · spondyloepiphyseal dysplasia · spondyloepiphyseal dysplasia with congenital joint dislocations · spondyloepiphyseal dysplasia with congenital joint dyslocations, CHST3 type

Data availability: 387 ClinVar variants · 4 GenCC gene-disease records.

Disease family

Classification path: disease › human disease › disease by developmental or physiological process › metabolic diseasemineral metabolism diseasespondyloepiphyseal dysplasia with congenital joint dislocations

Related subtypes (12): iron metabolism disease, phosphorus metabolism disease, potassium deficiency disease, calcium metabolic disease, diastrophic dysplasia, multiple epiphyseal dysplasia type 4, atelosteogenesis type II, achondrogenesis type IB, chondrodysplasia with joint dislocations, gPAPP type, spondyloepimetaphyseal dysplasia, PAPSS2 type, acquired mineral metabolism disease, sulfur metabolism disease

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

387 retrieved; paginated sample, class counts are floors:

168 uncertain significance, 134 likely benign, 32 pathogenic, 18 likely pathogenic, 12 benign, 12 conflicting classifications of pathogenicity, 6 benign/likely benign, 5 pathogenic/likely pathogenic

ClinVarVariant (HGVS)GeneClassificationReview
1039079NM_004273.5(CHST3):c.334G>T (p.Glu112Ter)CHST3Pathogeniccriteria provided, single submitter
1406206NM_004273.5(CHST3):c.362dup (p.Glu122fs)CHST3Pathogeniccriteria provided, single submitter
1445618NM_004273.5(CHST3):c.271C>T (p.Gln91Ter)CHST3Pathogeniccriteria provided, single submitter
1452559NM_004273.5(CHST3):c.1150_1166del (p.Glu384fs)CHST3Pathogeniccriteria provided, single submitter
1454776NM_004273.5(CHST3):c.763del (p.Leu255fs)CHST3Pathogeniccriteria provided, single submitter
1458337NM_004273.5(CHST3):c.238del (p.Ser79_Leu80insTer)CHST3Pathogeniccriteria provided, single submitter
1474566NM_004273.5(CHST3):c.1177del (p.Pro392_Leu393insTer)CHST3Pathogeniccriteria provided, single submitter
1685635NM_004273.5(CHST3):c.141-1G>CCHST3Pathogeniccriteria provided, single submitter
1996399NM_004273.5(CHST3):c.58_77dup (p.Leu26_Phe27insTer)CHST3Pathogeniccriteria provided, single submitter
2028477NM_004273.5(CHST3):c.375_378dup (p.Ala127fs)CHST3Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2083760NM_004273.5(CHST3):c.83T>A (p.Leu28Ter)CHST3Pathogeniccriteria provided, single submitter
2087753NM_004273.5(CHST3):c.855del (p.Leu286fs)CHST3Pathogeniccriteria provided, single submitter
2136893NM_004273.5(CHST3):c.661C>T (p.Arg221Cys)CHST3Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
225684NM_004273.5(CHST3):c.1063G>A (p.Gly355Arg)CHST3Pathogenicno assertion criteria provided
2428494NM_004273.5(CHST3):c.976dup (p.Asp326fs)CHST3Pathogeniccriteria provided, single submitter
3676659NM_004273.5(CHST3):c.685_686del (p.Cys229fs)CHST3Pathogeniccriteria provided, single submitter
3720938NM_004273.5(CHST3):c.718A>T (p.Lys240Ter)CHST3Pathogeniccriteria provided, single submitter
432012NM_004273.5(CHST3):c.533dup (p.Ala179fs)CHST3Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
4709735NM_004273.5(CHST3):c.802G>T (p.Glu268Ter)CHST3Pathogeniccriteria provided, single submitter
4716158NM_004273.5(CHST3):c.696dup (p.Val233fs)CHST3Pathogeniccriteria provided, single submitter
4742464NM_004273.5(CHST3):c.723dup (p.Phe242fs)CHST3Pathogeniccriteria provided, single submitter
478822NM_004273.5(CHST3):c.904G>C (p.Asp302His)CHST3Pathogeniccriteria provided, single submitter
478823NM_004273.5(CHST3):c.491C>T (p.Pro164Leu)CHST3Pathogeniccriteria provided, single submitter
522996NM_004273.5(CHST3):c.1312C>T (p.Gln438Ter)CHST3Pathogeniccriteria provided, single submitter
6041NM_004273.5(CHST3):c.776T>C (p.Leu259Pro)CHST3Pathogenicno assertion criteria provided
6042NM_004273.5(CHST3):c.1114G>A (p.Glu372Lys)CHST3Pathogenicno assertion criteria provided
6043NM_004273.5(CHST3):c.664C>T (p.Arg222Trp)CHST3Pathogenicno assertion criteria provided
6044NM_004273.5(CHST3):c.920T>C (p.Leu307Pro)CHST3Pathogenicno assertion criteria provided
6045NM_004273.5(CHST3):c.1086del (p.Arg363fs)CHST3Pathogenicno assertion criteria provided
6046NM_004273.5(CHST3):c.603C>A (p.Tyr201Ter)CHST3Pathogenicno assertion criteria provided

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 4 · Orphanet: 2 · 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
CHST3DefinitiveAutosomal recessivespondyloepiphyseal dysplasia with congenital joint dislocations4

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
CHST3Orphanet:263463CHST3-related skeletal dysplasia
MT-TVOrphanet:255210Mitochondrial DNA-associated Leigh syndrome

Cohort genes → proteins

2 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence2

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
CHST3HGNC:1971ENSG00000122863Q7LGC8Carbohydrate sulfotransferase 3gencc,clinvar
MT-TVHGNC:7500ENSG00000210077mitochondrially encoded tRNA-Val (GUN)clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
CHST3Carbohydrate sulfotransferase 3Sulfotransferase that utilizes 3’-phospho-5’-adenylyl sulfate (PAPS) as sulfonate donor to catalyze the transfer of sulfate to position 6 of the N-acetylgalactosamine (GalNAc) residue of chondroitin.

Protein-family classification

Druggable: 1 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.5

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
Enzyme (other)16.0×0.320
Other/Unknown10.9×0.805

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
CHST3Enzyme (other)yes2.8.2.17Sulfotransferase_dom, Carbohydrate_sulfotransferase, P-loop_NTPase
MT-TVOther/Unknownno

Expression context

Cohort genes with no expression data: 0.

2 cohort genes 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)2
unknown0

Top tissues across cohort

TissueCohort genes
cartilage tissue1
tibia1
ventricular zone1
amygdala1
prefrontal cortex1
sural nerve1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
CHST3233ubiquitousmarkertibia, cartilage tissue, ventricular zone
MT-TV118ubiquitousmarkersural nerve, prefrontal cortex, amygdala

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
CHST3579
MT-TV0

Structural data

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

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
CHST3Q7LGC880.58

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 3. Enrichment computed across 2 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
Defective CHST3 causes SEDCJD11427.5×0.002CHST3
CS-GAG biosynthesis1543.8×0.003CHST3
Keratan sulfate biosynthesis1380.7×0.003CHST3

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
N-acetylglucosamine metabolic process11203.7×0.002CHST3
sulfur compound metabolic process11123.5×0.002CHST3
keratan sulfate proteoglycan biosynthetic process1991.3×0.002CHST3
chondroitin sulfate proteoglycan biosynthetic process1624.1×0.002CHST3
T cell homeostasis1455.5×0.003CHST3
carbohydrate metabolic process1135.9×0.007CHST3

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 2

Druggability breadth: 0 of 2 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
CHST300
MT-TV00

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort enzymes (BRENDA EC)

SymbolEC numbersNames
CHST32.8.2.17chondroitin 6-sulfotransferase

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; 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 drug1CHST3
EDifficult family or no structure, no drug1MT-TV

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
CHST30
MT-TV0

Clinical trials & evidence

Clinical trials

Clinical trials: 0.