transcobalamin I deficiency

disease
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Also known as Haptocorrin deficiencyTCI deficiencytranscobalamin 1 deficiencytranscobalamin-1 deficiency

Summary

transcobalamin I deficiency (MONDO:0008659) is a disease with 1 cohort gene.

At a glance

  • Prevalence: <1 / 1 000 000 (Worldwide) [Orphanet-validated]
  • Cohort genes: 1
  • ClinVar variants: 123

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

TypeClassValueGeographyValidation
Point prevalence<1 / 1 000 000WorldwideValidated

Identifiers

Disease identifiers

FieldValue
Canonical nametranscobalamin I deficiency
Mondo IDMONDO:0008659
MeSHC562798
OMIM193090
Orphanet2967
SNOMED CT237933007
UMLSC0342700
MedGen90993
GARD0004522
Is cancer (heuristic)no

Also known as: Haptocorrin deficiency · TCI deficiency · transcobalamin 1 deficiency · transcobalamin I deficiency · transcobalamin-1 deficiency

Data availability: 123 ClinVar variants · 1 GenCC gene-disease record.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary diseaseinborn errors of metabolism › inborn vitamin metabolic disorder › inborn disorder of cobalamin metabolism and transporttranscobalamin I deficiency

Related subtypes (8): hereditary intrinsic factor deficiency, Imerslund-Grasbeck syndrome, transcobalamin II deficiency, methylmalonic acidemia due to transcobalamin receptor defect, methylmalonic aciduria and homocystinuria, vitamin B12-responsive methylmalonic acidemia, homocystinuria without methylmalonic aciduria, methylmalonic aciduria and/or homocystinuria, cblD type

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

123 retrieved; paginated sample, class counts are floors:

57 likely benign, 47 uncertain significance, 7 pathogenic, 6 benign, 4 conflicting classifications of pathogenicity, 2 benign/likely benign

ClinVarVariant (HGVS)GeneClassificationReview
1459208NM_001062.4(TCN1):c.69C>A (p.Cys23Ter)TCN1Pathogeniccriteria provided, single submitter
2745906NM_001062.4(TCN1):c.82del (p.Glu27_Val28insTer)TCN1Pathogeniccriteria provided, single submitter
2918494NM_001062.4(TCN1):c.524_530del (p.Lys175fs)TCN1Pathogeniccriteria provided, single submitter
3665609NM_001062.4(TCN1):c.217C>T (p.Gln73Ter)TCN1Pathogeniccriteria provided, single submitter
4742884NM_001062.4(TCN1):c.455del (p.Leu152fs)TCN1Pathogeniccriteria provided, single submitter
4762049NM_001062.4(TCN1):c.229C>T (p.Gln77Ter)TCN1Pathogeniccriteria provided, single submitter
568555NM_001062.4(TCN1):c.26del (p.Leu9fs)TCN1Pathogeniccriteria provided, single submitter
2278839NM_001062.4(TCN1):c.173T>C (p.Val58Ala)TCN1Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
779598NM_001062.4(TCN1):c.1000A>G (p.Ile334Val)TCN1Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
780975NM_001062.4(TCN1):c.172del (p.Val58fs)TCN1Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
800251NM_001062.4(TCN1):c.343G>A (p.Asp115Asn)TCN1Conflicting classifications of pathogenicitycriteria provided, conflicting classifications
1005971NM_001062.4(TCN1):c.358G>A (p.Asp120Asn)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1011214NM_001062.4(TCN1):c.1151C>T (p.Pro384Leu)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1018822NM_001062.4(TCN1):c.556+5G>ATCN1Uncertain significancecriteria provided, single submitter
1019642NC_000011.9:g.(?59628980)(59629175_?)dupTCN1Uncertain significancecriteria provided, single submitter
1025075NM_001062.4(TCN1):c.727A>C (p.Ser243Arg)TCN1Uncertain significancecriteria provided, single submitter
1035343NM_001062.4(TCN1):c.140A>G (p.Asn47Ser)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1036789NM_001062.4(TCN1):c.344A>T (p.Asp115Val)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1043075NM_001062.4(TCN1):c.497T>C (p.Val166Ala)TCN1Uncertain significancecriteria provided, single submitter
1057314NM_001062.4(TCN1):c.505C>T (p.His169Tyr)TCN1Uncertain significancecriteria provided, single submitter
1346260NM_001062.4(TCN1):c.623A>G (p.Lys208Arg)TCN1Uncertain significancecriteria provided, single submitter
1385555NM_001062.4(TCN1):c.322G>A (p.Ala108Thr)TCN1Uncertain significancecriteria provided, single submitter
1386775NM_001062.4(TCN1):c.542G>A (p.Ser181Asn)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1387853NM_001062.4(TCN1):c.557A>C (p.Asp186Ala)TCN1Uncertain significancecriteria provided, single submitter
1406523NM_001062.4(TCN1):c.597G>T (p.Lys199Asn)TCN1Uncertain significancecriteria provided, single submitter
1424205NM_001062.4(TCN1):c.112C>A (p.Pro38Thr)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1432371NM_001062.4(TCN1):c.473A>G (p.Asn158Ser)TCN1Uncertain significancecriteria provided, single submitter
1511533NM_001062.4(TCN1):c.730A>G (p.Thr244Ala)TCN1Uncertain significancecriteria provided, multiple submitters, no conflicts
1525440NM_001062.4(TCN1):c.214A>T (p.Met72Leu)TCN1Uncertain significancecriteria provided, single submitter
1715456NM_001062.4(TCN1):c.128T>C (p.Met43Thr)TCN1Uncertain significancecriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 1 · Orphanet: 0 · 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
TCN1ModerateAutosomal recessivetranscobalamin I deficiency

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
TCN1HGNC:11652ENSG00000134827P20061Transcobalamin-1gencc,clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
TCN1Transcobalamin-1Binds vitamin B12 with femtomolar affinity and protects it from the acidic environment of the stomach.

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)
TCN1Other/UnknownnoCbl-bd_prot, Transcobalamin-like_C, Cobalamin_Transport

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
olfactory segment of nasal mucosa1
pancreatic ductal cell1
trachea1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
TCN1187tissue_specificmarkerpancreatic ductal cell, olfactory segment of nasal mucosa, trachea

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
TCN1845

Structural data

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

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
TCN1P200612

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 9. 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
Transport of RCbl within the body11427.5×0.004TCN1
Uptake of dietary cobalamins into enterocytes11142.0×0.004TCN1
Cobalamin (Cbl, vitamin B12) transport and metabolism1634.4×0.005TCN1
Metabolism of water-soluble vitamins and cofactors1181.3×0.012TCN1
Metabolism of vitamins and cofactors1116.5×0.015TCN1
Innate Immune System125.5×0.056TCN1
Neutrophil degranulation123.1×0.056TCN1
Immune System113.0×0.086TCN1
Metabolism111.6×0.086TCN1

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
cobalt ion transport12407.4×5e-04TCN1
cobalamin transport11872.4×5e-04TCN1

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
TCN100

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 drug1TCN1

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
TCN10

Clinical trials & evidence

Clinical trials

Clinical trials: 0.