Tumoral calcinosis, hyperphosphatemic, familial, 3

disease
On this page

Also known as HFTC3

Summary

Tumoral calcinosis, hyperphosphatemic, familial, 3 (MONDO:0060715) is a disease with 1 cohort gene.

At a glance

  • Cohort genes: 1
  • ClinVar variants: 146

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nametumoral calcinosis, hyperphosphatemic, familial, 3
Mondo IDMONDO:0060715
EFOEFO:0009384
OMIM617994
UMLSC4693864
MedGen1638917
GARD0016282
Is cancer (heuristic)no

Also known as: HFTC3 · tumoral calcinosis, hyperphosphatemic, familial, 3

Data availability: 146 ClinVar variants · 3 GenCC gene-disease records.

Disease family

Classification path: disease › human disease › disease by body system or component › integumentary system disorder › familial tumoral calcinosistumoral calcinosis, hyperphosphatemic, familial, 3

Related subtypes (4): normophosphatemic familial tumoral calcinosis, tumoral calcinosis, hyperphosphatemic, familial, 2, familial hyperphosphatemic tumoral calcinosis/hyperphosphatemic hyperostosis syndrome, tumoral calcinosis, hyperphosphatemic, familial, 1

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

146 retrieved; paginated sample, class counts are floors:

81 uncertain significance, 19 benign, 19 conflicting classifications of pathogenicity, 15 likely benign, 12 benign/likely benign

ClinVarVariant (HGVS)GeneClassificationReview
311684NM_004795.4(KL):c.511C>A (p.Arg171Ser)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
311687NM_004795.4(KL):c.911A>G (p.Asn304Ser)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
311702NM_004795.4(KL):c.2259G>A (p.Leu753=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
311706NM_004795.4(KL):c.2626G>A (p.Asp876Asn)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
5346NM_004795.4(KL):c.578A>G (p.His193Arg)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
64548NM_004795.4(KL):c.2862G>A (p.Pro954=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
881364NM_004795.4(KL):c.15C>T (p.Ala5=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
881482NM_004795.4(KL):c.2298C>G (p.Gly766=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
881861NM_004795.4(KL):c.1188A>G (p.Gln396=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
882962NM_004795.4(KL):c.450C>A (p.Ser150=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883027NM_004795.4(KL):c.1419G>A (p.Arg473=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883029NM_004795.4(KL):c.1626C>T (p.Thr542=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883105NM_004795.4(KL):c.2874T>G (p.Thr958=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883106NM_004795.4(KL):c.2904C>T (p.Thr968=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883759NM_004795.4(KL):c.614G>A (p.Gly205Asp)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883761NM_004795.4(KL):c.747C>T (p.Ala249=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883811NM_004795.4(KL):c.1860C>A (p.Arg620=)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883812NM_004795.4(KL):c.1873G>A (p.Glu625Lys)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
883813NM_004795.4(KL):c.1945C>T (p.Arg649Cys)KLConflicting classifications of pathogenicitycriteria provided, conflicting classifications
1344758NM_004795.4(KL):c.2251A>G (p.Arg751Gly)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1345701NM_004795.4(KL):c.254A>G (p.Lys85Arg)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1361567NM_004795.4(KL):c.1462TTG[1] (p.Leu489del)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1367439NM_004795.4(KL):c.398T>A (p.Phe133Tyr)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1403649NM_004795.4(KL):c.1882C>T (p.Arg628Cys)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1409686NM_004795.4(KL):c.1764G>T (p.Met588Ile)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1417990NM_004795.4(KL):c.944A>G (p.Glu315Gly)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1426214NM_004795.4(KL):c.2069C>T (p.Pro690Leu)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1439474NM_004795.4(KL):c.257G>A (p.Gly86Asp)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1485727NM_004795.4(KL):c.4C>T (p.Pro2Ser)KLUncertain significancecriteria provided, multiple submitters, no conflicts
1493920NM_004795.4(KL):c.2513G>C (p.Trp838Ser)KLUncertain significancecriteria provided, multiple submitters, no conflicts

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
KLSupportiveAutosomal recessivetumoral calcinosis, hyperphosphatemic, familial, 14

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
KLOrphanet:306661Familial hyperphosphatemic tumoral calcinosis/Hyperphosphatemic hyperostosis syndrome

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
KLHGNC:6344ENSG00000133116Q9UEF7Klothogencc,clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
KLKlothoMay have weak glycosidase activity towards glucuronylated steroids.

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)
KLOther/UnknownnoGlyco_hydro_1, GH_hydrolase_sf, GH_1_N_CS

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
choroid plexus epithelium1
kidney epithelium1
nephron tubule1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
KL188tissue_specificmarkerchoroid plexus epithelium, nephron tubule, kidney epithelium

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
KL1,494

Structural data

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

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
KLQ9UEF76

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 12. 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
FGFR1c and Klotho ligand binding and activation12855.0×0.004KL
Phospholipase C-mediated cascade: FGFR11671.8×0.004KL
Downstream signaling of activated FGFR11543.8×0.004KL
PI-3K cascade:FGFR11519.1×0.004KL
SHC-mediated cascade:FGFR11496.5×0.004KL
FRS-mediated FGFR1 signaling1456.8×0.004KL
Negative regulation of FGFR1 signaling1368.4×0.005KL
PI3K Cascade1271.9×0.006KL
Constitutive Signaling by Aberrant PI3K in Cancer1126.9×0.011KL
PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling196.8×0.012KL
PIP3 activates AKT signaling166.8×0.016KL
RAF/MAP kinase cascade161.1×0.016KL

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
positive regulation of MAPKKK cascade by fibroblast growth factor receptor signaling pathway12808.7×0.002KL
norepinephrine biosynthetic process12106.5×0.002KL
response to angiotensin11872.4×0.002KL
negative regulation of systemic arterial blood pressure11053.2×0.002KL
energy reserve metabolic process11053.2×0.002KL
response to vitamin D1802.5×0.002KL
calcium ion homeostasis1443.5×0.003KL
determination of adult lifespan1432.1×0.003KL
positive regulation of bone mineralization1391.9×0.003KL
response to activity1324.1×0.004KL
fibroblast growth factor receptor signaling pathway1285.6×0.004KL
carbohydrate metabolic process1135.9×0.007KL

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
KL00

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

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

SymbolAssaysType breakdown
KL1ADMET:1

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 drug1KL

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
KL1

Clinical trials & evidence

Clinical trials

Clinical trials: 0.

  • Cohort genes: KL