Trichothiodystrophy 4, nonphotosensitive

disease
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Also known as MPLKIP nonphotosensitive trichothiodystrophynonphotosensitive trichothiodystrophy caused by mutation in MPLKIPPollitt syndromeTTD4

Summary

Trichothiodystrophy 4, nonphotosensitive (MONDO:0021013) is a disease caused by MPLKIP (GenCC Definitive), with 1 cohort gene.

At a glance

  • Causal gene: MPLKIP (GenCC Definitive)
  • Cohort genes: 1
  • ClinVar variants: 16

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nametrichothiodystrophy 4, nonphotosensitive
Mondo IDMONDO:0021013
OMIM234050
Orphanet75790
DOIDDOID:0050528
NCITC146899
SNOMED CT403796005
UMLSC1313961
MedGen272036
GARD0005271
Is cancer (heuristic)no

Also known as: MPLKIP nonphotosensitive trichothiodystrophy · nonphotosensitive trichothiodystrophy caused by mutation in MPLKIP · Pollitt syndrome · trichothiodystrophy 4, nonphotosensitive · TTD4

Data availability: 16 ClinVar variants · 4 GenCC gene-disease records · 4 cell lines.

Disease family

Classification path: disease › human disease › disease by body system or component › syndromic diseaseectodermal dysplasia syndrometrichothiodystrophytrichothiodystrophy 4, nonphotosensitive

Related subtypes (6): photosensitive trichothiodystrophy, trichothiodystrophy 5, nonphotosensitive, trichothiodystrophy 6, nonphotosensitive, trichothiodystrophy 8, nonphotosensitive, trichothiodystrophy 9, nonphotosensitive, trichothiodystrophy 7, nonphotosensitive

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

16 retrieved; paginated sample, class counts are floors:

8 pathogenic, 3 uncertain significance, 2 pathogenic/likely pathogenic, 2 likely pathogenic, 1 conflicting classifications of pathogenicity

ClinVarVariant (HGVS)GeneClassificationReview
1848NM_138701.4(MPLKIP):c.148_152del (p.His50fs)LOC129998295Pathogenicno assertion criteria provided
1332725NM_138701.4(MPLKIP):c.229del (p.Arg77fs)MPLKIPPathogeniccriteria provided, multiple submitters, no conflicts
1844NM_138701.4(MPLKIP):c.430A>G (p.Met144Val)MPLKIPPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
1845NM_138701.4(MPLKIP):c.137_138del (p.Gly46fs)MPLKIPPathogenicno assertion criteria provided
1846NC_000007.14:g.(40116368_?)_(?_40134601)delMPLKIPPathogenicno assertion criteria provided
1847NM_138701.4(MPLKIP):c.277del (p.Ser93fs)MPLKIPPathogeniccriteria provided, multiple submitters, no conflicts
224867NM_138701.4(MPLKIP):c.339+1G>AMPLKIPPathogeniccriteria provided, single submitter
3255600NM_138701.4(MPLKIP):c.135C>G (p.Tyr45Ter)MPLKIPPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
4528345NM_138701.4(MPLKIP):c.339G>A (p.Gln113=)MPLKIPPathogeniccriteria provided, single submitter
4528346NM_138701.4(MPLKIP):c.4C>T (p.Gln2Ter)MPLKIPPathogeniccriteria provided, single submitter
3382053NM_138701.4(MPLKIP):c.198_199del (p.Pro67fs)LOC129998295Likely pathogeniccriteria provided, single submitter
3242125NM_138701.4(MPLKIP):c.397delinsGGA (p.Ser133fs)MPLKIPLikely pathogeniccriteria provided, single submitter
1929397NM_138701.4(MPLKIP):c.372del (p.Arg126fs)MPLKIPConflicting classifications of pathogenicitycriteria provided, conflicting classifications
1031149NM_138701.4(MPLKIP):c.49G>A (p.Gly17Ser)MPLKIPUncertain significancecriteria provided, single submitter
1953936NM_138701.4(MPLKIP):c.377G>A (p.Arg126His)MPLKIPUncertain significancecriteria provided, multiple submitters, no conflicts
548559NM_138701.4(MPLKIP):c.494G>T (p.Ser165Ile)MPLKIPUncertain significancecriteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 5 · 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
MPLKIPDefinitiveAutosomal recessivetrichothiodystrophy 4, nonphotosensitive5

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
MPLKIPOrphanet:33364Trichothiodystrophy

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
MPLKIPHGNC:16002ENSG00000168303Q8TAP9M-phase-specific PLK1-interacting proteingencc,clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
MPLKIPM-phase-specific PLK1-interacting proteinMay play a role in maintenance of cell cycle integrity by regulating mitosis or cytokinesis.

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)
MPLKIPOther/UnknownnoMPLKIP-like_vertebrate, TTDN1/SICKLE

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
kidney epithelium1
superior surface of tongue1
upper arm skin1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
MPLKIP255ubiquitousmarkerkidney epithelium, upper arm skin, superior surface of tongue

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
MPLKIP652

Structural data

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

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
MPLKIPQ8TAP957.39

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
cell division146.2×0.022MPLKIP

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
MPLKIP00

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 drug1MPLKIP

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
MPLKIP0

Clinical trials & evidence

Clinical trials

Clinical trials: 0.