TPK1

Chr 7AR

thiamin pyrophosphokinase 1

Also known as: HTPK1, PP20, THMD5

The protein catalyzes the phosphorylation of thiamine to thiamine pyrophosphate (TPP), a critical cofactor for mitochondrial pyruvate dehydrogenase and enzymes involved in glycolysis and energy production. Mutations cause thiamine metabolism dysfunction syndrome 5, characterized by episodic encephalopathy, and follow autosomal recessive inheritance. The gene shows moderate constraint against loss-of-function variants (LOEUF 0.7), reflecting its essential role in cellular energy metabolism.

OMIMResearchSummary from RefSeq, OMIM, UniProt
DNmechanismARLOEUF 0.701 OMIM phenotype
Clinical SummaryTPK1
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Gene-Disease Validity (ClinGen)
Leigh syndrome · ARModerate

Moderate evidence — consider for supplementary testing

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.31) despite low pLI — interpret in context.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.70LOEUF
pLI 0.091
Z-score 2.33
OE 0.31 (0.150.70)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.34Z-score
OE missense 0.92 (0.791.07)
121 obs / 132.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.31 (0.150.70)
00.351.4
Missense OE0.92 (0.791.07)
00.61.4
Synonymous OE0.94
01.21.6
LoF obs/exp: 4 / 13.1Missense obs/exp: 121 / 132.1Syn Z: 0.34
DN
0.6746th %ile
GOF
0.4677th %ile
LOF
0.2287th %ile

The highest-scoring mechanism for this gene is dominant-negative.

DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

TPK1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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