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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
PDXK
pyridoxal kinase
Chromosome 21 Β· 21q22.3
NCBI Gene: 8566Ensembl: ENSG00000160209.19HGNC: HGNC:8819UniProt: F2Z2Y4
70PubMed Papers
21Diseases
0Drugs
4Pathogenic Variants
FUNCTIONAL ROLE
Kinase
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
magnesium ion bindingATP bindingzinc ion bindingpyridoxal kinase activityneuropathy, hereditary motor and sensory, type VIc, with optic atrophyneurodegenerative diseaseneuroinflammatory disorderovarian neoplasm
✦AI Summary

PDXK (pyridoxal kinase) is the rate-limiting enzyme for vitamin B6 metabolism, catalyzing the phosphorylation of dietary B6 vitamers (pyridoxal, pyridoxine, and pyridoxamine) to their active forms, primarily pyridoxal 5'-phosphate (PLP) 1. PLP serves as an essential cofactor for over 140 enzymatic reactions across diverse metabolic pathways 2. PDXK expression is regulated through a TATA-box-free promoter containing critical Sp1 binding sites 2. Biallelic PDXK mutations cause autosomal recessive hereditary motor and sensory neuropathy with optic atrophy, characterized by axonal polyneuropathy leading to progressive wheelchair dependence and blindness if untreated 13. Disease pathogenesis involves reduced PDXK enzymatic activity and consequently low circulating PLP levels 1. Notably, this is one of the few inherited neuropathies amenable to targeted treatment: pyridoxal 5'-phosphate supplementation rescues the biochemical profile and produces clinical improvement, including restoration of ambulation 1. Excessive pyridoxine supplementation induces peripheral neuropathy through PDXK inhibition and disrupted GABA neurotransmission in sensory neurons 4. Beyond neurological function, PDXK is selectively required for acute myeloid leukemia cell proliferation, representing a pharmacologically actionable metabolic dependency 5. PDXK also regulates thermoregulation through hypothalamic vitamin B6 metabolism 6.

Sources cited
1
PDXK mutations cause biallelic axonal polyneuropathy with optic atrophy; reduced PDXK activity decreases PLP levels; PLP supplementation provides clinical rescue
PMID: 31187503
2
PDXK promoter lacks TATA box but contains Sp1-binding GC-boxes critical for gene regulation; PLP is cofactor in >140 enzyme reactions
PMID: 28716709
3
High pyridoxine intake inhibits PDXK, causing preferential sensory neuron injury and peripheral neuropathy through disrupted GABA neurotransmission
PMID: 33912895
4
PDXK is a selective metabolic dependency in acute myeloid leukemia required for PLP production and cell proliferation
PMID: 31935373
5
PDXK-related neuropathy exhibits variable presentation; novel splice-altering variants reveal alternative disease mechanisms
PMID: 41888341
6
PDXK in hypothalamic preoptic area regulates thermoregulation and body temperature through vitamin B6 metabolism
PMID: 37752106
Disease Associationsβ“˜21
neuropathy, hereditary motor and sensory, type VIc, with optic atrophyOpen Targets
0.58Moderate
neurodegenerative diseaseOpen Targets
0.43Moderate
neuroinflammatory disorderOpen Targets
0.37Weak
ovarian neoplasmOpen Targets
0.32Weak
hypertensionOpen Targets
0.15Weak
essential hypertensionOpen Targets
0.14Weak
hepatocellular carcinomaOpen Targets
0.08Suggestive
Miyoshi myopathyOpen Targets
0.05Suggestive
cardiovascular diseaseOpen Targets
0.05Suggestive
coronary artery diseaseOpen Targets
0.05Suggestive
Abnormality of refractionOpen Targets
0.04Suggestive
colorectal carcinomaOpen Targets
0.04Suggestive
neoplasmOpen Targets
0.04Suggestive
Parkinson diseaseOpen Targets
0.04Suggestive
experimental autoimmune encephalomyelitisOpen Targets
0.03Suggestive
Increased blood pressureOpen Targets
0.03Suggestive
hair colorOpen Targets
0.02Suggestive
response to xenobiotic stimulusOpen Targets
0.02Suggestive
tooth diseaseOpen Targets
0.02Suggestive
multinodular goiterOpen Targets
0.02Suggestive
Neuropathy, hereditary motor and sensory, 6C, with optic atrophyUniProt
Pathogenic Variants4
NM_003681.5(PDXK):c.682G>A (p.Ala228Thr)Pathogenic
Neuropathy, hereditary motor and sensory, type VIc, with optic atrophy|PDXK-related disorder
β˜…β˜…β˜†β˜†2023β†’ Residue 228
NM_003681.5(PDXK):c.465-15_489delinsGCAPathogenic
not provided
β˜…β˜†β˜†β˜†2022
NM_003681.5(PDXK):c.225T>A (p.Asn75Lys)Likely pathogenic
Neuropathy, hereditary motor and sensory, type VIc, with optic atrophy
β˜…β˜†β˜†β˜†2020β†’ Residue 75
NM_003681.5(PDXK):c.659G>A (p.Arg220Gln)Pathogenic
Neuropathy, hereditary motor and sensory, type VIc, with optic atrophy
β˜†β˜†β˜†β˜†2019β†’ Residue 220
View on ClinVar β†—
Related Genes
PDXPProtein interaction97%PNPOProtein interaction96%AOX1Protein interaction92%PHOSPHO2Protein interaction90%PLPBPProtein interaction86%FDX2Protein interaction86%
Tissue Expression6 tissues
Bone Marrow
100%
Brain
73%
Lung
60%
Liver
54%
Ovary
47%
Heart
41%
Gene Interaction Network
Click a node to explore
PDXKPDXPPNPOAOX1PHOSPHO2PLPBPFDX2
PROTEIN STRUCTURE
Preparing viewer…
PDB8WR2 Β· 1.94 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.60Moderately Constrained
pLIβ“˜
0.77Intermediate
Observed/Expected LoF0.35 [0.22–0.60]
RankingsWhere PDXK stands among ~20K protein-coding genes
  • #6,744of 20,598
    Most Researched70
  • #3,776of 5,498
    Most Pathogenic Variants4
  • #4,081of 17,882
    Most Constrained (LOEUF)0.60 Β· top quartile
Genes detectedPDXK
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Vitamin B-6-Induced Neuropathy: Exploring the Mechanisms of Pyridoxine Toxicity.
PMID: 33912895
Adv Nutr Β· 2021
1.00
2
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis Β· 2022
0.90
3
Isolation and characterization of the 5'-flanking region of the human PDXK gene.
PMID: 28716709
Gene Β· 2017
0.80
4
Natural product P57 induces hypothermia through targeting pyridoxal kinase.
PMID: 37752106
Nat Commun Β· 2023
0.70
5
Identification of a prognostic cuproptosis-related signature in hepatocellular carcinoma.
PMID: 36750831
Biol Direct Β· 2023
0.60