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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.
NAXD
NAD(P)HX dehydratase
Chromosome 13 Β· 13q34
NCBI Gene: 55739Ensembl: ENSG00000213995.12HGNC: HGNC:25576UniProt: A0A7P0T9D8
27PubMed Papers
21Diseases
0Drugs
17Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingmitochondrionATP-dependent NAD(P)H-hydrate dehydratase activitymetabolite repairencephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathyNAD(P)HX dehydratase deficiencymitochondrial diseasetympanic membrane disease
✦AI Summary

NAXD (NAD(P)HX dehydratase) is an ATP-dependent enzyme that catalyzes the dehydration of the S-form of NAD(P)HX, a redox-inactive damaged derivative of the central metabolic cofactors NAD(P)H 1. Working in concert with NAD(P)HX epimerase (NAXE), NAXD repairs both epimers of NAD(P)HX that accumulate from enzymatic or heat-dependent hydration of NAD(P)H 1. NAXD exists as both mitochondrial and cytosolic isoforms generated from alternative start codons; the mitochondrial form is initiated from exon 1 containing a mitochondrial propeptide, while the cytosolic isoform starts in exon 2 1. NAXD deficiency causes progressive, early-onset encephalopathy with brain edema and/or leukoencephalopathy 2 (PEBEL2), an autosomal recessive metabolite repair disorder 2. Disease manifestations depend on affected isoforms: variants affecting both cytosolic and mitochondrial forms present with neurological defects, seizures, and skin lesions triggered by febrile illness 1. Mitochondrial-only variants cause myopathy and cardiac manifestations without neurological degeneration or skin involvement 1. At the molecular level, NAXD deficiency leads to accumulation of damaged NAD(P)HX species, mitochondrial dysfunction, impaired serine synthesis, and NAD+ depletion during metabolic stress 3 4. Niacin and nicotinamide riboside supplementation show therapeutic promise by restoring NAD(P)H metabolism 4.

Sources cited
1
NAXD catalyzes dehydration of S-form NAD(P)HX; exists as mitochondrial and cytosolic isoforms; variants affecting both isoforms cause neurological defects/seizures/skin lesions while mitochondrial-only variants cause myopathy/cardiac manifestations
PMID: 35866541
2
NAXD deficiency causes PEBEL2, an autosomal recessive metabolite repair disorder with progressive neurological deterioration triggered by febrile illness
PMID: 34161859
3
NAXD deficiency leads to accumulation of damaged NADHX species, mitochondrial dysfunction, and impaired metabolism under stress; fever/heat damages NAD(P)H generating NAD(P)HX
PMID: 30576410
4
NAXD deficiency impairs de novo serine synthesis through inhibition of 3-phosphoglycerate dehydrogenase; NAD+ depletion occurs under metabolic stress; nicotinamide riboside and inosine show therapeutic benefit
PMID: 39789421
⚠Limited data available β€” This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜21
encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathyOpen Targets
0.75Strong
NAD(P)HX dehydratase deficiencyOpen Targets
0.74Strong
mitochondrial diseaseOpen Targets
0.37Weak
tympanic membrane diseaseOpen Targets
0.32Weak
tympanic membrane perforationOpen Targets
0.32Weak
protozoa infectious diseaseOpen Targets
0.30Weak
cerebral atherosclerosisOpen Targets
0.30Weak
peritonitisOpen Targets
0.29Weak
central nervous system cancerOpen Targets
0.28Weak
genetic disorderOpen Targets
0.19Weak
autismOpen Targets
0.04Suggestive
Autosomal dominant striatal neurodegenerationOpen Targets
0.04Suggestive
autism spectrum disorderOpen Targets
0.04Suggestive
nephritisOpen Targets
0.04Suggestive
ovarian neoplasmOpen Targets
0.04Suggestive
intellectual disability, autosomal dominant 50Open Targets
0.04Suggestive
duodenitisOpen Targets
0.03Suggestive
intellectual disability, autosomal recessive 58Open Targets
0.03Suggestive
bone remodeling diseaseOpen Targets
0.02Suggestive
glioblastoma multiformeOpen Targets
0.01Suggestive
Encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy, 2UniProt
Pathogenic Variants17
NM_001242882.2(NAXD):c.54_57del (p.Ala20fs)Pathogenic
NAD(P)HX dehydratase deficiency|not provided|NAXD-related disorder
β˜…β˜…β˜†β˜†2023β†’ Residue 20
NM_001242882.2(NAXD):c.848del (p.Pro283fs)Likely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2025β†’ Residue 283
NM_001242882.2(NAXD):c.704C>T (p.Ser235Phe)Likely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2025β†’ Residue 235
NM_001242882.2(NAXD):c.442-2A>GLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2023
NM_001242882.2(NAXD):c.439C>T (p.Gln147Ter)Pathogenic
not provided|Squamous cell lung carcinoma
β˜…β˜†β˜†β˜†2023β†’ Residue 147
NM_001242882.2(NAXD):c.514C>T (p.Gln172Ter)Likely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2023β†’ Residue 172
NM_001242882.2(NAXD):c.442-1G>ALikely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2023
NM_001242882.2(NAXD):c.238A>T (p.Lys80Ter)Likely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2022β†’ Residue 80
NM_001242882.2(NAXD):c.441+3A>GLikely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2022
NM_001242882.2(NAXD):c.715C>T (p.Gln239Ter)Likely pathogenic
NAD(P)HX dehydratase deficiency
β˜…β˜†β˜†β˜†2021β†’ Residue 239
NC_000013.11:g.110615008_110622534delPathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2022
NM_001242882.2(NAXD):c.318C>G (p.Ile106Met)Pathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2022β†’ Residue 106
NM_001242882.2(NAXD):c.102_103del (p.Thr35fs)Pathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2022β†’ Residue 35
NM_001242882.2(NAXD):c.44del (p.Arg15fs)Pathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2020β†’ Residue 15
NM_001242882.2(NAXD):c.187G>A (p.Gly63Ser)Pathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2020β†’ Residue 63
NM_001242882.2(NAXD):c.839+1G>TPathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2020
NM_001242882.2(NAXD):c.948_949insTT (p.Ala317fs)Pathogenic
NAD(P)HX dehydratase deficiency
β˜†β˜†β˜†β˜†2020β†’ Residue 317
View on ClinVar β†—
Related Genes
SLC25A51Shared pathway100%YJEFN3Protein interaction87%EDC3Protein interaction78%NAXEProtein interaction74%NMRK1Shared pathway50%NMRK2Shared pathway33%
Tissue Expression6 tissues
Liver
100%
Ovary
84%
Heart
41%
Brain
40%
Lung
33%
Bone Marrow
17%
Gene Interaction Network
Click a node to explore
NAXDSLC25A51YJEFN3EDC3NAXENMRK1NMRK2
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q8IW45
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.84LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.56 [0.38–0.84]
RankingsWhere NAXD stands among ~20K protein-coding genes
  • #12,614of 20,598
    Most Researched27
  • #2,366of 5,498
    Most Pathogenic Variants17
  • #7,277of 17,882
    Most Constrained (LOEUF)0.84
Genes detectedNAXD
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Clinical and biochemical distinctions for a metabolite repair disorder caused by NAXD or NAXE deficiency.
PMID: 35866541
J Inherit Metab Dis Β· 2022
1.00
2
Cutaneous Manifestations of NAXD or NAXE Deficiency: A Literature Review for the Dermatologist.
PMID: 39887790
Pediatr Dermatol Β· 2025
0.90
3
Niacin therapy improves outcome and normalizes metabolic abnormalities in an NAXD-deficient patient.
PMID: 35231119
Brain Β· 2022
0.80
4
NAD(P)HX dehydratase (NAXD) deficiency: a novel neurodegenerative disorder exacerbated by febrile illnesses.
PMID: 30576410
Brain Β· 2019
0.70
5
NAD(P)HX dehydratase (NAXD) deficiency due to a novel biallelic missense variant and review of literature.
PMID: 34161859
Eur J Med Genet Β· 2021
0.60