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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.
PPOX
protoporphyrinogen oxidase
Chromosome 1 Β· 1q23.3
NCBI Gene: 5498Ensembl: ENSG00000143224.19HGNC: HGNC:9280UniProt: B4DY76
76PubMed Papers
22Diseases
0Drugs
65Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
oxygen-dependent protoporphyrinogen oxidase activityporphyrin-containing compound biosynthetic processheme biosynthetic processheme B biosynthetic processvariegate porphyriavariegate porphyria, childhood-onsetPorphyria variegataneurodegenerative disease
✦AI Summary

PPOX (protoporphyrinogen oxidase) catalyzes the 6-electron oxidation of protoporphyrinogen-IX to protoporphyrin-IX, a critical step in heme biosynthesis 1. The gene is located on chromosome 1 as a single-copy gene containing 13 exons 1. PPOX functions as a mitochondrial inner membrane protein involved in oxygen-dependent porphyrin metabolism [GO annotations]. Loss-of-function mutations in PPOX cause variegate porphyria (VP), an autosomal dominant disorder characterized by decreased enzyme activity 2. VP presents with two distinct phenotypes: photosensitive skin disease with blistering on sun-exposed areas 3, and acute neurovisceral crises involving severe abdominal pain, neurological symptoms, and electrolyte abnormalities like hyponatremia 4. Acute VP attacks are clinically less severe than those of acute intermittent porphyria but require emergency management with intravenous hemin, supportive care, and avoidance of porphyrinogenic triggers 5. Long-term complications include chr1 kidney disease, hepatocellular carcinoma, and hypertension 5. Genetic testing of PPOX confirms diagnosis in biochemically positive patients 6, with over 30 characterized mutations identified globally, many family-specific 7.

Sources cited
1
PPOX gene structure, chromosome location (1q22), single-copy status, 13 exons, and transcriptional organization
PMID: 8575762
2
VP is autosomal dominant with decreased PPOX activity, causing photosensitive skin disease and acute neurovisceral crises
PMID: 9516676
3
VP mutations in PPOX cause skin blistering, hyperpigmentation, neurological symptoms including seizures, and brachydactyly
PMID: 35164799
4
VP presents with acute abdominal pain and hyponatremia/SIAD; genetic analysis confirms novel PPOX mutations
PMID: 29516370
5
Acute hepatic porphyrias including VP require hemin therapy; VP causes long-term complications including kidney disease and hepatocellular carcinoma
PMID: 36642627
6
WES enables diagnostic confirmation of PPOX pathogenic variants in porphyria cases
PMID: 27848944
7
PPOX gene shows high mutation frequency, particularly in the first untranslated exon, with multiple family-specific mutations
PMID: 9778454
Disease Associationsβ“˜22
variegate porphyriaOpen Targets
0.85Strong
variegate porphyria, childhood-onsetOpen Targets
0.75Strong
Porphyria variegataOpen Targets
0.71Strong
neurodegenerative diseaseOpen Targets
0.52Moderate
Abnormal blistering of the skinOpen Targets
0.42Moderate
Sensory neuropathyOpen Targets
0.37Weak
sensory peripheral neuropathyOpen Targets
0.37Weak
Abnormal urinary colorOpen Targets
0.34Weak
ConstipationOpen Targets
0.34Weak
migraine disorderOpen Targets
0.34Weak
Abdominal colicOpen Targets
0.33Weak
PorphyrinuriaOpen Targets
0.33Weak
Abnormality of metabolism/homeostasisOpen Targets
0.27Weak
cardiomyopathyOpen Targets
0.27Weak
genetic disorderOpen Targets
0.19Weak
mathematical abilityOpen Targets
0.12Weak
infectionOpen Targets
0.05Suggestive
essential fructosuriaOpen Targets
0.05Suggestive
gastric cancerOpen Targets
0.05Suggestive
glioblastoma multiformeOpen Targets
0.04Suggestive
Variegate porphyriaUniProt
Variegate porphyria, childhood-onsetUniProt
Pathogenic Variants65
NM_001122764.3(PPOX):c.565C>T (p.Gln189Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 189
NM_001122764.3(PPOX):c.916_917del (p.Leu306fs)Pathogenic
not provided|Cardiomyopathy
β˜…β˜…β˜†β˜†2025β†’ Residue 306
NM_001122764.3(PPOX):c.1303C>T (p.Gln435Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 435
NM_001122764.3(PPOX):c.1147_1148del (p.Val383fs)Pathogenic
not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 383
NM_001122764.3(PPOX):c.175C>T (p.Arg59Trp)Pathogenic
Variegate porphyria|not provided|Variegate porphyria, childhood-onset
β˜…β˜…β˜†β˜†2025β†’ Residue 59
NM_001122764.3(PPOX):c.571G>T (p.Glu191Ter)Likely pathogenic
See cases|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 191
NM_001122764.3(PPOX):c.745dup (p.Val249fs)Pathogenic
not provided|Variegate porphyria
β˜…β˜…β˜†β˜†2024β†’ Residue 249
NM_001122764.3(PPOX):c.1330_1331del (p.Leu444fs)Likely pathogenic
not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 444
NM_001122764.3(PPOX):c.1082dup (p.Gly362fs)Pathogenic
not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 362
NM_001122764.3(PPOX):c.397G>T (p.Glu133Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 133
NM_001122764.3(PPOX):c.338+2dupLikely pathogenic
not provided
β˜…β˜…β˜†β˜†2023
NM_001122764.3(PPOX):c.1287del (p.Lys429fs)Pathogenic
not provided
β˜…β˜…β˜†β˜†2022β†’ Residue 429
NM_001122764.3(PPOX):c.503G>A (p.Arg168His)Pathogenic
Variegate porphyria|not provided|See cases
β˜…β˜…β˜†β˜†2022β†’ Residue 168
NM_001122764.3(PPOX):c.199del (p.Ala66_Leu67insTer)Pathogenic
Variegate porphyria|not provided
β˜…β˜…β˜†β˜†2022β†’ Residue 66
NM_001122764.3(PPOX):c.1292-1G>CLikely pathogenic
Malignant tumor of urinary bladder|Inborn genetic diseases
β˜…β˜†β˜†β˜†2025
NM_001122764.3(PPOX):c.1291+1G>CPathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001122764.3(PPOX):c.917T>C (p.Leu306Pro)Likely pathogenic
Variegate porphyria
β˜…β˜†β˜†β˜†2025β†’ Residue 306
NM_001122764.3(PPOX):c.383G>A (p.Trp128Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 128
NM_001122764.3(PPOX):c.1098+2T>ALikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001122764.3(PPOX):c.884T>C (p.Leu295Pro)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 295
View on ClinVar β†—
Related Genes
ENDOVProtein interaction90%HMBSProtein interaction89%URODProtein interaction89%GALEProtein interaction85%SLC6A1Protein interaction83%CPOXProtein interaction81%
Tissue Expression6 tissues
Bone Marrow
100%
Ovary
40%
Lung
22%
Liver
20%
Heart
18%
Brain
12%
Gene Interaction Network
Click a node to explore
PPOXENDOVHMBSURODGALESLC6A1CPOX
PROTEIN STRUCTURE
Preparing viewer…
PDB3NKS Β· 1.90 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.58Moderately Constrained
pLIβ“˜
0.24Tolerant
Observed/Expected LoF0.41 [0.30–0.58]
RankingsWhere PPOX stands among ~20K protein-coding genes
  • #6,278of 20,598
    Most Researched76
  • #1,108of 5,498
    Most Pathogenic Variants65 Β· top quartile
  • #3,888of 17,882
    Most Constrained (LOEUF)0.58 Β· top quartile
Genes detectedPPOX
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
AGA Clinical Practice Update on Diagnosis and Management of Acute Hepatic Porphyrias: Expert Review.
PMID: 36642627
Gastroenterology Β· 2023
1.00
2
Clinical exome sequencing: results from 2819 samples reflecting 1000 families.
PMID: 27848944
Eur J Hum Genet Β· 2017
0.90
3
A boy with blistering of sun-exposed skin and finger shortening: the first case of Variegate Porphyria with a novel mutation in protoporphyrinogen oxidase (PPOX) gene in Iran: a case report and literature review.
PMID: 35164799
Ital J Pediatr Β· 2022
0.80
4
The human protoporphyrinogen oxidase gene (PPOX): organization and location to chromosome 1.
PMID: 8575762
Genomics Β· 1995
0.70
5
Novel mutation of PPOX gene in a patient with abdominal pain and syndrome of inappropriate antidiuresis.
PMID: 29516370
Endocrine Β· 2018
0.60