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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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UROS
uroporphyrinogen III synthase
Chromosome 10 Β· 10q26.2
NCBI Gene: 7390Ensembl: ENSG00000188690.16HGNC: HGNC:12592UniProt: A0A0S2Z4T8
50PubMed Papers
21Diseases
0Drugs
30Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
uroporphyrinogen III biosynthetic processprotoporphyrinogen IX biosynthetic processheme biosynthetic processuroporphyrinogen-III synthase activityCongenital erythropoietic porphyriacutaneous porphyrianeurodegenerative diseasegenetic disorder
✦AI Summary

UROS (uroporphyrinogen III synthase) catalyzes the cyclization of hydroxymethylbilane to uroporphyrinogen III, a critical branch point in heme biosynthesis 1. As the fourth enzyme in the heme biosynthetic pathway, UROS enables production of porphyrins that serve as essential cofactors for diverse cellular processes, including oxygen transport (heme) and methionine synthesis (vitamin B12) 1. UROS is localized to chromosome 10.2-q26.3 2. Congenital erythropoietic porphyria (CEP) results from biallelic UROS mutations causing deficient enzymatic activity 3. This autosomal recessive disorder leads to pathologic accumulation of Type I porphyrins, particularly the photoreactive uroporphyrinogen I and coproporphyrinogen I, which damage erythrocytes 4. Clinical manifestations range from severe (non-immune hydrops fetalis in utero) to mild (adult-onset dermatologic disease) depending on residual UROS activity 3. Patients exhibit exquisite photosensitivity causing bullous lesions, erythrodontia, corneal scarring, and hemolytic anemia 4. Management includes strict light avoidance and, in severe transfusion-dependent cases, hematopoietic stem cell transplantation offers curative potential 4. Emerging therapeutic approaches include chaperone-mediated UROS rescue, gene therapy, and proteasome inhibition 3.

Sources cited
1
UROS catalyzes cyclization of hydroxymethylbilane to uroporphyrinogen III and porphyrins serve as cofactors for processes including oxygen transport and vitamin B12 synthesis
PMID: 11689424
2
UROS gene is localized to chromosome 10q25.2-q26.3
PMID: 2037278
3
CEP is autosomal recessive disorder caused by biallelic UROS variants resulting in decreased enzymatic activity and Type I porphyrin accumulation; severity ranges from hydrops fetalis to mild disease; chaperone therapy under investigation
PMID: 38717058
4
CEP mutations cause absent or markedly reduced UROS activity leading to photoreactive porphyrinogen accumulation; clinical features include photosensitivity, bullous lesions, erythrodontia, hemolytic anemia; hematopoietic stem cell transplantation is curative in severe cases
PMID: 30685241
⚠Limited data available β€” This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜21
Congenital erythropoietic porphyriaOpen Targets
0.82Strong
cutaneous porphyriaOpen Targets
0.77Strong
neurodegenerative diseaseOpen Targets
0.43Moderate
genetic disorderOpen Targets
0.19Weak
Hemolytic anemia due to red cell pyruvate kinase deficiencyOpen Targets
0.06Suggestive
dehydrated hereditary stomatocytosisOpen Targets
0.06Suggestive
Adult-onset autosomal recessive sideroblastic anemiaOpen Targets
0.06Suggestive
Congenital dyserythropoietic anemia type IOpen Targets
0.06Suggestive
overhydrated hereditary stomatocytosisOpen Targets
0.06Suggestive
transient myeloproliferative syndromeOpen Targets
0.06Suggestive
sideroblastic anemia 3Open Targets
0.06Suggestive
non-spherocytic hemolytic anemia due to hexokinase deficiencyOpen Targets
0.05Suggestive
severe congenital hypochromic anemia with ringed sideroblastsOpen Targets
0.05Suggestive
hemoglobin E diseaseOpen Targets
0.05Suggestive
hemoglobin D diseaseOpen Targets
0.05Suggestive
dominant beta-thalassemiaOpen Targets
0.05Suggestive
hemoglobin H diseaseOpen Targets
0.05Suggestive
congenital dyserythropoietic anemia type 4Open Targets
0.05Suggestive
Congenital dyserythropoietic anemia type IVOpen Targets
0.05Suggestive
dehydrated hereditary stomatocytosis with or without pseudohyperkalemia and/or perinatal edemaOpen Targets
0.05Suggestive
Congenital erythropoietic porphyriaUniProt
Pathogenic Variants30
NM_000375.3(UROS):c.10C>T (p.Leu4Phe)Pathogenic
Cutaneous porphyria|not provided|UROS-related disorder
β˜…β˜…β˜†β˜†2026β†’ Residue 4
NM_000375.3(UROS):c.217T>C (p.Cys73Arg)Pathogenic
Cutaneous porphyria|not provided|UROS-related disorder
β˜…β˜…β˜†β˜†2025β†’ Residue 73
NM_000375.3(UROS):c.63+1G>APathogenic
Cutaneous porphyria|not provided|UROS-related disorder
β˜…β˜…β˜†β˜†2025
NM_000375.3(UROS):c.673G>A (p.Gly225Ser)Pathogenic
Cutaneous porphyria|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 225
NM_000375.3(UROS):c.7G>T (p.Val3Phe)Likely pathogenic
not provided|Cutaneous porphyria
β˜…β˜…β˜†β˜†2023β†’ Residue 3
NM_000375.3(UROS):c.562-2A>GLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_000375.3(UROS):c.139T>C (p.Ser47Pro)Likely pathogenic
Cutaneous porphyria
β˜…β˜†β˜†β˜†2024β†’ Residue 47
NM_000375.3(UROS):c.556C>T (p.Gln186Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 186
NM_000375.3(UROS):c.661-31T>GLikely pathogenic
Cutaneous porphyria
β˜…β˜†β˜†β˜†2023
NM_000375.3(UROS):c.-26-183G>APathogenic
Cutaneous porphyria|not provided
β˜…β˜†β˜†β˜†2023
NM_000375.3(UROS):c.693_749del (p.Ala234_Ala252del)Pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 234
NM_000375.3(UROS):c.634T>C (p.Ser212Pro)Pathogenic
not provided
β˜…β˜†β˜†β˜†2022β†’ Residue 212
NM_000375.3(UROS):c.395-2A>CPathogenic
not provided
β˜…β˜†β˜†β˜†2021
NM_000375.3(UROS):c.223G>T (p.Glu75Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2021β†’ Residue 75
NM_000375.3(UROS):c.710T>C (p.Leu237Pro)Pathogenic
not provided
β˜…β˜†β˜†β˜†2021β†’ Residue 237
NM_000375.3(UROS):c.633dup (p.Ser212fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2021β†’ Residue 212
NM_000375.3(UROS):c.296T>C (p.Val99Ala)Pathogenic
not provided
β˜…β˜†β˜†β˜†2021β†’ Residue 99
NM_000375.3(UROS):c.320-1G>CLikely pathogenic
Cutaneous porphyria
β˜…β˜†β˜†β˜†2021
NM_000375.3(UROS):c.-26-193C>APathogenic
Cutaneous porphyria|not provided
β˜…β˜†β˜†β˜†2021
NM_000375.3(UROS):c.56A>G (p.Tyr19Cys)Pathogenic
Cutaneous porphyria
β˜…β˜†β˜†β˜†2019β†’ Residue 19
View on ClinVar β†—
Related Genes
BCCIPProtein interaction96%ENDOVProtein interaction90%HMBSProtein interaction89%DHX32Protein interaction89%PPOXProtein interaction81%URODProtein interaction81%
Tissue Expression6 tissues
Bone Marrow
100%
Heart
72%
Liver
68%
Brain
67%
Ovary
35%
Lung
30%
Gene Interaction Network
Click a node to explore
UROSBCCIPENDOVHMBSDHX32PPOXUROD
PROTEIN STRUCTURE
Preparing viewer…
PDB1JR2 Β· 1.84 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.76LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.52 [0.36–0.76]
RankingsWhere UROS stands among ~20K protein-coding genes
  • #8,892of 20,598
    Most Researched50
  • #1,814of 5,498
    Most Pathogenic Variants30
  • #6,058of 17,882
    Most Constrained (LOEUF)0.76
Genes detectedUROS
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Congenital erythropoietic porphyria.
PMID: 38717058
Liver Int Β· 2024
1.00
2
Congenital erythropoietic porphyria: Recent advances.
PMID: 30685241
Mol Genet Metab Β· 2019
0.90
3
Regional assignment of the human uroporphyrinogen III synthase (UROS) gene to chromosome 10q25.2----q26.3.
PMID: 2037278
Hum Genet Β· 1991
0.80
4
Clinical Outcomes of Hypertonic Saline vs Mannitol Treatment Among Children With Traumatic Brain Injury.
PMID: 40067302
JAMA Netw Open Β· 2025
0.70
5
Ellagitannins, urolithins, and neuroprotection: Human evidence and the possible link to the gut microbiota.
PMID: 35940941
Mol Aspects Med Β· 2023
0.60