Back to discover

Protein / target

25-hydroxyvitamin D-1 alpha hydroxylase, mitochondrial

Encoded byCYP27B1O15528Homo sapiensSwiss-Prot
Small-molecule tractable
Druggability
High-Quality Ligand
1
Research papers

Protein at a glance

Biological role

Secalciferol 1-monooxygenase

Strongest disease association

Genetic Diseases, Inborn

Via encoding gene CYP27B1 · Genetic evidence · score 0.68

Research activity

Emerging research

1 papers · latest 2023

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

A cytochrome P450 monooxygenase involved in vitamin D metabolism and in calcium and phosphorus homeostasis.

View complete UniProt function annotation

A cytochrome P450 monooxygenase involved in vitamin D metabolism and in calcium and phosphorus homeostasis. Catalyzes the rate-limiting step in the activation of vitamin D in the kidney, namely the hydroxylation of 25-hydroxyvitamin D3/calcidiol at the C1alpha-position to form the hormonally active form of vitamin D3, 1alpha,25-dihydroxyvitamin D3/calcitriol that acts via the vitamin D receptor (VDR) (PubMed:10518789, PubMed:10566658, PubMed:12050193, PubMed:22862690, PubMed:9486994). Has 1alpha-hydroxylase activity on vitamin D intermediates of the CYP24A1-mediated inactivation pathway (PubMed:10518789, PubMed:22862690). Converts 24R,25-dihydroxyvitamin D3/secalciferol to 1-alpha,24,25-trihydroxyvitamin D3, an active ligand of VDR. Also active on 25-hydroxyvitamin D2 (PubMed:10518789). Mechanistically, uses molecular oxygen inserting one oxygen atom into a substrate, and reducing the second into a water molecule, with two electrons provided by NADPH via FDXR/adrenodoxin reductase and FDX1/adrenodoxin (PubMed:22862690)

Subcellular location

Mitochondrion membrane
Domains and Gene Ontology detail (26)

Gene Ontology

  • Ccytoplasm
  • Cmitochondrial outer membrane
  • Cmitochondrion
  • Fcalcidiol 1-monooxygenase activity
  • Fheme binding
  • Firon ion binding
  • Fsecalciferol 1-monooxygenase activity
  • Pbone mineralization
  • Pcalcitriol biosynthetic process from calciol
  • Pcalcium ion homeostasis
  • Pcalcium ion transport
  • Pcellular response to vitamin D

508 aa · 57 kDa

Biological roles

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Cell proliferation & survivalGOMetabolic enzyme activityGO
View supporting evidence

Cell proliferation & survival

  • ·negative regulation of cell population proliferation

Metabolic enzyme activity

  • ·vitamin D metabolic process
  • ·vitamin metabolic process

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene CYP27B1

Gene-level evidence surfaced through the gene CYP27B1 that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Genetic Diseases, Inborn
0.68Moderately supported

Genetic evidence dominant · Open Targets 0.42

Rickets
0.27Preliminary

Pathway evidence dominant · Open Targets 0.38 · no direct causal or clinical evidence

Arthritis, Rheumatoid
0.22Preliminary

Animal model evidence dominant · Open Targets 0.09 · no direct causal or clinical evidence

Diabetes Mellitus, Type 1
0.10Preliminary

Literature evidence dominant · Open Targets 0.09 · no direct causal or clinical evidence

View evidence synthesis (4)
Genetic Diseases, InbornModerately supported
0.68
agreement 0.540.82
Genetic100%Literature0%

Open Targets aggregate 0.42 · 2 independent evidence families

RicketsPreliminary
0.27
agreement 0.100.45
Pathway86%Literature14%

Open Targets aggregate 0.38 · 2 independent evidence families · no direct causal or clinical evidence

Arthritis, RheumatoidPreliminary
0.22
agreement 0.040.40
Animal model56%Literature44%

Open Targets aggregate 0.09 · 2 independent evidence families · no direct causal or clinical evidence

Diabetes Mellitus, Type 1Preliminary
0.10
agreement 0.000.38
Literature100%

Open Targets aggregate 0.09 · 1 independent evidence family · no direct causal or clinical evidence

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Genetic Diseases, Inborn0.42
Rickets0.38
Arthritis, Rheumatoid0.09
Diabetes Mellitus, Type 10.09

Tractability

Small moleculesEmerging

Feasibility evidence (high-quality ligand and druggable family) — no clinical-stage drug of this modality recorded.

Protein degradersEmerging

Feasibility evidence (small molecule binder) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (3)
SM · High-Quality LigandSM · Druggable FamilyPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

nephrotoxicityClinPGx

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

Research activity

1 papers · to 2023

Papers linked directly to this protein. This is the protein's own literature — descriptor-derived papers are kept separate below.

Most cited

Hrabia A · International journal of molecular sciences · 2023

Recent

Vitamin D<sub>3</sub> Receptors and Metabolic Enzymes in Hen Reproductive Tissues.

Hrabia A · International journal of molecular sciences · 2023

Europe PMC papers linked directly to this protein.