CYP27B1

Chr 12AR

cytochrome P450 family 27 subfamily B member 1

Also known as: CP2B, CYP1, CYP1alpha, CYP27B, P450c1, PDDR, VDD1, VDDR

The protein is a cytochrome P450 monooxygenase that catalyzes the rate-limiting step in vitamin D activation by converting 25-hydroxyvitamin D3 to the hormonally active form 1α,25-dihydroxyvitamin D3, which regulates calcium and phosphorus homeostasis through the vitamin D receptor. Mutations cause vitamin D-dependent rickets type I with autosomal recessive inheritance, affecting bone mineralization and calcium metabolism. The gene shows very low constraint against loss-of-function variants (pLI near zero), consistent with recessive disease requiring biallelic mutations.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismARLOEUF 0.981 OMIM phenotype
Clinical SummaryCYP27B1
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Gene-Disease Validity (ClinGen)
vitamin D-dependent rickets, type 1A · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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Clinical Trials
2 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.98LOEUF
pLI 0.000
Z-score 1.62
OE 0.63 (0.410.98)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.07Z-score
OE missense 0.99 (0.901.09)
278 obs / 281.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.63 (0.410.98)
00.351.4
Missense OE0.99 (0.901.09)
00.61.4
Synonymous OE1.19
01.21.6
LoF obs/exp: 14 / 22.3Missense obs/exp: 278 / 281.1Syn Z: -1.67
DN
0.7132th %ile
GOF
0.7127th %ile
LOF
0.2873th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median
GOFprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

CYP27B1 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold
Clinical Literature
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