PCBD1

Chr 10AR

pterin-4 alpha-carbinolamine dehydratase 1

Also known as: DCOH, PCBD, PCD, PHS

The encoded protein functions as a dehydratase in tetrahydrobiopterin biosynthesis and as a cofactor for HNF1A-dependent transcription. Mutations cause BH4-deficient hyperphenylalaninemia type D through autosomal recessive inheritance. Enzyme deficiency disrupts tetrahydrobiopterin metabolism, leading to elevated phenylalanine levels.

Summary from RefSeq, OMIM, UniProt, Mechanism
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Primary Disease Associations & Inheritance

Hyperphenylalaninemia, BH4-deficient, DMIM #264070
AR
0
Active trials
5
Pubs (1 yr)
0
P/LP submissions
P/LP missense
1.83
LOEUF
LOF
Mechanism· G2P
Clinical SummaryPCBD1
🧬
Gene-Disease Validity (ClinGen)
pterin-4 alpha-carbinolamine dehydratase 1 deficiency · 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.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.83LOEUF
pLI 0.000
Z-score -0.11
OE 1.05 (0.541.83)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
0.72Z-score
OE missense 0.73 (0.570.95)
42 obs / 57.2 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE1.05 (0.541.83)
00.351.4
Missense OE0.73 (0.570.95)
00.61.4
Synonymous OE1.13
01.21.6
LoF obs/exp: 5 / 4.7Missense obs/exp: 42 / 57.2Syn Z: -0.49
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePCBD1-related hyperphenylalaninemia, BH4-deficient, DLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.7132th %ile
GOF
0.6442th %ile
LOF
0.3454th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PCBD1 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC