Gene Information

PAH

phenylalanine hydroxylase [Source:HGNC Symbol;Acc:HGNC:8582]

protein_codingChromosome 12ENSG00000171759

Overview

PAH variants are associated with 5 diseases including maternal phenylketonuria and classic phenylketonuria and 3 others, with 30 documented clinical phenotypes.


Associated Diseases

The PAH gene is associated with 5 medical conditions, including:

maternal phenylketonuria

MONDO:0016366

classic phenylketonuria

MONDO:0019259

tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria

MONDO:0017389

mild hyperphenylalaninemia

MONDO:0019335

mild phenylketonuria

MONDO:0019258


Clinical Phenotypes

Research has identified 30 phenotypic features associated with PAH variants:

View all


ClinVar Variants

ClinVar contains 10 variants associated with PAH. Below are some notable variants:

NM_000277.3(PAH):c.1093del (p.Leu365fs)

Likely pathogenic

VCV004059237

Review: no assertion criteria provided

NM_000277.3(PAH):c.851G>A (p.Cys284Tyr)

Likely pathogenic

VCV004059225

Review: no assertion criteria provided

NM_000277.3(PAH):c.838G>T (p.Glu280Ter)

Likely pathogenic

VCV004059218

Review: no assertion criteria provided

NM_000277.3(PAH):c.185_188dup (p.His64fs)

Pathogenic

VCV003907324

Review: criteria provided, single submitter

NM_000277.3(PAH):c.139G>T (p.Ala47Ser)

Uncertain significance

VCV003907193

Review: criteria provided, single submitter


Recent Publications

There are 10,183 publications about PAH indexed in PubMed. Below are some recent papers:

Whole genome sequencing, characterization and analysis of coronene degrading bacterial strain Halomonas elongata.

Rafic T, Alarawi M, Alkhnbashi OS

PMID: 41259345

2025

PLoS One

Bihourly Monitoring Reveals the Significance of Photochemical Removal of PAHs in the Urban Atmosphere.

Cheng Z, Qiu X, Shi X

PMID: 41258652

2025 Nov 19

Environ Sci Technol

Characteristics, Treatment Patterns and Outcomes of Patients with Pulmonary Arterial Hypertension by Race and Ethnicity Using Real-World Data from the Combined OPUS/OrPHeUS Studies.

Melendres-Groves LD, Channick RN, Chin KM

PMID: 41258625

2025 Nov 19

Adv Ther

Liver angiocrine myeloid-derived growth factor protects against endothelial dysfunction in pulmonary arterial hypertension.

Singh N, Volpicelli T, Pi H

PMID: 41256637

2025 Oct 4

bioRxiv

VUV Photoionization Study of the Gas Formation of the Indene-A Molecular Building Block of Nonplanar PAHs.

Li W, Zhang YF, Wang CY

PMID: 41254491

2025 Nov 18

J Phys Chem A

View all 10,183 publications on PubMed →

Analyze PAH Variants with AI

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Trusted Data Sources

EnsemblMonarch InitiativeClinVarGeneReviewsPubMed

Last updated: 11/20/2025

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