Target intelligence / Profile preview

Phenylalanine Hydroxylase (PAH)

Target: PAH
Molecular classification: Enzyme, Aromatic amino acid hydroxylase

Overview

Phenylalanine hydroxylase (PAH) is an enzyme that catalyzes the conversion of phenylalanine to tyrosine. This reaction is essential for the catabolism of phenylalanine and prevents toxic accumulation in tissues. Mutations in PAH cause phenylketonuria (PKU), leading to elevated phenylalanine levels and potential neurological damage if untreated.

Mechanism of action

N/A

Biological functions

Phenylalanine catabolismTyrosine synthesisAmino acid metabolism

Disease associations

Phenylketonuria (PKU)

Safety considerations

Elevated phenylalanine levels leading to neurological damage if PAH is deficientPotential for neurological damage due to high levels of phenylalanine in PKU patients

Biomarkers

Phenylalanine levels (for PKU diagnosis and monitoring)

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