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GTP-cyclohydrolase 1 (GCH1)
- Target
- GCH1
- Molecular classification
- Enzyme
01
Overview
GTP-cyclohydrolase 1 (GCH1) is an enzyme that catalyzes the hydrolysis of guanosine triphosphate (GTP) to form 7,8-dihydroneopterin triphosphate, the first and rate-limiting step in the biosynthesis of tetrahydrobiopterin (BH4). BH4 is a critical cofactor for several enzymes involved in amino acid metabolism and neurotransmitter synthesis. Mutations in GCH1 are associated with dopa-responsive dystonia and hyperphenylalaninemia. GCH1 activity is regulated allosterically by the feedback regulatory protein GFRP.
- Other names
- GTP cyclohydrolase IGTPCH
02
Mechanism of action
Not applicable; L-Dopa is a substrate of downstream enzymes, not a direct GCH1 modulator.
03
Biological functions
Tetrahydrobiopterin biosynthesisNeurotransmitter synthesisRegulation of nitric oxide synthesisPhenylalanine metabolism
04
Disease associations
Dopa-responsive dystoniaHyperphenylalaninemia/PKUChronic pain syndromesCardiovascular diseaseNeurodegenerative diseaseCancer
05
Safety considerations
Potential for neurological side effects due to altered neurotransmitter levelsRisk of hyperphenylalaninemia if BH4 deficiency is not properly managed
06
Interacting drugs
L-Dopa
07
Biomarkers
BH4 levelsPhenylalanine levelsGCH1 activity
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