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Target intelligence / Profile preview
Palmitoyl-protein thioesterase 1 (PPT1)
- Target
- PPT1
- Molecular classification
- Enzyme, Thioester hydrolase
01
Overview
Palmitoyl-protein thioesterase 1 (PPT1) is a lysosomal enzyme that removes long-chain fatty acids, such as palmitate, from cysteine residues on S-palmitoylated proteins. This depalmitoylation is crucial for protein degradation and lysosomal recycling. Deficiency in PPT1 leads to neuronal ceroid lipofuscinosis type 1 (CLN1), a severe neurodegenerative lysosomal storage disorder.
- Other names
- PPTPPT-1CLN1INCLpalmitoyl-protein hydrolase 1ceroid-lipofuscinosis neuronal 1
02
Mechanism of action
Hydrolyzes thioester bonds of S-palmitoylated proteins
03
Biological functions
Protein catabolismLysosomal recyclingSynapse developmentProtein depalmitoylation
04
Disease associations
Neuronal ceroid lipofuscinosis type 1 (CLN1)Infantile Batten diseaseNeurodegenerative diseaseLysosomal storage disorder
05
Safety considerations
Neurotoxicity due to accumulation of ceroid lipofuscinProgressive mental and motor deteriorationSeizuresVision lossEarly death
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