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Target intelligence / Profile preview
Tripeptidyl Peptidase 1 (TPP1)
- Target
- TPP1
- Molecular classification
- Enzyme, Serine Protease, Sedolisin family S53 serine proteases
01
Overview
Tripeptidyl peptidase I/TPP1 is an essential lysosomal enzyme involved in protein catabolism through sequential removal of N-terminal tripeptides from oligopeptide substrates. Its deficiency leads directly to neurodegenerative disease due to toxic accumulation within neurons—a hallmark feature underlying late-infantile neuronal ceroid lipofuscinosis (CLN2).
- Other names
- CLN2LPICSCAR7GIG1Tripeptidyl-peptidase I
02
Mechanism of action
Enzyme replacement therapy aims to restore TPP1 enzymatic activity in affected individuals. Small molecule chaperones may stabilize mutant TPP1.
03
Biological functions
Protein catabolismPeptide hydrolysisLysosomal protein degradationProtein recycling
04
Disease associations
Late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)Spinocerebellar ataxia autosomal recessive type 7 (SCAR7)Neurodegenerative disease
05
Safety considerations
Immune response to enzyme replacement therapyDelivery of enzyme to the central nervous system
06
Biomarkers
TPP1 enzyme activityTPP1 protein levelsCLN2 mutations
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