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N-glycanase 1 (NGLY1)
- Target
- NGLY1
- Molecular classification
- Enzyme, Glycosidase
01
Overview
N-glycanase 1 (NGLY1) is a cytosolic enzyme that removes N-linked glycans from misfolded glycoproteins, a critical step in ER-associated degradation (ERAD) and protein quality control. Mutations in NGLY1 cause NGLY1 deficiency, a congenital disorder characterized by developmental delay, neurological issues, and liver disease.
- Other names
- NGL1PNGase
02
Mechanism of action
Small molecules to stabilize mutant forms or compensate for loss-of-function effects
03
Biological functions
ER-associated degradation (ERAD)Proteasomal degradationProtein quality controlDeglycosylation
04
Disease associations
NGLY1 deficiencyCongenital disorder of deglycosylationNeurodegenerative disease (potential)
05
Safety considerations
Loss of NGLY1 function leads to accumulation of improperly processed glycoproteinsMultisystemic effects due to impaired protein clearance
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