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Target intelligence / Profile preview
Alpha-N-acetyl-glucosaminidase (NAGLU)
- Target
- NAGLU
- Molecular classification
- Enzyme, Glycosidase, Lysosomal enzyme
01
Overview
Alpha-N-acetylglucosaminidase (NAGLU) is a lysosomal enzyme that catalyzes the hydrolysis of terminal non-reducing N-acetyl-D-glucosamine residues from heparan sulfate, a glycosaminoglycan. It is essential for the degradation of heparan sulfate. Deficiency of NAGLU causes Sanfilippo syndrome type B (MPS IIIB), a lysosomal storage disorder characterized by the accumulation of heparan sulfate, leading to neurodegeneration and other clinical manifestations.
- Other names
- α-N-acetylglucosaminidaseAlpha-acetylglucosaminidaseN-acetyl-α-D-glucosaminidaseN-acetyl-alpha-glucosaminidaseα-D-2-acetamido-2-deoxyglucosidase
02
Mechanism of action
Hydrolyzes terminal non-reducing N-acetyl-D-glucosamine residues from heparan sulfate and other substrates.
03
Biological functions
Glycosaminoglycan catabolic processHeparan sulfate catabolismHydrolysis of glycosidic bonds
04
Disease associations
Sanfilippo syndrome type BMucopolysaccharidosis IIIBLysosomal storage disorderNeurodegenerative disease
05
Safety considerations
Enzyme replacement therapy efficacyGene therapy efficacyBlood-brain barrier penetration for CNS effectsPotential for immune response against therapeutic enzyme
06
Biomarkers
Alpha-N-acetylglucosaminidase activity (for diagnosis)Heparan sulfate levels
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