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Target intelligence / Profile preview
Alpha-N-acetylglucosaminidase (NAGLU)
- Target
- NAGLU
- Molecular classification
- Enzyme, Glycosidase, Lysosomal enzyme
01
Overview
Alpha-N-acetylglucosaminidase is a lysosomal enzyme encoded by the NAGLU gene that catalyzes the hydrolysis of terminal non-reducing N-acetyl-D-glucosamine residues from N-acetyl-α-D-glucosaminides, playing a critical role in the degradation of heparan sulfate. Deficiency leads to mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B).
- Other names
- α-acetylglucosaminidaseN-acetyl-alpha-D-glucosaminidasealpha-D-2-acetamido-2-deoxyglucosidase
02
Mechanism of action
Hydrolysis of terminal non-reducing N-acetyl-D-glucosamine residues from N-acetyl-α-D-glucosaminides
03
Biological functions
Glycosaminoglycan catabolismHeparan sulfate degradationLysosomal storage
04
Disease associations
Mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B)Lysosomal storage disorderNeurodegenerative disease
05
Safety considerations
Blood-brain barrier penetration for CNS-related manifestations of MPS IIIBEnzyme replacement therapy challengesGene therapy delivery
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