Molecular Classification
Enzyme, Glycosidase, Lysosomal enzyme
Other Names
α-acetylglucosaminidase, N-acetyl-alpha-D-glucosaminidase, alpha-D-2-acetamido-2-deoxyglucosidase
Disease Roles
Alpha-N-acetylglucosaminidase 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).
Mechanism of Action
Hydrolysis of terminal non-reducing N-acetyl-D-glucosamine residues from N-acetyl-α-D-glucosaminides
Biological Functions
Glycosaminoglycan catabolism
Heparan sulfate degradation
Lysosomal storage
Disease Associations
Mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B)
Lysosomal storage disorder
Neurodegenerative disease
Safety Considerations
- Blood-brain barrier penetration for CNS-related manifestations of MPS IIIB
- Enzyme replacement therapy challenges
- Gene therapy delivery