Target intelligence / Profile preview

N-sulphoglucosamine sulphohydrolase (SGSH)

Target
SGSH
Molecular classification
Enzyme, Hydrolase
01

Overview

N-sulphoglucosamine sulphohydrolase is a lysosomal enzyme that catalyzes the removal of sulfate groups from glucosamine residues in heparan sulfate. It is essential for the degradation of glycosaminoglycans (GAGs) within lysosomes. Mutations in SGSH lead to mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a severe lysosomal storage disorder.

Other names
SulfamidaseHeparan N-sulfataseHeparan sulfate sulfataseHSSSFMDN-sulfoglucosamine sulfohydrolase
02

Mechanism of action

Hydrolyzes terminal N-sulfated glucosamines on heparan sulfate, removing sulfate groups.

03

Biological functions

Glycosaminoglycan catabolismLysosomal functionHeparan sulfate degradation
04

Disease associations

Mucopolysaccharidosis type IIIASanfilippo syndrome type A
05

Safety considerations

Therapeutic challenges in enzyme replacement therapy or gene therapy for MPS IIIA, including delivery to the central nervous system and potential immune responses.

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