Literature summary for 3.10.1.1 extracted from

Boado, R.J.; Lu, J.Z.; Hui, E.K.; Pardridge, W.M.
Reduction in brain heparan sulfate with systemic administration of an IgG Trojan horse-sulfamidase fusion protein in the mucopolysaccharidosis type IIIA mouse (2018), Mol. Pharm., 15, 602-608 .

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Organism

Organism	UniProt	Comment	Textmining
Mus musculus	Q9JHK6	-	-

Synonyms

Synonyms	Comment	Organism
SGSH	-	Mus musculus
sulfamidase	-	Mus musculus

General Information

General Information	Comment	Organism
malfunction	mucopolysaccharidosis Type IIIA (MPSIIIA, Sanfilippo A syndrome), is an inherited neurodegenerative disease caused by mutations in the lysosomal enzyme, N-sulfoglucosamine sulfohydrolase. Mutations in the SGSH enzyme, the only mammalian heparan N-sulfatase, cause accumulation of lysosomal inclusion bodies in brain cells comprising heparan sulfate glycosaminoglycans. Systemic administration of MPSIIIA mice with the cTfRMAb-SGSH (IgG-SGSH fusion protein, where the IgG domain is a chimeric monoclonal antibody (mAb) against the mouse transferrin receptor (TfR)) fusion protein causes a 70% reduction in brain heparan sulfate, the pathologic glycosaminoglycan of the central nervous system in MPSIIIA	Mus musculus

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Release 2023.1 (January 2023)