SGSH

N-sulfoglucosamine sulfohydrolase

Normal Function

Health Conditions Related to Genetic Changes

Mucopolysaccharidosis type III

More than 80 mutations in the SGSH gene have been found to cause mucopolysaccharidosis type IIIA (MPS IIIA). Most of these mutations change single DNA building blocks (nucleotides) in the gene. All of the mutations that cause MPS IIIA reduce or eliminate the function of sulfamidase.

The lack of sulfamidase activity disrupts the breakdown of a subset of GAGs called heparan sulfate. As a result, partially broken down heparan sulfate accumulates within lysosomes. Researchers believe that the accumulation of GAGs interferes with the functions of other proteins inside the lysosomes and disrupts the normal functions of cells. It is unknown why the buildup of heparan sulfate mostly affects the central nervous system in MPS IIIA.

More About This Health Condition

Related Conditions

Mucopolysaccharidosis type III

Health Conditions Related to Genetic Changes

More than 80 mutations in the SGSH gene have been found to cause mucopolysaccharidosis type IIIA (MPS IIIA). Most of these mutations change single DNA building blocks (nucleotides) in the gene. All of the mutations that cause MPS IIIA reduce or eliminate the function of sulfamidase.

The lack of sulfamidase activity disrupts the breakdown of a subset of GAGs called heparan sulfate. As a result, partially broken down heparan sulfate accumulates within lysosomes. Researchers believe that the accumulation of GAGs interferes with the functions of other proteins inside the lysosomes and disrupts the normal functions of cells. It is unknown why the buildup of heparan sulfate mostly affects the central nervous system in MPS IIIA.