Title : Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.

Pub. Date : 2007 Nov 15

PMID : 17725987






2 Functional Relationships(s)
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Protein Name
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1 Mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome) is a lysosomal storage disorder caused by the congenital deficiency of sulfamidase (SGSH) enzyme and consequent accumulation of partially degraded heparan sulfate (HS) in lysosomes. Heparitin Sulfate N-sulfoglucosamine sulfohydrolase (sulfamidase) Mus musculus
2 Mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome) is a lysosomal storage disorder caused by the congenital deficiency of sulfamidase (SGSH) enzyme and consequent accumulation of partially degraded heparan sulfate (HS) in lysosomes. Heparitin Sulfate N-sulfoglucosamine sulfohydrolase (sulfamidase) Mus musculus