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Mucopolysaccharidosis type IIIA (MPS-IIIA) is a severe neurodegenerative lysosomal storage disorder caused by a deficiency of N-sulfoglucosamine sulfohydrolase (SGSH) activity with subsequent accumulation of partially-degraded heparan sulfate and other glycolipids. In this study, we have evaluated a gene therapy approach using a helper-dependent canine adenovirus vector that expresses human SGSH as...
Sulphamidase is a lysosomal enzyme necessary for the degradation of heparan sulphate. The deficiency of this hydrolase causes a disorder known as mucopolysaccharidosis type IIIA, characterized by a profound neurological deterioration. Human and mouse exon-intron structures were reported without any characterization of their promoter regions [DNA Res. 3 (1996) 269; Mamm. Genome 11 (2000) 436]. The...
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