Search Results - "Tamang, Sarah J."
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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
Published in Molecular therapy. Methods & clinical development (12-06-2020)“…Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme sulfamidase (SGSH), which is responsible for the degradation of heparan…”
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MPS-IIIA mice acquire autistic behaviours with age
Published in Journal of inherited metabolic disease (01-07-2018)“…Mucopolysaccharidosis (MPS) type IIIA is an inherited, neurodegenerative lysosomal storage disorder resulting from mutations in the SGSH gene. Consequently, N…”
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A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome
Published in Journal of inherited metabolic disease (01-09-2017)“…Mucopolysaccharidosis (MPS) type IIIA, or Sanfilippo syndrome, is a neurodegenerative lysosomal storage disorder caused by a deficiency of the lysosomal enzyme…”
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Journal Article