Article

NDST1 as a substrate-reduction target in Mucopolysaccharidosis type IIIC: virtual screening, microsecond molecular dynamics, and peptide design

2026-08-11

Abstract excerpt

<h4>ABSTRACT</h4> Mucopolysaccharidosis IIIC (Sanfilippo syndrome type C) is a rare lysosomal storage disorder caused by loss-of-function mutations in HGSNAT , which encodes an enzyme involved in heparan sulfate (HS) degradation, leading to impaired HS catabolism, lysosomal accumulation, and progressive neurodegeneration. Because enzyme replacement therapies have limited penetration across the blood–brain barrie...

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Literature Corpus work: 136fa692-ecb0-5e40-a749-1fc86fc48f36
DOI: 10.64898/2026.08.09.743834

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