Article

Extracellular vesicles derived from cells overexpressing HGSNAT rescue defects in Mucopolysaccharidosis IIIC neurons

2026-07-23

Abstract excerpt

<h4>Summary</h4> Mucopolysaccharidosis III type C (MPS IIIC) is a rare neurological lysosomal storage disorder caused by genetic deficiency of the lysosomal membrane enzyme, heparan-α-glucosaminide N-acetyltransferase (HGSNAT). To assess the feasibility of therapeutic strategies based on cross-correction of neurons by HGSNAT secreted from transplanted cells overexpressing the enzyme, we generated induced cortical...

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Literature Corpus work: 09a51303-9b9f-5bd8-8f4a-b54cc93f64ac
DOI: 10.64898/2026.07.22.740105

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