Early defects in mucopolysaccharidosis type IIIC disrupt excitatory synaptic transmission

The majority of patients affected with lysosomal storage disorders (LSD) exhibit neurological symptoms. For mucopolysaccharidosis type IIIC (MPSIIIC), the major burdens are progressive and severe neuropsychiatric problems and dementia, primarily thought to stem from neurodegeneration. Using the MPSI...

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Bibliographic Details
Main Authors: Camila Pará, Poulomee Bose, Luigi Bruno, Erika Freemantle, Mahsa Taherzadeh, Xuefang Pan, Chanshuai Han, Peter S. McPherson, Jean-Claude Lacaille, Éric Bonneil, Pierre Thibault, Claire O’Leary, Brian Bigger, Carlos Ramon Morales, Graziella Di Cristo, Alexey V. Pshezhetsky
Format: Article
Language:English
Published: American Society for Clinical investigation 2021-08-01
Series:JCI Insight
Subjects:
Online Access:https://doi.org/10.1172/jci.insight.142073