An Engineered <i>sgsh</i> Mutant Zebrafish Recapitulates Molecular and Behavioural Pathobiology of Sanfilippo Syndrome A/MPS IIIA

An Engineered <i>sgsh</i> Mutant Zebrafish Recapitulates Molecular and Behavioural Pathobiology of Sanfilippo Syndrome A/MPS IIIA

Mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo syndrome type A), a paediatric neurological lysosomal storage disease, is caused by impaired function of the enzyme <i>N</i>-sulfoglucosamine sulfohydrolase (SGSH) resulting in impaired catabolism of heparan sulfate glycosaminoglycan (HS G...

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Bibliographic Details
Main Authors: Alon M. Douek, Mitra Amiri Khabooshan, Jason Henry, Sebastian-Alexander Stamatis, Florian Kreuder, Georg Ramm, Minna-Liisa Änkö, Donald Wlodkowic, Jan Kaslin
Format: Article
Language:English
Published: MDPI AG 2021-05-01
Series:International Journal of Molecular Sciences
Subjects:
mucopolysaccharidosis
Sanfilippo syndrome
lysosomal storage disorder
childhood dementia
heparan sulfate
zebrafish
Online Access:https://www.mdpi.com/1422-0067/22/11/5948

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https://www.mdpi.com/1422-0067/22/11/5948

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