An Engineered
CRISPR/Cas9
Sanfilippo syndrome
animal disease model
childhood dementia
heparan sulfate
lysosomal storage disorder
mucopolysaccharidosis
neuroinflammation
zebrafish
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
31 May 2021
31 May 2021
Historique:
received:
16
04
2021
revised:
26
05
2021
accepted:
27
05
2021
entrez:
2
6
2021
pubmed:
3
6
2021
medline:
22
7
2021
Statut:
epublish
Résumé
Mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo syndrome type A), a paediatric neurological lysosomal storage disease, is caused by impaired function of the enzyme
Identifiants
pubmed: 34073041
pii: ijms22115948
doi: 10.3390/ijms22115948
pmc: PMC8197930
pii:
doi:
Substances chimiques
Hydrolases
EC 3.-
N-sulfoglucosamine sulfohydrolase
EC 3.10.1.1
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : National Health and Medical Research Council
ID : GNT1068411
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