Journal
Journal of medicinal chemistry
ISSN: 1520-4804
Titre abrégé: J Med Chem
Pays: United States
ID NLM: 9716531
Informations de publication
Date de publication:
09 02 2023
09 02 2023
Historique:
pubmed:
26
1
2023
medline:
11
2
2023
entrez:
25
1
2023
Statut:
ppublish
Résumé
Sanfilippo syndrome comprises a group of four genetic diseases due to the lack or decreased activity of enzymes involved in heparan sulfate (HS) catabolism. HS accumulation in lysosomes and other cellular compartments results in tissue and organ dysfunctions, leading to a wide range of clinical symptoms including severe neurodegeneration. To date, no approved treatments for Sanfilippo disease exist. Here, we report the ability of
Identifiants
pubmed: 36696678
doi: 10.1021/acs.jmedchem.2c01617
pmc: PMC9923752
doi:
Substances chimiques
Heparitin Sulfate
9050-30-0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
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