Chemical Therapies for Congenital Disorders of Glycosylation.
Journal
ACS chemical biology
ISSN: 1554-8937
Titre abrégé: ACS Chem Biol
Pays: United States
ID NLM: 101282906
Informations de publication
Date de publication:
18 11 2022
18 11 2022
Historique:
pubmed:
18
11
2021
medline:
22
11
2022
entrez:
17
11
2021
Statut:
ppublish
Résumé
Congenital disorders of glycosylation (CDG) are ultrarare, genetically and clinically heterogeneous metabolic disorders. Although the number of identified CDG is growing rapidly, there are few therapeutic options. Most treatments involve dietary supplementation with monosaccharides or other precursors. These approaches are relatively safe, but in many cases, the molecular and biochemical underpinnings are incomplete. Recent studies demonstrate that yeast, worm, fly, and zebrafish models of CDG are powerful tools in screening repurposed drugs, ushering a new avenue to search for novel therapeutic options. Here we present a perspective on compounds that are currently in use for CDG treatment or have a potential to be applied as therapeutics in the near future.
Identifiants
pubmed: 34788024
doi: 10.1021/acschembio.1c00601
pmc: PMC9126425
mid: NIHMS1807387
doi:
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
2962-2971Subventions
Organisme : NIDDK NIH HHS
ID : R01 DK099551
Pays : United States
Organisme : NINDS NIH HHS
ID : U54 NS115198
Pays : United States
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