Systematic Review: Drug Repositioning for Congenital Disorders of Glycosylation (CDG).
AI in drug discovery
biomarkers
congenital disorders of glycosylation
disease models
drug repositioning
orphan drugs
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:
05 Aug 2022
05 Aug 2022
Historique:
received:
27
06
2022
revised:
02
08
2022
accepted:
02
08
2022
entrez:
12
8
2022
pubmed:
13
8
2022
medline:
16
8
2022
Statut:
epublish
Résumé
Advances in research have boosted therapy development for congenital disorders of glycosylation (CDG), a group of rare genetic disorders affecting protein and lipid glycosylation and glycosylphosphatidylinositol anchor biosynthesis. The (re)use of known drugs for novel medical purposes, known as drug repositioning, is growing for both common and rare disorders. The latest innovation concerns the rational search for repositioned molecules which also benefits from artificial intelligence (AI). Compared to traditional methods, drug repositioning accelerates the overall drug discovery process while saving costs. This is particularly valuable for rare diseases. AI tools have proven their worth in diagnosis, in disease classification and characterization, and ultimately in therapy discovery in rare diseases. The availability of biomarkers and reliable disease models is critical for research and development of new drugs, especially for rare and heterogeneous diseases such as CDG. This work reviews the literature related to repositioned drugs for CDG, discovered by serendipity or through a systemic approach. Recent advances in biomarkers and disease models are also outlined as well as stakeholders' views on AI for therapy discovery in CDG.
Identifiants
pubmed: 35955863
pii: ijms23158725
doi: 10.3390/ijms23158725
pmc: PMC9369176
pii:
doi:
Substances chimiques
Biomarkers
0
Types de publication
Journal Article
Review
Systematic Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Fundação para a Ciência e Tecnologia
ID : SFRH/BD/138647/2018
Organisme : Fundação para a Ciência e Tecnologia
ID : SFRH/BD/124326/2016
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