Repurposing Dipyridamole in Niemann Pick Type C Disease: A Proof of Concept Study.
A2A receptor
Niemann Pick type C disease
adenosine
dipyridamole
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:
22 Mar 2022
22 Mar 2022
Historique:
received:
02
03
2022
revised:
17
03
2022
accepted:
21
03
2022
entrez:
12
4
2022
pubmed:
13
4
2022
medline:
14
4
2022
Statut:
epublish
Résumé
Niemann Pick type C disease (NPC) is a rare disorder characterized by lysosomal lipid accumulation that damages peripheral organs and the central nervous system. Currently, only miglustat is authorized for NPC treatment in Europe, and thus the identification of new therapies is necessary. The hypothesis addressed in this study is that increasing adenosine levels may represent a new therapeutic approach for NPC. In fact, a reduced level of adenosine has been shown in the brain of animal models of NPC; moreover, the compound T1-11, which is able to weakly stimulate A
Identifiants
pubmed: 35408815
pii: ijms23073456
doi: 10.3390/ijms23073456
pmc: PMC8999038
pii:
doi:
Substances chimiques
Dipyridamole
64ALC7F90C
Adenosine
K72T3FS567
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Istituto Superiore di Sanità
ID : Ricerca Corrente
Organisme : Istituto Superiore di Sanità
ID : ISS20-5cf22604a04d
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