Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy.
CUGBP1
DMSXL mice
GSK3 inhibitor
GSK3β
HSALR mice
Myotonic Dystrophy (DM1)
brain atrophy
congenital Myotonic Dystrophy
development of therapy
myotonia
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:
26 Jun 2023
26 Jun 2023
Historique:
received:
01
06
2023
revised:
19
06
2023
accepted:
21
06
2023
medline:
17
7
2023
pubmed:
14
7
2023
entrez:
14
7
2023
Statut:
epublish
Résumé
Myotonic Dystrophy type 1 (DM1) is a neuromuscular disease associated with toxic RNA containing expanded CUG repeats. The developing therapeutic approaches to DM1 target mutant RNA or correct early toxic events downstream of the mutant RNA. We have previously described the benefits of the correction of the GSK3β-CUGBP1 pathway in DM1 mice (
Identifiants
pubmed: 37445828
pii: ijms241310650
doi: 10.3390/ijms241310650
pmc: PMC10342152
pii:
doi:
Substances chimiques
Glycogen Synthase Kinase 3 beta
EC 2.7.11.1
Glycogen Synthase Kinase 3
EC 2.7.11.26
RNA
63231-63-0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : NIAMS NIH HHS
ID : R01 AR073379
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS115662
Pays : United States
Organisme : NIH HHS
ID : 5RO1AR073379
Pays : United States
Organisme : NIH HHS
ID : 5RO1NS115662
Pays : United States
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