Correction of Glycogen Synthase Kinase 3β in Myotonic Dystrophy 1 Reduces the Mutant RNA and Improves Postnatal Survival of DMSXL Mice.

Animals CELF1 Protein / genetics DNA-Binding Proteins / genetics Disease Models, Animal Glycogen Synthase Kinase 3 / metabolism Glycogen Synthase Kinase 3 beta / antagonists & inhibitors Humans Mice Muscle, Skeletal / metabolism Myoblasts / metabolism Myotonic Dystrophy / genetics Primary Cell Culture RNA / metabolism RNA, Messenger / metabolism RNA-Binding Proteins / genetics Thiadiazoles / pharmacology
GSK3β congenital myotonic dystrophy myotonic dystrophy type 1

Journal

Molecular and cellular biology
ISSN: 1098-5549
Titre abrégé: Mol Cell Biol
Pays: United States
ID NLM: 8109087

Informations de publication

Date de publication:
01 11 2019
Historique:
received: 09 04 2019
accepted: 01 08 2019
pubmed: 7 8 2019
medline: 27 2 2020
entrez: 7 8 2019
Statut: epublish

Résumé

Myotonic dystrophy type 1 (DM1) is a multisystem neuromuscular disease without cure. One of the possible therapeutic approaches for DM1 is correction of the RNA-binding proteins CUGBP1 and MBNL1, misregulated in DM1. CUGBP1 activity is controlled by glycogen synthase kinase 3β (GSK3β), which is elevated in skeletal muscle of patients with DM1, and inhibitors of GSK3 were suggested as therapeutic molecules to correct CUGBP1 activity in DM1. Here, we describe that correction of GSK3β with a small-molecule inhibitor of GSK3, tideglusib (TG), not only normalizes the GSK3β-CUGBP1 pathway but also reduces the mutant

Identifiants

DOI: 10.1128/MCB.00155-19 PMID: 31383751 PMC: PMC6791656
pubmed: 31383751
pii: MCB.00155-19
doi: 10.1128/MCB.00155-19
pmc: PMC6791656
pii:
doi:

Substances chimiques

CELF1 Protein 0
CELF1 protein, mouse 0
DNA-Binding Proteins 0
Mbnl1 protein, mouse 0
RNA, Messenger 0
RNA-Binding Proteins 0
Thiadiazoles 0
RNA 63231-63-0
Glycogen Synthase Kinase 3 beta EC 2.7.11.1
Gsk3b protein, mouse EC 2.7.11.1
Glycogen Synthase Kinase 3 EC 2.7.11.26
tideglusib Q747Y6TT42

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NIAMS NIH HHS
ID : R01 AR073379
Pays : United States

Informations de copyright

Copyright © 2019 American Society for Microbiology.

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Auteurs

Mei Wang (M)

Division of Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio, USA.

Wen-Chin Weng (WC)

Division of Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio, USA.

Lauren Stock (L)

Division of Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio, USA.

Diana Lindquist (D)

Imaging Research Center, Cincinnati Children's Hospital, Cincinnati, Ohio, USA.

Ana Martinez (A)

Centro de Investigaciones Biológicas (CIB, CSIC), Madrid, Spain.

Genevieve Gourdon (G)

Inserm UMR1163, Institut Imagine, Université Paris Descartes-Sorbonne Paris Cité, Paris, France.

Nikolai Timchenko (N)

Departments of Surgery, Cincinnati Children's Hospital, University of Cincinnati, College of Medicine, Cincinnati, Ohio, USA.
Departments of Pediatrics, University of Cincinnati, College of Medicine, Cincinnati, Ohio, USA.

Mike Snape (M)

AMO Pharma Ltd., Wonersh, Surrey, United Kingdom.

Lubov Timchenko (L)

Division of Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio, USA Lubov.Timchenko@cchmc.org.
Departments of Pediatrics, University of Cincinnati, College of Medicine, Cincinnati, Ohio, USA.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines de liaison à l'ARN › Protéines à motif de reconnaissance de l'ARN › Protéines CELF › Protéine CELF1 › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines de liaison à l'ADN › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Techniques d'investigation › Modèles théoriques › Modèles biologiques › Modèles animaux de maladie humaine › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines et peptides de signalisation intracellulaire › Protein-Serine-Threonine Kinases › Proline-directed protein kinases › Glycogen Synthase Kinase 3 › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines et peptides de signalisation intracellulaire › Protein-Serine-Threonine Kinases › Proline-directed protein kinases › Glycogen Synthase Kinase 3 › Glycogen synthase kinase 3 beta › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Tissus › Muscles › Muscle strié › Muscles squelettiques › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Cellules › Cellules souches › Myoblastes › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Dystrophies musculaires › Dystrophie myotonique › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Techniques d'investigation › Techniques in vitro › Techniques de culture › Techniques de culture cellulaire › Culture de cellules primaires › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › ARN messager › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines de liaison à l'ARN › Correction of Glycogen Synthase Kinase 3β in...
questionsmedicales.fr › Composés hétérocycliques › Composés hétéromonocycliques › Azoles › Thiazoles › Thiadiazoles › Correction of Glycogen Synthase Kinase 3β in...