Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy.
DM1
HSALR mice
Mbnl1
antagomiR
antisense oligonucleotides
miRNA
myotonic dystrophy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
04 Sep 2020
04 Sep 2020
Historique:
received:
16
04
2020
revised:
06
05
2020
accepted:
15
07
2020
pubmed:
18
8
2020
medline:
18
8
2020
entrez:
18
8
2020
Statut:
ppublish
Résumé
Myotonic dystrophy type 1 (DM1) is a chronically debilitating, rare genetic disease that originates from an expansion of a noncoding CTG repeat in the dystrophia myotonica protein kinase (DMPK) gene. The expansion becomes pathogenic when DMPK transcripts contain 50 or more repetitions due to the sequestration of the muscleblind-like (MBNL) family of proteins. Depletion of MBNLs causes alterations in splicing patterns in transcripts that contribute to clinical symptoms such as myotonia and muscle weakness and wasting. We previously found that microRNA (miR)-23b directly regulates MBNL1 in DM1 myoblasts and mice and that antisense technology ("antagomiRs") blocking this microRNA (miRNA) boosts MBNL1 protein levels. Here, we show the therapeutic effect over time in response to administration of antagomiR-23b as a treatment in human skeletal actin long repeat (HSA
Identifiants
pubmed: 32805487
pii: S2162-2531(20)30210-9
doi: 10.1016/j.omtn.2020.07.021
pmc: PMC7452101
pii:
doi:
Types de publication
Journal Article
Langues
eng
Pagination
837-849Informations de copyright
Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.
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