Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.
CTG repeat expansions
MBNL1 protein
alternative splicing
antisense oligonucleotide
microRNAs
myotonic dystrophy
therapeutic gene modulation
tissue distribution
transcriptomics
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:
03 Dec 2021
03 Dec 2021
Historique:
received:
18
10
2020
accepted:
17
07
2021
entrez:
13
9
2021
pubmed:
14
9
2021
medline:
14
9
2021
Statut:
epublish
Résumé
Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by expansion of unstable CTG repeats in a non-coding region of the
Identifiants
pubmed: 34513303
doi: 10.1016/j.omtn.2021.07.017
pii: S2162-2531(21)00185-2
pmc: PMC8413838
doi:
Types de publication
Journal Article
Langues
eng
Pagination
174-191Informations de copyright
© 2021 The Author(s).
Déclaration de conflit d'intérêts
B.L., R.A., J.M.F.-C., and E.C.-H. are inventors in patent PCT/EP2017/073685. This patent is currently licensed to Arthex Biotech, of which B.L. and R.A. are founders and CEO and scientific consultant, respectively. B.L. and R.A. each hold more than 5% ownership of Arthex, a company with related interests.
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