Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.
MBNL proteins
MT: Oligonucleotides: Therapies and Applications
PMO chemistry
alternative splicing
antisense oligonucleotides
biodistribution
cell-penetrating peptides
microRNAs
muscle dysfunction
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:
12 Dec 2023
12 Dec 2023
Historique:
received:
12
07
2022
accepted:
01
09
2023
medline:
25
9
2023
pubmed:
25
9
2023
entrez:
25
9
2023
Statut:
epublish
Résumé
Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by a CTG repeat expansion in the
Identifiants
pubmed: 37744174
doi: 10.1016/j.omtn.2023.09.001
pii: S2162-2531(23)00237-8
pmc: PMC10514136
doi:
Types de publication
Journal Article
Langues
eng
Pagination
102024Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
B.L. and R.A. are founders, and CEO and scientific consultant of Arthex Biotech, respectively. Arthex Biotech is developing antimiRs for use in the treatment of DM1.
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