Benefits of therapy by dynamin-2-mutant-specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy.
RNA interference
allele-specific silencing
dominant centronuclear myopathy
dynamin 2
gene therapy
in vivo long-term benefits
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:
08 Mar 2022
08 Mar 2022
Historique:
received:
11
08
2021
accepted:
10
02
2022
entrez:
14
3
2022
pubmed:
15
3
2022
medline:
15
3
2022
Statut:
epublish
Résumé
Dominant dynamin 2 (DNM2) mutations are responsible for the autosomal dominant centronuclear myopathy (AD-CNM), a rare progressive neuromuscular disorder ranging from severe neonatal to mild adult forms. We previously demonstrated that mutant-specific RNA interference is an efficient therapeutic strategy to rescue the muscle phenotype at the onset of the symptoms in the AD-CNM knockin-
Identifiants
pubmed: 35282416
doi: 10.1016/j.omtn.2022.02.009
pii: S2162-2531(22)00037-3
pmc: PMC8889367
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1179-1190Informations de copyright
© 2022 The Author(s).
Déclaration de conflit d'intérêts
The authors declare no competing interests.
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