Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations.
MT: RNA/DNA Editing
RNA interference
adhesion
allele-specific silencing
dominant centronuclear myopathy
dynamin 2
endocytosis
gene therapy
migration
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:
13 Sep 2022
13 Sep 2022
Historique:
received:
28
03
2022
accepted:
10
08
2022
entrez:
12
9
2022
pubmed:
13
9
2022
medline:
13
9
2022
Statut:
epublish
Résumé
Dominant centronuclear myopathy (CNM) is a rare form of congenital myopathy associated with a wide clinical spectrum, from severe neonatal to milder adult forms. There is no available treatment for this disease due to heterozygous mutations in the
Identifiants
pubmed: 36090755
doi: 10.1016/j.omtn.2022.08.016
pii: S2162-2531(22)00215-3
pmc: PMC9439966
doi:
Types de publication
Journal Article
Langues
eng
Pagination
733-748Informations de copyright
© 2022 The Authors.
Déclaration de conflit d'intérêts
The authors declare no competing interests.
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