Renadirsen, a Novel 2'OMeRNA/ENA
Alternative Splicing
Animals
Chromatography, Liquid
Dystrophin
/ genetics
Male
Mice
Mice, Inbred mdx
Molecular Structure
Muscle, Skeletal
/ metabolism
Myocardium
/ metabolism
Oligodeoxyribonucleotides
/ chemistry
Oligonucleotides, Antisense
/ administration & dosage
Oligoribonucleotides
/ chemistry
Tandem Mass Spectrometry
Tissue Distribution
2′-O,4′-C-ethylene-bridged nucleic acid (ENA)
Duchenne muscular dystrophy
antisense oligonucleotide
cardiac muscle
dystrophin
exon skipping
renadirsen sodium
Journal
Current issues in molecular biology
ISSN: 1467-3045
Titre abrégé: Curr Issues Mol Biol
Pays: Switzerland
ID NLM: 100931761
Informations de publication
Date de publication:
25 Sep 2021
25 Sep 2021
Historique:
received:
02
08
2021
revised:
22
09
2021
accepted:
22
09
2021
entrez:
26
10
2021
pubmed:
27
10
2021
medline:
16
2
2022
Statut:
epublish
Résumé
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by out-of-frame or nonsense mutation in the dystrophin gene. It begins with a loss of ambulation between 9 and 14 years of age, followed by various other symptoms including cardiac dysfunction. Exon skipping of patients'
Identifiants
pubmed: 34698059
pii: cimb43030090
doi: 10.3390/cimb43030090
pmc: PMC8928966
doi:
Substances chimiques
Dystrophin
0
Oligodeoxyribonucleotides
0
Oligonucleotides, Antisense
0
Oligoribonucleotides
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1267-1281Subventions
Organisme : Daiichi Sankyo Co., Ltd.
Organisme : Orphan Disease Treatment Institute Co., Ltd.
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