Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy.
2′-O-methyl-phosphorothioate (2′-OMe PS)
Duchenne muscular dystrophy
antisense oligonucleotide
bile acid
conjugation
exon skipping
lipophilic compound
ursodeoxycholic acid (UDCA)
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
12 Apr 2022
12 Apr 2022
Historique:
received:
16
03
2022
revised:
11
04
2022
accepted:
12
04
2022
entrez:
23
4
2022
pubmed:
24
4
2022
medline:
27
4
2022
Statut:
epublish
Résumé
Our groups previously reported that conjugation at 3'-end with ursodeoxycholic acid (UDCA) significantly enhanced in vitro exon skipping properties of ASO 51 oligonucleotide targeting the human DMD exon 51. In this study, we designed a series of lipophilic conjugates of ASO 51, to explore the influence of the lipophilic moiety on exon skipping efficiency. To this end, three bile acids and two fatty acids have been derivatized and/or modified and conjugated to ASO 51 by automatized solid phase synthesis. We measured the melting temperature (
Identifiants
pubmed: 35457088
pii: ijms23084270
doi: 10.3390/ijms23084270
pmc: PMC9032562
pii:
doi:
Substances chimiques
Dystrophin
0
Oligonucleotides
0
Oligonucleotides, Antisense
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
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