Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting
antisense oligonucleotide
bile acid
conjugation
dystrophin
exon-skipping
solid phase synthesis
solid support
ursodeoxycholic acid
Journal
Molecules (Basel, Switzerland)
ISSN: 1420-3049
Titre abrégé: Molecules
Pays: Switzerland
ID NLM: 100964009
Informations de publication
Date de publication:
17 Dec 2021
17 Dec 2021
Historique:
received:
11
11
2021
revised:
13
12
2021
accepted:
16
12
2021
entrez:
24
12
2021
pubmed:
25
12
2021
medline:
12
2
2022
Statut:
epublish
Résumé
Steric blocking antisense oligonucleotides (ASO) are promising tools for splice modulation such as exon-skipping, although their therapeutic effect may be compromised by insufficient delivery. To address this issue, we investigated the synthesis of a 20-mer 2'-OMe PS oligonucleotide conjugated at 3'-end with ursodeoxycholic acid (UDCA) involved in the targeting of human
Identifiants
pubmed: 34946743
pii: molecules26247662
doi: 10.3390/molecules26247662
pmc: PMC8707236
pii:
doi:
Substances chimiques
Oligonucleotides, Antisense
0
RNA Precursors
0
Ursodeoxycholic Acid
724L30Y2QR
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
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