Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy

Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy

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 influe...

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Bibliographic Details
Main Authors: Elena Marchesi, Rita Cortesi, Lorenzo Preti, Paola Rimessi, Maddalena Sguizzato, Matteo Bovolenta, Daniela Perrone
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:International Journal of Molecular Sciences
Subjects:
antisense oligonucleotide
exon skipping
conjugation
2′-<i>O</i>-methyl-phosphorothioate (2′-OMe PS)
bile acid
ursodeoxycholic acid (UDCA)
Online Access:https://www.mdpi.com/1422-0067/23/8/4270

Internet

https://www.mdpi.com/1422-0067/23/8/4270

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