Synthesis and Exon-Skipping Properties of a 3′-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach

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′...

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Detalles Bibliográficos
Autores principales: Marchesi, Elena, Bovolenta, Matteo, Preti, Lorenzo, Capobianco, Massimo L., Mamchaoui, Kamel, Bertoldo, Monica, Perrone, Daniela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8707236/
https://www.ncbi.nlm.nih.gov/pubmed/34946743
http://dx.doi.org/10.3390/molecules26247662

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8707236/
https://www.ncbi.nlm.nih.gov/pubmed/34946743
http://dx.doi.org/10.3390/molecules26247662

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