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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Detalles Bibliográficos
Autores principales: Marchesi, Elena, Cortesi, Rita, Preti, Lorenzo, Rimessi, Paola, Sguizzato, Maddalena, Bovolenta, Matteo, Perrone, Daniela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9032562/
https://www.ncbi.nlm.nih.gov/pubmed/35457088
http://dx.doi.org/10.3390/ijms23084270

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9032562/
https://www.ncbi.nlm.nih.gov/pubmed/35457088
http://dx.doi.org/10.3390/ijms23084270

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