Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation

Splice-switching antisense oligonucleotide- (SSO-) mediated correction of framedisrupting mutation-containing premessenger RNA (mRNA) transcripts using exon skipping is a highly promising treatment method for muscular diseases such as Duchenne muscular dystrophy (DMD). Phosphorothioate (PS) chemistr...

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Detalles Bibliográficos
Autores principales: Aoki, Yoshitsugu, Rocha, Cristina S. J., Lehto, Taavi, Miyatake, Shouta, Johansson, Henrik, Hashimoto, Yasumasa, Nordin, Joel Z., Mager, Imre, Aoki, Misako, Graham, McClorey, Sathyaprakash, Chaitra, Roberts, Thomas C., Wood, Matthew J. A., Behlke, Mark A., Andaloussi, Samir El
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Physiology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8548633/
https://www.ncbi.nlm.nih.gov/pubmed/34721051
http://dx.doi.org/10.3389/fphys.2021.689179

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