Fine tuning of phosphorothioate inclusion in 2'-O-methyl oligonucleotides contributes to specific cell targeting for splice-switching modulation

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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Bibliographic Details
Main Authors:	Aoki, Y, Rocha, CSJ, Lehto, T, Miyatake, S, Johansson, H, Hashimoto, Y, Nordin, JZ, Mager, I, Aoki, M, Graham, M, Sathyaprakash, C, Roberts, TC, Wood, MJA, Behlke, MA, Andaloussi, SE
Format:	Journal article
Language:	English
Published:	Frontiers Media 2021

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