Journal article
Fine tuning of phosphorothioate inclusion in 2'-O-methyl oligonucleotides contributes to specific cell targeting for splice-switching modulation
- Abstract:
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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) chemistry, a commonly used oligonucleotide modification, has been shown to increase the stability of and improve the pharmacokinetics of SSOs. However, the effect of PS inclusion in 2'-O-methyl SSOs (2OMe)...
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- Publication status:
- Published
- Peer review status:
- Peer reviewed
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(Preview, Version of record, pdf, 1.4MB, Terms of use)
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- Publisher copy:
- 10.3389/fphys.2021.689179
Authors
Bibliographic Details
- Publisher:
- Frontiers Media
- Journal:
- Frontiers in Physiology More from this journal
- Volume:
- 12
- Article number:
- 689179
- Place of publication:
- Switzerland
- Publication date:
- 2021-10-13
- Acceptance date:
- 2021-09-06
- DOI:
- EISSN:
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1664-042X
- ISSN:
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1664-042X
- Pmid:
-
34721051
Item Description
- Language:
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English
- Keywords:
- Pubs id:
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1207792
- Local pid:
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pubs:1207792
- Deposit date:
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2023-09-21
Terms of use
- Copyright holder:
- Aoki et al
- Copyright date:
- 2021
- Rights statement:
- © 2021 Aoki, Rocha, Lehto, Miyatake, Johansson, Hashimoto, Nordin, Mager, Aoki, Graham, Sathyaprakash, Roberts, Wood, Behlke and Andaloussi. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
- Licence:
- CC Attribution (CC BY)
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