In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization

With many safety and technical limitations partly mitigated through chemical modifications, antisense oligonucleotides (ASOs) are gaining recognition as therapeutic entities. The increase in potency realized by ‘third generation chemistries’ may, however, simultaneously increase affinity to unintend...

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Bibliografski detalji
Izdano u:Nucleic Acids Res
Glavni autori: Kamola, Piotr J., Kitson, Jeremy D. A., Turner, Gemma, Maratou, Klio, Eriksson, Sofie, Panjwani, Aliza, Warnock, Linda C., Douillard Guilloux, Gaelle A., Moores, Kitty, Koppe, Emma L., Wixted, William E., Wilson, Paul A., Gooderham, Nigel J., Gant, Timothy W., Clark, Kenneth L., Hughes, Stephen A., Edbrooke, Mark R., Parry, Joel D.
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2015
Teme:
Chemical Biology and Nucleic Acid Chemistry
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4605310/
https://ncbi.nlm.nih.gov/pubmed/26338776
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkv857
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