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

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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Bibliographic Details
Main Authors: 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: Online
Language:English
Published: Oxford University Press 2015
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4605310/

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