Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

High affinity antisense oligonucleotides (ASOs) containing bicylic modifications (BNA) such as locked nucleic acid (LNA) designed to induce target RNA cleavage have been shown to have enhanced potency along with a higher propensity to cause hepatotoxicity. In order to understand the mechanism of thi...

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Udgivet i:Nucleic Acids Res
Main Authors: Burel, Sebastien A., Hart, Christopher E., Cauntay, Patrick, Hsiao, Jill, Machemer, Todd, Katz, Melanie, Watt, Andy, Bui, Huynh-hoa, Younis, Husam, Sabripour, Mahyar, Freier, Susan M., Hung, Gene, Dan, Amy, Prakash, T.P., Seth, Punit P., Swayze, Eric E., Bennett, C. Frank, Crooke, Stanley T., Henry, Scott P.
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2016
Fag:
Gene regulation, Chromatin and Epigenetics
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4797265/
https://ncbi.nlm.nih.gov/pubmed/26553810
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkv1210
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