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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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書誌詳細
出版年:Nucleic Acids Res
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2016
主題:
オンライン・アクセス: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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