Metabolite Profiling of the Antisense Oligonucleotide Eluforsen Using Liquid Chromatography-Mass Spectrometry

Eluforsen (previously known as QR-010) is a 33-mer 2′-O-methyl modified phosphorothioate antisense oligonucleotide targeting the F508del mutation in the gene encoding CFTR protein of cystic fibrosis patients. In this study, eluforsen was incubated with endo- and exonucleases and mouse liver homogena...

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發表在:Mol Ther Nucleic Acids
Main Authors: Kim, Jaeah, Basiri, Babak, Hassan, Chopie, Punt, Carine, van der Hage, Erik, den Besten, Cathaline, Bartlett, Michael G.
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2019
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6704339/
https://ncbi.nlm.nih.gov/pubmed/31422288
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.07.006
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