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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...
Autores principales: | Kim, Jaeah, Basiri, Babak, Hassan, Chopie, Punt, Carine, van der Hage, Erik, den Besten, Cathaline, Bartlett, Michael G. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6704339/ https://www.ncbi.nlm.nih.gov/pubmed/31422288 http://dx.doi.org/10.1016/j.omtn.2019.07.006 |
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