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

• Hauptverfasser: Jaeah Kim (Verfasst von), Babak Basiri (Verfasst von), Chopie Hassan (Verfasst von), Carine Punt (Verfasst von), Erik van der Hage (Verfasst von), Cathaline den Besten (Verfasst von), Michael G. Bartlett (Verfasst von)
• Format: Buch
• Veröffentlicht: Elsevier, 2019-09-01T00:00:00Z.
• Schlagworte: article
• Online-Zugang: Connect to this object online.

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 hom...