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...

Full description

Bibliographic Details
Main Authors: Jaeah Kim, Babak Basiri, Chopie Hassan, Carine Punt, Erik van der Hage, Cathaline den Besten, Michael G. Bartlett
Format: Article
Language:English
Published: Elsevier 2019-09-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119301969