Caecilie M. M. Benckendorff

A 4′‐Thioribouridine Modification Mimics 2′‐ O ‐Methoxyethyl in ASO‐Like 20‐Mers Targeting PTEN Knockdown

Gapmer antisense oligonucleotides (ASOs) represent a clinically validated therapeutic platform, with a 2′‐ O ‐methoxyethyl modification commonplace within the flanking regions. An alternative sugar modification, incorporation of 4′‐thioribose, has demonstrated enhanced nuclease r...