2′-Ribose-modified nucleotides: A strategy for reducing off-target effects of oligonucleotide drugs

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Honglei Zhang, Yangjian Liu, Jingxuan Ma, Huajie Huang, Wannian Zhang, Donghui Xiu, Teng Ma, Man Zhang, Fei Yu, Gengshen Song
DOI: 10.1016/j.omtn.2026.102835
Cited by: 1
References: 59

Abstract

silencing activity against proprotein convertase subtilisin/kexin type 9 (PCSK9) gene was significantly enhanced and the off-target effects of specific genes could be effectively mitigated. These data prove that 2'-ribose-modified nucleotides can be effectively utilized in preclinical and clinical disease model studies for gene silencing and reducing off-target effects.

Fields of Study & Subjects

DNA and Nucleic Acid Chemistry PARP inhibition in cancer therapy RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
1
Pages
102835–102835
Publication date
15 January 2026
Language
EN