Skip to Main content Skip to Navigation
Journal articles

The powerful world of antisense oligonucleotides: From bench to bedside

Abstract : Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for personalized medicine. In the last decade, major chemical developments and improvements of the backbone structure of ASOs have transformed them into true approved and commercialized drugs. ASOs target both DNA and RNA, including pre-mRNA, mRNA, and ncRDA, based on sequence complementary. They are designed to be specific for each identified molecular and genetic alteration to restore a normal, physiological situation. Thus, the characterization of the underpinning mechanisms and alterations that sustain pathology is critical for accurate ASO-design. ASOs can be used to cure both rare and common diseases, such as orphan genetic alterations and cancer. Through pioneering examples, this review shows the versatility of the mechanisms of action that provide ASOs with the potential capacity to achieve custom treatment, revolutionizing personalized medicine. This article is categorized under RNA in Disease and Development > RNA in Disease RNA Interactions with Proteins and Other Molecules > Small Molecule-RNA Interactions
Document type :
Journal articles
Complete list of metadata

Cited literature [154 references]  Display  Hide  Download
Contributor : Laurent Jonchère <>
Submitted on : Friday, May 29, 2020 - 2:50:26 PM
Last modification on : Tuesday, January 19, 2021 - 4:15:50 PM


Quemener et al-2020-The powerf...
Publication funded by an institution


Distributed under a Creative Commons Attribution 4.0 International License



Anais M. Quemener, Laura Bachelot, Anne Forestier, Emmanuelle Donnou-Fournet, David Gilot, et al.. The powerful world of antisense oligonucleotides: From bench to bedside. Wiley Interdisciplinary Reviews-RNA, 2020, 11 (5), pp.e1594. ⟨10.1002/wrna.1594⟩. ⟨hal-02565099⟩



Record views


Files downloads