CHAPTER 2: Mechanisms of Antisense Oligonucleotides

Zhongtian Liu, David R Corey

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Scopus citations

Abstract

Antisense oligonucleotides (ASOs) are versatile agents for controlling the translation and splicing of mRNA. ASOs have the potential to control the expression of every gene, providing a versatile platform for the development of therapeutic agents. Unlocking this potential requires understanding the mechanisms of action of ASOs. Most ASOs function by one of two mechanisms. Some recruit RNase H and promote cleavage of an RNA target. Others bind and block RNAs to prevent translation or redirect alternative splicing. Understanding the mechanism is crucial to optimization of function and the design of effective ASOs as experimental tools and therapeutics.

Original languageEnglish (US)
Title of host publicationTherapies for Retinal Degeneration
Subtitle of host publicationTargeting Common Processes
EditorsSudhir Agrawal, Michael J. Gait
PublisherRoyal Society of Chemistry
Pages22-31
Number of pages10
Edition68
DOIs
StatePublished - Jan 1 2019

Publication series

NameRSC Drug Discovery Series
Number68
Volume2019-January
ISSN (Print)2041-3203
ISSN (Electronic)2041-3211

ASJC Scopus subject areas

  • Drug Discovery

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  • Cite this

    Liu, Z., & Corey, D. R. (2019). CHAPTER 2: Mechanisms of Antisense Oligonucleotides. In S. Agrawal, & M. J. Gait (Eds.), Therapies for Retinal Degeneration: Targeting Common Processes (68 ed., pp. 22-31). (RSC Drug Discovery Series; Vol. 2019-January, No. 68). Royal Society of Chemistry. https://doi.org/10.1039/9781788015714-00022