CHAPTER 2: Mechanisms of Antisense Oligonucleotides

Zhongtian Liu, David R Corey

Research output: Chapter in Book/Report/Conference proceedingChapter

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

Fingerprint

Antisense Oligonucleotides
RNA Cleavage
Ribonuclease H
Alternative Splicing
Protein Biosynthesis
RNA
Gene Expression
Therapeutics

ASJC Scopus subject areas

  • Drug Discovery

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

CHAPTER 2 : Mechanisms of Antisense Oligonucleotides. / Liu, Zhongtian; Corey, David R.

Therapies for Retinal Degeneration: Targeting Common Processes. ed. / Sudhir Agrawal; Michael J. Gait. 68. ed. Royal Society of Chemistry, 2019. p. 22-31 (RSC Drug Discovery Series; Vol. 2019-January, No. 68).

Research output: Chapter in Book/Report/Conference proceedingChapter

Liu, Z & Corey, DR 2019, CHAPTER 2: Mechanisms of Antisense Oligonucleotides. in S Agrawal & MJ Gait (eds), Therapies for Retinal Degeneration: Targeting Common Processes. 68 edn, RSC Drug Discovery Series, no. 68, vol. 2019-January, Royal Society of Chemistry, pp. 22-31. https://doi.org/10.1039/9781788015714-00022
Liu Z, Corey DR. CHAPTER 2: Mechanisms of Antisense Oligonucleotides. In Agrawal S, Gait MJ, editors, Therapies for Retinal Degeneration: Targeting Common Processes. 68 ed. Royal Society of Chemistry. 2019. p. 22-31. (RSC Drug Discovery Series; 68). https://doi.org/10.1039/9781788015714-00022
Liu, Zhongtian ; Corey, David R. / CHAPTER 2 : Mechanisms of Antisense Oligonucleotides. Therapies for Retinal Degeneration: Targeting Common Processes. editor / Sudhir Agrawal ; Michael J. Gait. 68. ed. Royal Society of Chemistry, 2019. pp. 22-31 (RSC Drug Discovery Series; 68).
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