Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022:2434:33-49.
doi: 10.1007/978-1-0716-2010-6_2.

Antisense RNA Therapeutics: A Brief Overview

Virginia Arechavala-Gomeza  1   2 , Alejandro Garanto  3   4   5
Affiliations

Antisense RNA Therapeutics: A Brief Overview

Virginia Arechavala-Gomeza et al. Methods Mol Biol. 2022.

Abstract

Nucleic acid therapeutics is a growing field aiming to treat human conditions that has gained special attention due to the successful development of mRNA vaccines against SARS-CoV-2. Another type of nucleic acid therapeutics is antisense oligonucleotides, versatile tools that can be used in multiple ways to target pre-mRNA and mRNA. While some years ago these molecules were just considered a useful research tool and a curiosity in the clinical market, this has rapidly changed. These molecules are promising strategies for personalized treatments for rare genetic diseases and they are in development for very common disorders too. In this chapter, we provide a brief description of the different mechanisms of action of these RNA therapeutic molecules, with clear examples at preclinical and clinical stages.

Keywords: Antisense oligonucleotides; Clinical trials; Personalized medicine; RNA therapy; Splicing.

PubMed Disclaimer

References

    1. Arechavala-Gomeza V, Khoo B, Aartsma-Rus A (2014) Splicing modulation therapy in the treatment of genetic diseases. Appl Clin Genet 7:245–252. https://doi.org/10.2147/TACG.S71506 doi: 10.2147/TACG.S71506. - DOI - PMC - PubMed
    1. Lewandowska MA (2013) The missing puzzle piece: splicing mutations. Int J Clin Exp Pathol 6(12):2675–2682 - PMC - PubMed
    1. Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, Kinali M, Morgan JE, van Deutekom JC, Wilton SD, Dickson G, Muntoni F (2007) Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther 18(9):798–810. https://doi.org/10.1089/hum.2006.061 doi: 10.1089/hum.2006.061. - DOI - PubMed
    1. Popplewell LJ, Adkin C, Arechavala-Gomeza V, Aartsma-Rus A, de Winter CL, Wilton SD, Morgan JE, Muntoni F, Graham IR, Dickson G (2010) Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: implications for future clinical trials. Neuromuscul Disord 20(2):102–110. https://doi.org/10.1016/j.nmd.2009.10.013 doi: 10.1016/j.nmd.2009.10.013. - DOI - PubMed
    1. Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F (2009) Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol 8(10):918–928. https://doi.org/10.1016/S1474-4422(09)70211-X doi: 10.1016/S1474-4422(09)70211-X. - DOI - PMC - PubMed

Publication types