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
Review
. 2021 Oct 15;18(sup1):41-50.
doi: 10.1080/15476286.2021.1983288. Epub 2021 Sep 27.

Site-directed RNA editing: recent advances and open challenges

Affiliations
Review

Site-directed RNA editing: recent advances and open challenges

Hamid Mansouri Khosravi et al. RNA Biol. .

Abstract

RNA editing by cytosine and adenosine deaminases changes the identity of the edited bases. While cytosines are converted to uracils, adenines are converted to inosines. If coding regions of mRNAs are affected, the coding potential of the RNA can be changed, depending on the codon affected. The recoding potential of nucleotide deaminases has recently gained attention for their ability to correct genetic mutations by either reverting the mutation itself or by manipulating processing steps such as RNA splicing. In contrast to CRISPR-based DNA-editing approaches, RNA editing events are transient in nature, therefore reducing the risk of long-lasting inadvertent side-effects. Moreover, some RNA-based therapeutics are already FDA approved and their use in targeting multiple cells or organs to restore genetic function has already been shown. In this review, we provide an overview on the current status and technical differences of site-directed RNA-editing approaches. We also discuss advantages and challenges of individual approaches.

PubMed Disclaimer

Conflict of interest statement

No potential conflict of interest was reported by the author(s).

Figures

Figure 1.
Figure 1.
Overview on current strategies for site-directed RNA editing

Similar articles

Cited by

References

    1. Chen G, Katrekar D, Mali P.. RNA-Guided Adenosine Deaminases: advances and Challenges for Therapeutic RNA Editing. Biochemistry. 2019;58(15):1947–1957. - PubMed
    1. Rees HA, Liu DR.. Base editing: precision chemistry on the genome and transcriptome of living cells. Nat Rev Genet. 2018;19(12):770–788. - PMC - PubMed
    1. Orphanet: About rare diseases . 2020. [cited 2021 Jul] Available from: www.orpha.net.
    1. Ginn SL, Alexander IE, Edelstein ML, et al. Gene therapy clinical trials worldwide to 2012 – an update. J Gene Med. 2013;15(2):65–77. - PubMed
    1. Treatment for genetic disorders . 2020. [cited 2021 Aug] Available from: https://www.rx-genes.com/.

Publication types

LinkOut - more resources