Site-directed RNA editing: recent advances and open challenges
- PMID: 34569891
- PMCID: PMC8677011
- DOI: 10.1080/15476286.2021.1983288
Site-directed RNA editing: recent advances and open challenges
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.
Conflict of interest statement
No potential conflict of interest was reported by the author(s).
Figures
References
-
- Chen G, Katrekar D, Mali P.. RNA-Guided Adenosine Deaminases: advances and Challenges for Therapeutic RNA Editing. Biochemistry. 2019;58(15):1947–1957. - PubMed
-
- Orphanet: About rare diseases . 2020. [cited 2021 Jul] Available from: www.orpha.net.
-
- 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
-
- Treatment for genetic disorders . 2020. [cited 2021 Aug] Available from: https://www.rx-genes.com/.
Publication types
MeSH terms
Grants and funding
LinkOut - more resources
Full Text Sources
Other Literature Sources