Site-directed RNA editing: recent advances and open challenges

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.

Automated summary

RNA editing by cytosine and adenosine deaminases changes the identity of edited bases, potentially affecting the coding potential of RNA. Nucleotide deaminases have gained attention for their recoding potential in correcting genetic mutations. RNA editing events are transient, reducing the risk of long-lasting side effects, while some RNA-based therapeutics have been FDA approved for targeting multiple cells or organs to restore genetic function.