Why the study?
The recoding potential of nucleotide deaminases has gained attention for correcting genetic mutations, offering transient edits that reduce risks compared to CRISPR-based DNA-editing approaches.
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Design
Review
RNA editing via deaminases may enable mutation correction; leaves open therapeutic translation pending targeted trials.
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.
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Michael F. Jantsch (2021) studied this question.
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