ArticleProceedings of the National Academy of Sciences of the United States of America2025
An ADAR2-mimic base editor for efficient C-to-U RNA editing in vivo.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.Molecular biomedicine · 2026Review
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8 authors.
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Abstract
RNA base editing has the capability to rewrite genetic codes while offering improved safety due to its reversibility and temporal-spatial tunability. The development of cytosine-to-uridine editors faces issues of DNA mutagenic activity of APOBECs and the large-size anchoring domains associated with immunogenicity due to their microbial origin. To address these issues, we took an approach by returning to the original formula of adenosine deaminase acting on RNA (ADAR), i.e., to convert ADAR2 to a cytosine deaminase acting on RNA by introducing 17 mutant substitutions derived from RESCUE-S-it is named ADAR2-mimic base editor for C-to-U RNA editing (AMBER). AMBER displays similar sequence contexts preference to that of RESCUE, and exhibits robust performance across various tested cell lines. By applying AMBER to correct several pathogenic transcript mutations, we achieved substantial editing with efficiency ranging from 8 to 38%. For example, for the
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