Progress of research on RNA base editing toward therapeutic
CRISPR-based base editors are powerful tools for biological research and has given promise for the treatment of genetic diseases.However,undesired off-target editing is also permanent,which poses considerable risks for therapeutics.Alternatively,RNA base editing also corrects disease-causing mutations and is relatively flexible,reversible,and does not lead to potentially persistent genotoxic effects.In recent years,RNA base editing has made significant progress in correcting disease-associated point mutations.These breakthroughs have had a profound impact on the development of biotechnology,biomedical research and therapeutics.In this paper,we summarize emerging RNA editors based on A-to-I,C-to-U,pseudouridylation,etc.We provide a comprehensive overview of the design and performance of contemporary RNA base editors,while highlighting the latest breakthroughs and their application in disease-related settings.Finally,we compare different editing tools and discuss the limitations and opportunities RNA base editing for therapeutic purposes.
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维普
