RNA碱基编辑及其在疾病治疗中的研究进展
摘要
<p indent="0mm">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.