研究人员展示了利用III型CRISPR复合物产生的位点特异性RNA断裂可在人类细胞中修复，这种修复可用于人类转录本中的可编程缺失，从而恢复基因功能。总之，这项工作建立了一种具有潜在治疗应用价值的精确RNA操作技术。
研究人员表示，使用CRISPR RNA引导的内切酶进行基因组编辑会产生DNA断裂，而细胞DNA修复机器可以解决这些问题。然而，操纵RNA的类似方法仍然不可用。
附：英文原文
Title: Repair of CRISPR-guided RNA breaks enables site-specific RNA excision in human cells
Author: Anna Nemudraia, Artem Nemudryi, Blake Wiedenheft
Issue&Volume: 2024-04-25
Abstract: Genome editing with CRISPR RNA-guided endonucleases generates DNA breaks that are resolved by cellular DNA repair machinery. However, analogous methods to manipulate RNA remain unavailable. Here, we show that site-specific RNA breaks generated with type III CRISPR complexes are repaired in human cells, and this repair can be used for programmable deletions in human transcripts to restore gene function. Collectively, this work establishes a technology for precise RNA manipulation with potential therapeutic applications.
DOI: adk5518
Source: https://www.science.org/doi/10.1126/science.adk5518