CRIPSR 引导的 RNA 断裂修复可实现人类细胞中特定位点的 RNA 切除。
Repair of CRISPR-guided RNA breaks enables site-specific RNA excision in human cells.
机构信息
Department of Microbiology and Cell Biology, Montana State University, Bozeman, MT 59717, USA.
出版信息
Science. 2024 May 17;384(6697):808-814. doi: 10.1126/science.adk5518. Epub 2024 Apr 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. We show that site-specific RNA breaks generated with type-III CRISPR complexes are repaired in human cells and that 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.
摘要
利用 CRISPR RNA 引导的内切酶进行基因组编辑会产生 DNA 断裂，这些断裂由细胞 DNA 修复机制来解决。然而，类似的操纵 RNA 的方法仍然不可用。我们表明，使用 III 型 CRISPR 复合物产生的特异性 RNA 断裂在人类细胞中被修复，并且这种修复可用于人类转录本中的可编程缺失，以恢复基因功能。总的来说，这项工作为精确的 RNA 操作建立了一种技术，具有潜在的治疗应用。
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