Repair of CRISPR-guided RNA breaks enables site-specific RNA excision in human cells

• 成果类型: Article
• 署名作者: Nemudraia, Anna; Nemudryi, Artem; Wiedenheft, Blake
• 署名单位: Montana State University System; Montana State University Bozeman
• 刊物名称: SCIENCE
• ISSN/ISSBN: 0036-12657
• DOI: 10.1126/science.adk5518
• 发表日期: 2024-05-17
• 页码: 808-814
• 关键词: messenger-rna; cleavage; endonuclease; RECOGNITION; knockdown; complex; SYSTEM; genes

摘要

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.