可编程RNA基编辑与可光激活的CRISPR-Cas13
Jeonghye Yu1, Jongpil Shin1, Jihwan Yu1
1Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea.
Nature communications
|January 22, 2024
概括
研究人员开发了一种可感应光的CRISPR-Cas13系统 (paCas13) 用于RNA操纵. 该系统能够精确控制细胞和动物模型中的RNA降解和基编辑,提供新的治疗途径.
科学领域:
- 分子生物学分子生物学
- 基因编辑技术的技术
- 生物技术是生物技术.
背景情况:
- CRISPR-Cas13系统是用于RNA操纵的多功能工具,包括干扰,成像和编辑.
- 现有的CRISPR-Cas13系统缺乏对其活动的精确时间和空间控制.
- 诱导系统的开发对于精确的生物研究和治疗应用至关重要.
研究的目的:
- 开发一种可感应光的CRISPR-Cas13系统,用于对RNA调制的时空控制.
- 为可逆RNA编辑创建一个光感应基编辑器.
- 为了证明这些新型RNA调节系统的体外和体内适用性.
主要方法:
- 开发与Magnet融合的分裂-Cas13系统 (paCas13),确定最佳的N351/C350分裂地点.
- 将ADAR2与催化不活的paCas13片段融合,以创建一个可诱导光的基编辑器 (padCas13).
- 在哺乳动物细胞和小鼠模型中测试paCas13和padCas13系统的RNA扰动,基编辑 (A-to-I,C-to-U) 和转录调制.
主要成果:
- 该paCas13系统显示了高诱导性和低背景活性,用于光诱导的RNA扰动.
- 在光控制下,padCas13编辑器启用了可逆RNA基编辑 (A-to-I,C-to-U) 在体外.
- 在padCas13编辑器成功地调整了翻译后的修改,并在小鼠模型中激活了目标转录 in vivo.
结论:
- 一个新的光感应CRISPR-Cas13系统 (paCas13) 和一个基编辑器 (padCas13) 成功开发.
- 这些系统提供精确的,光线依赖的控制RNA降解和基编辑在体外和体内.
- paCas13系统在各种疾病状态和生理过程中对RNA操纵具有广泛的适用性,促进了研究和治疗开发.
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