Bao Weihao, Fan Wei, Zhang Yongshuai, Lan Feng, Ma Shuhong
State Key Laboratory of Cardiovascular Disease, Key Laboratory of Pluripotent Stem Cells in Cardiac Repair and Regeneration, Fuwai Hospital, National Center for Cardiovascular Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100037, China.
Department of Cardiovascular Surgery, Metabolic Vascular Diseases Key Laboratory of Sichuan Province, Key Laboratory of Cardiovascular Remodeling and Dysfunction, The Affiliated Hospital, Southwest Medical University, Luzhou, 646000, Sichuan, P.R. China.
Stem Cell Rev Rep. 2025 Apr 7. doi: 10.1007/s12015-025-10871-2.
The CRISPR system has been widely used for human pluripotent stem cell (hPSC) disease modeling. Circular RNA can effectively reduce RNA immunogenicity and improve RNA stability, thus contributing to in vivo DNA editing. In this study, we briefly describe the process of circularizing guide RNA and CRISPR base editing elements and using them to establish stem cell disease models. Our work provides step-by-step guidance for constructing gene point editing cell lines, offering a reliable, low-immunogenic alternative for disease modeling and therapeutic research.
CRISPR系统已被广泛应用于人类多能干细胞（hPSC）疾病建模。环状RNA可以有效降低RNA免疫原性并提高RNA稳定性，从而有助于体内DNA编辑。在本研究中，我们简要描述了环状引导RNA和CRISPR碱基编辑元件的过程，并使用它们建立干细胞疾病模型。我们的工作为构建基因点编辑细胞系提供了循序渐进的指导，为疾病建模和治疗研究提供了一种可靠的、低免疫原性的替代方法。
