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基于CRISPR/Cas9的诱导多能干细胞精确基因组编辑方案

CRISPR/Cas9-Based Protocol for Precise Genome Editing in Induced Pluripotent Stem Cells.

作者信息

Singh Avinash, Babu Swathy, Phan Marcus, Yuan Shauna H

机构信息

Department of Neurology, University of Minnesota, Twin Cities, Minneapolis, MN, USA.

Minneapolis Veterans Administration Health Care System, Minneapolis, MN, USA.

出版信息

Bio Protoc. 2024 Dec 20;14(24):e5141. doi: 10.21769/BioProtoc.5141.

Abstract

The advent of clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based genome editing has marked a significant advancement in genetic engineering technology. However, the editing of induced pluripotent stem cells (iPSCs) with CRISPR presents notable challenges in ensuring cell survival and achieving high editing efficiency. These challenges become even more complex when considering the specific target site. P53 activation as a result of traditional CRISPR editing can lead to apoptosis, potentially worsening cell health or even resulting in cell death. Mitigating this apoptotic response can enhance cell survival post-CRISPR editing, which will ultimately increase editing efficiency. In our study, we observed that combining p53 inhibition with pro-survival small molecules yields a homologous recombination rate of over 90% when using CRISPR in human iPSCs. This protocol significantly streamlines the editing process and reduces the time and resources necessary for creating isogenic lines. Key features • The combination of p53 inhibition and pro-survival small molecules promotes cell survival and increases the efficiency of genome editing. • Genome editing can be completed in as little as 8 weeks for iPSCs, significantly reducing the total time required. • Achieves a homologous recombination rate of over 90% in human iPSCs.

摘要

成簇规律间隔短回文重复序列(CRISPR)/Cas9 基因编辑技术的出现标志着基因工程技术取得了重大进展。然而,利用 CRISPR 对诱导多能干细胞(iPSC)进行编辑在确保细胞存活和实现高编辑效率方面面临显著挑战。考虑到特定靶点时,这些挑战会变得更加复杂。传统 CRISPR 编辑导致的 p53 激活会引发细胞凋亡,可能会使细胞健康状况恶化甚至导致细胞死亡。减轻这种凋亡反应可以提高 CRISPR 编辑后的细胞存活率,最终提高编辑效率。在我们的研究中,我们观察到在人类 iPSC 中使用 CRISPR 时,将 p53 抑制与促存活小分子相结合,可产生超过 90% 的同源重组率。该方案显著简化了编辑过程,减少了创建同基因系所需的时间和资源。关键特性 • p53 抑制与促存活小分子的组合可促进细胞存活并提高基因组编辑效率。 • iPSC 的基因组编辑可在短短 8 周内完成,显著缩短了所需的总时间。 • 在人类 iPSC 中实现超过 90% 的同源重组率。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5dda/11669859/8c5442dcecae/BioProtoc-14-24-5141-g001.jpg

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