Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions.
Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions.
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人类多能干细胞中的基因组编辑：方法，陷阱和解决方案。
DOI：
10.1016/j.stem.2015.12.002
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发表时间：
2016-01-07
期刊：
影响因子：
23.9
通讯作者：
Cowan CA
中科院分区：
文献类型：
--
作者：
Hendriks WT;Warren CR;Cowan CA
使用定制的核酸酶可以产生具有敲除或突变等位基因的人类多能干细胞(HPSCs)。转录激活子样效应子核酸酶(TALEN)和规则间隔短回文重复序列(CRISPR)-Cas9核酸酶是编辑hPSC基因组最常用的技术。在这份议定书综述中，我们简要概述了hPSC中基因编辑背景下的定制工程核酸酶，重点是TALEN和CRISPR/CAS9的应用。我们将强调每种方法的优缺点，并讨论实验设计的理论和技术考虑。
Human pluripotent stem cells (hPSCs) with knockout or mutant alleles can be generated using custom-engineered nucleases. Transcription Activator-Like Effector Nucleases (TALENs) and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 - nucleases are the most commonly employed technologies for editing hPSC genomes. In this Protocol Review we provide a brief overview of custom-engineered nucleases in the context of gene editing in hPSCs with a focus on the application of TALENs and CRISPR/Cas9. We will highlight the advantages and disadvantages of each method and discuss theoretical and technical considerations for experimental design.