Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System.
Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System.
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DOI：
10.1371/currents.hd.0242d2e7ad72225efa72f6964589369a
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发表时间：
2014-04-15
期刊：
影响因子：
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通讯作者：
Ellerby, Lisa M
中科院分区：
文献类型：
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作者：
An, Mahru C;O'Brien, Robert N;Ellerby, Lisa M
我们先前已经报道了使用传统的同源重组（HR）方法对亨廷顿病（HD）患者来源的诱导多能干细胞进行遗传校正。为了扩展这项工作，我们采用了基于CRISPR的基因组编辑方法来提高重组效率，以便在人类细胞中生成等位基因等基因HD模型。结合基于抗体的快速筛选方法来测量重组提供了一种强有力的方法来确定基因组编辑的相对效率，用于建模多聚谷氨酰胺疾病或了解调节CRISPR/Cas9 HR的因素。
We have previously reported the genetic correction of Huntington's disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombination in order to generate allelic isogenic HD models in human cells. Incorporation of a rapid antibody-based screening approach to measure recombination provides a powerful method to determine relative efficiency of genome editing for modeling polyglutamine diseases or understanding factors that modulate CRISPR/Cas9 HR.