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基因编辑技术 (genome editing) 可以精确地破坏、插入或替换基因组中特定位点的 DNA 序列, 在基因功能的研究和遗传疾病的治疗中发挥着巨大的作用。人工核酸内切酶 (engineered endonuclease, EEN) 介导的基因编辑技术极大地改善了早期基于同源重组的基因打靶技术 (gene targeting) 效率低的问题, 使得科研工作者可以高效地...
目前CRISPR/Cas9 Gene Knock in系统可用于目的基因引入点突变，从而模拟人类遗传疾病模型；或者将报告基因(如EGFP，mCherry，BFP等)通过同源重组的方式引入目的基因的特定位点，从而可以通过报告基因的表达跟踪目标基因的表达；亦或将功能缺失的DNA片段修复为有功能的DNA片段，即可实现基因治疗的目的。此系统的应用范围不胜...
2、Gillmore, J. D. et al. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med, doi:10.1056/NEJMoa2107454 (2021).3、Ledford, H. Landmark CRISPR trial shows promise against deadly disease. Nature, doi:10.1038/d41586-021-01776-4 (2021).4、Marcoux, J. et ...
1【题目】基因编辑(gene editing)技术是一项对基因组进行定点修饰的新技术。CRISPR/Cas9基因编辑技术被《Nature Methods》评为在过去十年中对生物学研究影响最深的十大技术之一、它类似于WORD文字编辑,可以精确地定位到目标基因组的某一位点上,可以修复一些突变的基因,或者敲除、插入目的基因。请回答下列与基因编辑技术...
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