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| 1 | Applications of CRISPR-Cas9 mediated genome engineering显示文摘Targeted mutagenesis based on homologous recombination has been a powerful tool for understanding the mechanisms underlying development, normal physiology, and disease. A recent breakthrough in genome engineering technology based on the class of RNA-guided endonucleases, such as clustered regularly interspaced short palindromic repeats(CRISPR)-associated Cas9, is further revolutionizing biology and medical studies. The simplicity of the CRISPR-Cas9 system has enabled its widespread applications in generating germline animal models, somatic genome engineering, and functional genomic screening and in treating genetic and infectious diseases. This technology will likely be used in all fields of biomedicine, ranging from basic research to human gene therapy. | Xiao Yang | 2015 | Journal of Medical Colleges of PLA(China)2015，30，1： | 4 |
| 2 | CRISPR/Cas9技术及其在药物研发中的应用显示文摘CRISPR/Cas是新近在细菌及古细菌中发现的一种可以降解外源核酸的获得性免疫调节系统,它由成簇规律间隔的短回文重复序列(Clustered regularly interspaced short palindromic repeats,CRISPR)和蛋白质Cas组成,其中Ⅱ型CRISPR/Cas9系统成分最简单,包括:cr RNA(CRISPR RNA)、tracr RNA(trans-activating cr RNA)、Cas9(CRISPR-associated protein),tracr RNA通过RNaseⅢ功能促进cr RNA的成熟,由cr RNA通过碱基配对识别并向导Cas9结合靶标DNA,最后Cas9蛋白通过自身内切核酸酶的活性剪切DNA,达到定点编辑DNA的作用。该技术已被广泛应用于多项科研领域,仿效CRISPR/Cas9作用机理,人工构建融合了tracr RNA和cr RNA序列的一种特殊小向导RNA(small guide RNA,sg RNA)与Cas9的复合体,即能实现定点编辑基因的效果,且操作简捷、高效。文章综述了CRISPR/Cas9技术在药物研发中的应用,如细胞株改造、基因修复、动物模型构建、药物靶点筛选、遗传疾病治疗等。 | 寇天赐 胡又佳 | 2015 | 药物生物技术2015，22，6： | 3 |
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