Broad Institute of MIT and Harvard, 7 Cambridge Center, MA 02142, USA.
McGovern Institute for Brain Research, Department of Brain and Cognitive Sciences, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Science. 2014 Jan 3;343(6166):84-87. doi: 10.1126/science.1247005. Epub 2013 Dec 12.
The simplicity of programming the CRISPR (clustered regularly interspaced short palindromic repeats)-associated nuclease Cas9 to modify specific genomic loci suggests a new way to interrogate gene function on a genome-wide scale. We show that lentiviral delivery of a genome-scale CRISPR-Cas9 knockout (GeCKO) library targeting 18,080 genes with 64,751 unique guide sequences enables both negative and positive selection screening in human cells. First, we used the GeCKO library to identify genes essential for cell viability in cancer and pluripotent stem cells. Next, in a melanoma model, we screened for genes whose loss is involved in resistance to vemurafenib, a therapeutic RAF inhibitor. Our highest-ranking candidates include previously validated genes NF1 and MED12, as well as novel hits NF2, CUL3, TADA2B, and TADA1. We observe a high level of consistency between independent guide RNAs targeting the same gene and a high rate of hit confirmation, demonstrating the promise of genome-scale screening with Cas9.
CRISPR(成簇规律间隔短回文重复)相关核酸酶 Cas9 编程的简单性提示了一种在全基因组范围内研究基因功能的新方法。我们展示了慢病毒递送靶向 18080 个基因的全基因组 CRISPR-Cas9 敲除(GeCKO)文库,该文库包含 64751 个独特的指导序列,可在人类细胞中进行负筛选和正筛选。首先,我们使用 GeCKO 文库鉴定了对癌细胞和多能干细胞存活至关重要的基因。接下来,在黑色素瘤模型中,我们筛选了与 vemurafenib(一种治疗性 RAF 抑制剂)耐药相关的基因缺失。我们排名最高的候选基因包括先前验证的 NF1 和 MED12 基因,以及新的 NF2、CUL3、TADA2B 和 TADA1 基因。我们观察到针对同一基因的独立向导 RNA 之间具有高度一致性,并且命中确认率很高,这证明了 Cas9 进行全基因组筛选的潜力。
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