Broad Institute of MIT and Harvard, 7 Cambridge Center, Cambridge, MA 02142, USA.
Science. 2013 Feb 15;339(6121):819-23. doi: 10.1126/science.1231143. Epub 2013 Jan 3.
Functional elucidation of causal genetic variants and elements requires precise genome editing technologies. The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage. We engineered two different type II CRISPR/Cas systems and demonstrate that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells. Cas9 can also be converted into a nicking enzyme to facilitate homology-directed repair with minimal mutagenic activity. Lastly, multiple guide sequences can be encoded into a single CRISPR array to enable simultaneous editing of several sites within the mammalian genome, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
功能阐明因果遗传变异和元件需要精确的基因组编辑技术。已表明 II 型原核 CRISPR(成簇规律间隔短回文重复序列)/Cas 适应性免疫系统促进 RNA 引导的靶标特异性 DNA 切割。我们设计了两种不同的 II 型 CRISPR/Cas 系统,并证明 Cas9 核酸酶可以被短 RNA 引导,在人和鼠细胞的内源性基因组靶标上诱导精确切割。Cas9 也可以被转化为一个切口酶,以最小的诱变活性促进同源定向修复。最后,多个引导序列可以被编码到单个 CRISPR 阵列中,从而实现哺乳动物基因组中多个靶标同时编辑,证明了 RNA 引导的核酸酶技术具有易于编程和广泛适用性。
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