CRISPR clear? Dimeric Cas9-Fok1 nucleases improve genome-editing specificity.
CRISPR clear? Dimeric Cas9-Fok1 nucleases improve genome-editing specificity.
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DOI：
10.1016/j.gendis.2014.08.004
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发表时间：
2014-09
期刊：
影响因子：
6.8
通讯作者：
He, Tong-Chuan
中科院分区：
文献类型：
--
作者：
Zhang, Hongmei;Yan, Zhengjian;Li, Melissa;Peabody, Michael;He, Tong-Chuan
几十年来，细菌和古菌已经进化出一种适应性免疫防御系统，称为簇状规则间隔短回文重复(CRISPR)/CRISPR相关(CAS)系统，以降解外来核酸。最近，这些来自CRISPR/Cas系统的RNA引导的Cas9核酸酶在改变我们编辑哺乳动物基因组的能力方面显示出了希望。1虽然锌指核酸酶(ZFN)和转录激活物样效应核酸酶(TALEN)已经显示出类似的前景，但通过产生独特的序列定向核酸酶来指导定点修饰，容易产生靶向RNA，这使得CRISPR/Cas9系统成为一种有吸引力的基因组编辑方法。短引导RNA(SgRNA)可以将Cas9导向特定的基因组序列，在那里它诱导双链断裂，当修复不完美时，产生突变。1Cas9还可通过同源重组催化基因置换。1毫无疑问，Cas9介导的基因组编辑和调控应该对基础科学、基因组工程和治疗学具有变革的潜力。1
Bacteria and archaea have been known for decades having evolved adaptive immune defenses called clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems to degrade foreign nucleic acids. Recently, these RNA-guided Cas9 nucleases derived from CRISPR/Cas systems have shown promise in transforming our ability to edit mammalian genomes. 1 While zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) have shown similar promise, the ease of producing targeting RNAs over the generation of unique sequence-directed nucleases to guide site-specific modifications makes the CRISPR/Cas9 system an appealing method for genome editing. A short guide RNA (sgRNA) can direct Cas9 to a specific genomic sequence where it induces doublestrand breaks that, when imperfectly repaired, yield mutations. 1 Cas9 can also catalyze gene replacement through homologous recombination. 1 Undoubtedly, Cas9-mediated genome editing and regulation should have transformative potential for basic science, genome engineering and therapeutics. 1