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The next generation of CRISPR-Cas technologies and applications.
Nature Reviews Molecular Cell Biology ( IF 81.3 ) Pub Date : 2019-08-01 , DOI: 10.1038/s41580-019-0131-5
Adrian Pickar-Oliver 1, 2 , Charles A Gersbach 1, 2, 3
Affiliation  

The prokaryote-derived CRISPR-Cas genome editing systems have transformed our ability to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells of diverse species. The ease of use and robustness of this technology have revolutionized genome editing for research ranging from fundamental science to translational medicine. Initial successes have inspired efforts to discover new systems for targeting and manipulating nucleic acids, including those from Cas9, Cas12, Cascade and Cas13 orthologues. Genome editing by CRISPR-Cas can utilize non-homologous end joining and homology-directed repair for DNA repair, as well as single-base editing enzymes. In addition to targeting DNA, CRISPR-Cas-based RNA-targeting tools are being developed for research, medicine and diagnostics. Nuclease-inactive and RNA-targeting Cas proteins have been fused to a plethora of effector proteins to regulate gene expression, epigenetic modifications and chromatin interactions. Collectively, the new advances are considerably improving our understanding of biological processes and are propelling CRISPR-Cas-based tools towards clinical use in gene and cell therapies.

中文翻译:


下一代 CRISPR-Cas 技术和应用。



原核生物衍生的 CRISPR-Cas 基因组编辑系统改变了我们操作、检测、成像和注释不同物种活细胞中特定 DNA 和 RNA 序列的能力。这项技术的易用性和稳健性彻底改变了从基础科学到转化医学等研究领域的基因组编辑。初步的成功激发了人们努力发现用于靶向和操纵核酸的新系统,包括来自 Cas9、Cas12、Cascade 和 Cas13 直系同源物的系统。 CRISPR-Cas 的基因组编辑可以利用非同源末端连接和同源定向修复来修复 DNA,以及单碱基编辑酶。除了靶向 DNA 之外,基于 CRISPR-Cas 的 RNA 靶向工具也正在开发用于研究、医学和诊断。无核酸酶活性和 RNA 靶向 Cas 蛋白已与大量效应蛋白融合,以调节基因表达、表观遗传修饰和染色质相互作用。总的来说,这些新进展极大地提高了我们对生物过程的理解,并推动基于 CRISPR-Cas 的工具走向基因和细胞疗法的临床应用。
更新日期:2019-05-31
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