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Use of Customizable Nucleases for Gene Editing and Other Novel Applications

机译:使用可定制的核酸酶进行基因编辑和其他新型应用

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摘要

The development of novel genome editing tools has unlocked new opportunities that were not previously possible in basic and biomedical research. During the last two decades, several new genome editing methods have been developed that can be customized to modify specific regions of the genome. However, in the past couple of years, many newer and more exciting genome editing techniques have been developed that are more efficient, precise, and easier to use. These genome editing tools have helped to improve our understanding of genetic disorders by modeling them in cells and animal models, in addition to correcting the disease-causing mutations. Among the genome editing tools, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has proven to be the most popular one due to its versatility and has been successfully used in a wide variety of laboratory animal models and plants. In this review, we summarize the customizable nucleases currently used for genome editing and their uses beyond the modification of genome. We also discuss the potential future applications of gene editing tools for both basic research and clinical purposes.
机译:新型基因组编辑工具的开发已解锁以前在基本和生物医学研究中不能进行的新机会。在过去二十年中,已经开发了几种新的基因组编辑方法,可以定制以修改基因组的特定区域。然而,在过去几年中,已经开发了许多更新和更令人兴奋的基因组编辑技术,这些技术更有效,精确,更容易使用。这些基因组编辑工具通过在细胞和动物模型中建模,帮助改善我们对遗传疾病的理解,除了纠正引起疾病的突变之外。在基因组编辑工具中,由于其多功能性,集群经常间隙的短语重复(CASPR)/ CRISPR相关(CAS)系统已被证明是最受欢迎的,并且已成功用于各种实验室动物模型和植物。在本次审查中,我们总结了目前用于基因组编辑的可定制核酸酶及其超出基因组修改的用途。我们还讨论了基因编辑工具对基本研究和临床目的的潜在未来应用。

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