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Recent Advances in CRISPR‐Cas9 Genome Editing Technology for Biological and Biomedical Investigations

机译:CRISPR-CAS9基因组编辑技术对生物和生物医学研究的最新进展

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ABSTRACT The Type II CRISPR‐Cas9 system is a simple, efficient, and versatile tool for targeted genome editing in a wide range of organisms and cell types. It continues to gain more scientific interest and has established itself as an extremely powerful technology within our synthetic biology toolkit. It works upon a targeted site and generates a double strand breaks that become repaired by either the NHEJ or the HDR pathway, modifying or permanently replacing the genomic target sequences of interest. These can include viral targets, single‐mutation genetic diseases, and multiple‐site corrections for wide scale disease states, offering the potential to manage and cure some of mankind's most persistent biomedical menaces. Here, we present the developing progress and future potential of CRISPR‐Cas9 in biological and biomedical investigations, toward numerous therapeutic, biomedical, and biotechnological applications, as well as some of the challenges within. J. Cell. Biochem. 119: 81–94, 2018. ? 2017 Wiley Periodicals, Inc.
机译:摘要II型CRISPR-CAS9系统是一种简单,高效,且多功能的工具,可用于各种生物和细胞类型的靶向基因组编辑。它继续获得更科学的兴趣,并使自己在合成生物学工具包中成为一种极其强大的技术。它适用于目标位点,并产生由NHEJ或HDR途径修复的双链断裂,修饰或永久地替换感兴趣的基因组目标序列。这些可包括病毒靶,单突变遗传疾病和广泛疾病状态的多站点校正,提供管理和治愈某些人类最持久的生物医学威胁的可能性。在这里,我们展示了生物和生物医学调查中CRISPR-CAS9的发展进展和未来潜力,朝着许多治疗,生物医学和生物技术应用以及一些挑战。 J.Cell。生物学习。 119:81-94,2018 2017年Wiley期刊,Inc。

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