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新药研发前沿动态 / 医药领域趋势进展

洪秀, 李臣诚, 徐寒梅. 优化规律间隔成簇短回文重复序列相关核酸酶9敲入效率的研究进展[J]. 药学进展, 2023, 47(12): 925-939. DOI: 10.20053/j.issn1001-5094.2023.12.006
引用本文: 洪秀, 李臣诚, 徐寒梅. 优化规律间隔成簇短回文重复序列相关核酸酶9敲入效率的研究进展[J]. 药学进展, 2023, 47(12): 925-939. DOI: 10.20053/j.issn1001-5094.2023.12.006
HONG Xiu, LI Chencheng, XU Hanmei. Research Progress in Optimizing Clustered Regularly Interspaced Short Palindromic RepeatsAssociated Nuclease 9 Knock-in Efficiency[J]. Progress in Pharmaceutical Sciences, 2023, 47(12): 925-939. DOI: 10.20053/j.issn1001-5094.2023.12.006
Citation: HONG Xiu, LI Chencheng, XU Hanmei. Research Progress in Optimizing Clustered Regularly Interspaced Short Palindromic RepeatsAssociated Nuclease 9 Knock-in Efficiency[J]. Progress in Pharmaceutical Sciences, 2023, 47(12): 925-939. DOI: 10.20053/j.issn1001-5094.2023.12.006

优化规律间隔成簇短回文重复序列相关核酸酶9敲入效率的研究进展

Research Progress in Optimizing Clustered Regularly Interspaced Short Palindromic RepeatsAssociated Nuclease 9 Knock-in Efficiency

  • 摘要: 基因编辑技术可以通过在基因组DNA中精确插入、缺失或替换而人为地修饰靶位点的遗传物质。近年来,随着规律间隔成簇短回文重复序列及其相关核酸酶9(clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease9,CRISPR/Cas9)系统的发展,研究人员对各种细胞基因组进行编辑的能力大大提高,但由于同源定向修复(homologous directedrepair,HDR)效率低下,许多通过抑制非同源末端连接(non-homologous end joining,NHEJ)或增强HDR的方法,如化学调节、同步表达和同源臂优化等被开发出来用以提高基于CRISPR/Cas9系统的精确基因敲入效率,重点介绍这些方法如何优化Cas9敲入效率的最新进展。

     

    Abstract: Gene editing technology can artificially modify genetic material at target sites by inserting, deleting or replacing them precisely in genomic DNA. The development of the clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9 (CRISPR/ Cas9) system in recent years has greatly improved researchers,s ability to edit the genomes of various cells. However, due to the low efficiency of homologous directed repair (HDR), many approaches to improve precise gene knock-in efficiency based on the CRISPR/Cas9 system have been developed by inhibiting non-homologous end joining (NHEJ) or enhancing HDR, including chemical regulation, synchronous expression, and homologous arm optimization. Here, this review focuses on recent advances in how these methods can optimize Cas9 knock-in efficiency.

     

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