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Improving Precise CRISPR Genome Editing by Small Molecules: Is there a Magic Potion?
Cells ( IF 5.1 ) Pub Date : 2020-05-25 , DOI: 10.3390/cells9051318
Nadja Bischoff 1 , Sandra Wimberger 2 , Marcello Maresca 2 , Cord Brakebusch 1
Affiliation  

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing has become a standard method in molecular biology, for the establishment of genetically modified cellular and animal models, for the identification and validation of drug targets in animals, and is heavily tested for use in gene therapy of humans. While the efficiency of CRISPR mediated gene targeting is much higher than of classical targeted mutagenesis, the efficiency of CRISPR genome editing to introduce defined changes into the genome is still low. Overcoming this problem will have a great impact on the use of CRISPR genome editing in academic and industrial research and the clinic. This review will present efforts to achieve this goal by small molecules, which modify the DNA repair mechanisms to facilitate the precise alteration of the genome.

中文翻译:

改进小分子对CRISPR基因组的精确编辑:有魔药吗?

簇状规则间隔的短回文重复序列(CRISPR)基因组编辑已成为分子生物学中的一种标准方法,用于建立转基因的细胞和动物模型,用于识别和验证动物中的药物靶标,并经过严格的基因测试人类的疗法。尽管CRISPR介导的基因靶向的效率远高于经典的靶向诱变,但CRISPR基因组编辑以将确定的改变引入基因组的效率仍然很低。克服这个问题将对CRISPR基因组编辑在学术和工业研究以及临床中的使用产生重大影响。这篇综述将提出通过小分子来实现这一目标的努力,这些小分子修饰了DNA修复机制以促进基因组的精确改变。
更新日期:2020-05-25
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