Reshetnikov Vasiliy V, Chirinskaite Angelina V, Sopova Julia V, Ivanov Roman A, Leonova Elena I
Department of Biotechnology, Sirius University of Science and Technology, Sochi, Russia.
Department of Molecular Genetics, Institute of Cytology and Genetics, Novosibirsk, Russia.
Front Bioeng Biotechnol. 2022 Aug 10;10:942440. doi: 10.3389/fbioe.2022.942440. eCollection 2022.
Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base-editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments and . Additionally, to understand the therapeutic potential of these systems, the advantages and disadvantages of base-editing systems are examined.
全球数百万人患有由DNA序列中的各种突变引起的罕见遗传病。罕见遗传病的传统治疗方法往往无效，因此人们对基因编辑方法寄予厚望。基于nCas9（具有切口酶活性的Cas9）或dCas9（一种催化无活性的靶向DNA的Cas9酶）的DNA碱基编辑系统能够在不产生双链断裂的情况下进行编辑。这些工具在不断改进，这增加了它们在治疗方面的潜在用途。在这篇综述中，我们描述了碱基编辑系统的主要类型及其在实验中治疗单基因疾病的应用。此外，为了了解这些系统的治疗潜力，我们还研究了碱基编辑系统的优缺点。
