Benefits of Using the CRISPR/Cas9 System for the Correction of Genetic Mutations

IF 0.4 4区生物学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY Molecular Genetics, Microbiology and Virology Pub Date : 2023-09-01 DOI:10.3103/s0891416823030084

引用次数: 0

Abstract

The discovery of the CRISPR/Cas9 gene scissors technology was a breakthrough in the development of gene therapy methods and attracted the attention of the entire scientific community. This technology provides prospects for the treatment of many neurodegenerative diseases and cancers that were previously considered almost incurable. To date, the mechanisms of the development and progression of these groups of nosologies are becoming increasingly studied due to discoveries in the field of molecular biology and genetics. However, the application of the CRISPR/Cas9 technology has some limitations and difficulties that should be considered. The development of works on the creation and selection of specific gene carriers, a thorough safety assessment of modern genetic manipulations, and the integrated participation of specialists from different fields of science will be important to solve the problems associated with the use of the CRISPR/Cas9 technology and achieve the desired therapeutic effect.

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使用 CRISPR/Cas9 系统纠正基因突变的好处

摘要 CRISPR/Cas9 基因剪刀技术的发现是基因治疗方法发展的一个突破，吸引了整个科学界的关注。这项技术为治疗许多以前被认为几乎无法治愈的神经退行性疾病和癌症提供了前景。迄今为止，由于分子生物学和遗传学领域的发现，对这几类疾病的发生和发展机制的研究日益深入。然而，CRISPR/Cas9 技术的应用也有一些局限性和困难需要考虑。要解决与使用 CRISPR/Cas9 技术相关的问题并达到预期的治疗效果，就必须开展关于创建和选择特定基因载体的工作，对现代基因操作进行全面的安全评估，以及不同科学领域专家的共同参与。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Molecular Genetics, Microbiology and Virology BIOCHEMISTRY & MOLECULAR BIOLOGY-MICROBIOLOGY

CiteScore

0.70

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Molecular Genetics, Microbiology and Virology is a journal that covers most topical theoretical and applied problems of molecular genetics of pro- and eukaryotic organisms, molecular microbiology and molecular virology. An important part the journal assigns to investigations of the genetic apparatus of microorganisms, searching for forms of genetic exchange, genetic mapping of pathogenic causative agents, to ascertainment of the structure and functions of extrachromosomal factors of heredity and migratory genetic elements, to theoretical studies into the mechanisms of genetic regulation. The journal publishes results of research on molecular and genetic bases of an eukaryotic cell, functioning of chromosomes and chromatin, nature of genetic changes in malignization and a set of hereditary diseases. On the pages of the journal there is covered the formulation of molecular bases of virology including issues of integration of viral and cellular genomes, and issues of persistence. The journal plans to put materials on genetic engineering, envisaging synthesis and isolation of genes from natural reservoirs, creation of plasmid- and virus-based vector, production of recombinant DNA molecules, the creation of Gene Banks for Microbes, animals, and human; and also on biotechnological production of hormones, components of antiviral vaccines, diagnostic and therapeutic preparations.