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.

中文翻译：

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使用 CRISPR/Cas9 系统校正基因突变的好处

摘要

CRISPR/Cas9基因剪刀技术的发现是基因治疗方法发展的突破，引起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，这些分类学的发展和进展机制正在得到越来越多的研究。然而，CRISPR/Cas9技术的应用存在一些应考虑的局限性和困难。特定基因载体的创建和选择工作的开展、现代基因操作的彻底安全性评估以及来自不同科学领域的专家的综合参与对于解决与使用 CRISPR/Cas9 相关的问题至关重要。技术并达到预期的治疗效果。