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CRISPR/Cas9 系统在阿尔茨海默病及神经退行性疾病治疗中的应用。

Application of CRISPR/Cas9 System in the Treatment of Alzheimer's Disease and Neurodegenerative Diseases.

机构信息

Student Research Committee, Urmia University of Medical Sciences, Urmia, Iran.

Department of Clinical Biochemistry, Faculty of Medicine, Urmia University Medical Sciences (UMSU), Urmia, Iran.

出版信息

Mol Neurobiol. 2024 Nov;61(11):9416-9431. doi: 10.1007/s12035-024-04143-2. Epub 2024 Apr 19.

DOI:10.1007/s12035-024-04143-2
PMID:38639864
Abstract

Alzheimer's, Parkinson's, and Huntington's are some of the most common neurological disorders, which affect millions of people worldwide. Although there have been many treatments for these diseases, there are still no effective treatments to treat or completely stop these disorders. Perhaps the lack of proper treatment for these diseases can be related to various reasons, but the poor results related to recent clinical research also prompted doctors to look for new treatment approaches. In this regard, various researchers from all over the world have provided many new treatments, one of which is CRISPR/Cas9. Today, the CRISPR/Cas9 system is mostly used for genetic modifications in various species. In addition, by using the abilities available in the CRISPR/Cas9 system, researchers can either remove or modify DNA sequences, which in this way can establish a suitable and useful treatment method for the treatment of genetic diseases that have undergone mutations. We conducted a non-systematic review of articles and study results from various databases, including PubMed, Medline, Web of Science, and Scopus, in recent years. and have investigated new treatment methods in neurodegenerative diseases with a focus on Alzheimer's disease. Then, in the following sections, the treatment methods were classified into three groups: anti-tau, anti-amyloid, and anti-APOE regimens. Finally, we discussed various applications of the CRISPR/Cas-9 system in Alzheimer's disease. Today, using CRISPR/Cas-9 technology, scientists create Alzheimer's disease models that have a more realistic phenotype and reveal the processes of pathogenesis; following the screening of defective genes, they establish treatments for this disease.

摘要

阿尔茨海默病、帕金森病和亨廷顿病是一些最常见的神经退行性疾病,影响着全球数百万人。尽管这些疾病有许多治疗方法,但仍然没有有效的治疗方法来治疗或完全阻止这些疾病。也许这些疾病缺乏适当的治疗方法可能与各种原因有关,但最近临床研究结果不佳也促使医生寻找新的治疗方法。在这方面,来自世界各地的各种研究人员提供了许多新的治疗方法,其中之一是 CRISPR/Cas9。如今,CRISPR/Cas9 系统主要用于各种物种的基因修饰。此外,通过利用 CRISPR/Cas9 系统中提供的能力,研究人员可以删除或修改 DNA 序列,从而为治疗发生突变的遗传疾病建立合适和有用的治疗方法。我们对近年来来自各种数据库(包括 PubMed、Medline、Web of Science 和 Scopus)的文章和研究结果进行了非系统性综述,并重点研究了神经退行性疾病的新治疗方法。然后,在以下各节中,将治疗方法分为三组:抗 tau、抗淀粉样蛋白和抗 APOE 方案。最后,我们讨论了 CRISPR/Cas-9 系统在阿尔茨海默病中的各种应用。今天,科学家们使用 CRISPR/Cas-9 技术创建了具有更现实表型的阿尔茨海默病模型,并揭示了发病机制的过程;在筛选出缺陷基因后,他们为这种疾病建立了治疗方法。

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Applications of CRISPR-Cas9 in Alzheimer's Disease and Related Disorders.CRISPR-Cas9 在阿尔茨海默病及相关疾病中的应用。
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