Department of Chemical, Biological, Pharmaceutical and Environmental Sciences, University of Messina, Viale Ferdinando Stagno d'Alcontres, 31, 98168 Messina, Italy.
Department of Drug and Health Sciences, University of Catania, Viale Andrea Doria 6, 95125 Catania, Italy.
Int J Mol Sci. 2022 Aug 5;23(15):8714. doi: 10.3390/ijms23158714.
Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and Huntington's disease represent some of the most prevalent neurodegenerative disorders afflicting millions of people worldwide. Unfortunately, there is a lack of efficacious treatments to cure or stop the progression of these disorders. While the causes of such a lack of therapies can be attributed to various reasons, the disappointing results of recent clinical trials suggest the need for novel and innovative approaches. Since its discovery, there has been a growing excitement around the potential for CRISPR-Cas9 mediated gene editing to identify novel mechanistic insights into disease pathogenesis and to mediate accurate gene therapy. To this end, the literature is rich with experiments aimed at generating novel models of these disorders and offering proof-of-concept studies in preclinical animal models validating the great potential and versatility of this gene-editing system. In this review, we provide an overview of how the CRISPR-Cas9 systems have been used in these neurodegenerative disorders.
阿尔茨海默病、帕金森病、肌萎缩侧索硬化症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。不幸的是，目前还没有有效的治疗方法来治愈或阻止这些疾病的进展。虽然这种缺乏治疗方法的原因可以归因于多种原因，但最近临床试验令人失望的结果表明需要新的创新方法。自发现以来，CRISPR-Cas9 介导的基因编辑在识别疾病发病机制的新机制见解和介导精确的基因治疗方面的潜力越来越令人兴奋。为此，文献中充满了旨在生成这些疾病的新型模型的实验，并在临床前动物模型中提供了概念验证研究，验证了该基因编辑系统的巨大潜力和多功能性。在这篇综述中，我们概述了 CRISPR-Cas9 系统在这些神经退行性疾病中的应用。
