International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 42-47 http://medscipublisher.com/index.php/ijmms 42 Review and Progress Open Access Application and Prospects of Gene Editing Technology in the Treatment of Neurological Disorders JennyWu Institute of Life Science, Jiyang College of Zhejiang A&F University, Zhuji, 311800, China Corresponding email: 2314548193@qq.com International Journal of Molecular Medical Science, 2024, Vol.14, No.1 doi: 10.5376/ijmms.2024.14.0006 Received: 16 Feb., 2024 Accepted: 18 Mar., 2024 Published: 29 Mar., 2024 Copyright © 2024 Wu, This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Preferred citation for this article: Wu J., 2024, Application and prospects of gene editing technology in the treatment of neurological disorders, International Journal of Molecular Medical Science, 14(1): 42-47 (doi: 10.5376/ijmms.2024.14.0006) Abstract Neurological diseases are a serious threat to human health, causing a huge burden on patients and society. Traditional treatment methods often cannot effectively cure neurological diseases, so it is necessary to explore new treatment strategies. The rapid development of gene editing technology has provided new ideas for the treatment of neurological diseases. This review will systematically review the application of gene editing technology in the treatment of neurological diseases. The review provides an overview of the principles and tools of gene editing technology, analyzes the characteristics of neurological diseases and the challenges of existing treatments, and explores in detail the application cases of gene editing technology in the treatment of neurological diseases such as neurodegenerative diseases and congenital neurological diseases. In addition, this review also discusses the challenges and safety considerations faced by gene editing technology, and looks forward to the future prospects and development directions. Comprehensive analysis shows that gene editing technology has enormous advantages and potential in the treatment of neurological diseases, bringing new hope to people. Keywords Gene editing; Neurological disorders; CRISPR-Cas9; Neurodegenerative diseases; Congenital neurological disorders Neurological disorders are a type of disease that seriously affects human health and quality of life, including neurodegenerative diseases and congenital neurological disorders. These diseases bring enormous psychological, economic, and social burdens to patients and their families, while also posing significant challenges to the entire healthcare system. Traditional treatment methods often only alleviate symptoms and cannot cure diseases, so there is an urgent need to find more effective treatment methods. The rise of gene editing technology has brought hope for the treatment of neurological diseases. As a powerful tool, gene editing technology has shown great potential in changing the treatment methods of neurological diseases. Gene editing technology can achieve precise disease treatment by directly modifying abnormal genes in the genome, repairing damaged genes, or enhancing gene expression. The CRISPR-Cas9 system, which has recently attracted widespread attention, has made significant breakthroughs in the field of gene therapy as an efficient, precise, and programmable gene editing tool (Bhattacharjee, 2022). By precisely modifying the genetic material within cells, gene editing technology provides a new approach to treat the underlying genetic causes of various neurological diseases. This groundbreaking technology has great potential for treating congenital and genetic neurological diseases such as Huntington's disease, amyotrophic lateral sclerosis, and certain types of epilepsy. In addition, gene editing technology can also be used to manipulate genes related to neurodegenerative diseases, neuroinflammation, and even malignant brain tumors (Gumusgoz et al., 2021). As the field continues to progress and research deepens, gene editing technology has opened up new avenues for personalized medicine in the treatment of neurological diseases, bringing new hope and better treatment outcomes to patients. This review systematically reviews the application of gene editing technology in the treatment of neurological diseases and looks forward to its prospects. The paper introduces the principle of gene editing technology, discusses in detail the application of gene editing technology in the treatment of neurodegenerative diseases, explores the potential of gene editing technology in the treatment of congenital neurological diseases, and looks
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