IJMMS_2024v14n1

International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 42-47 http://medscipublisher.com/index.php/ijmms 46 3.2 Technical limitations and security issues Although gene editing technology has great potential in the treatment of neurological diseases, it also faces some technical limitations and safety issues. The delivery challenge is one of them, and delivering editing tools to neural cells is a challenge in gene editing technology. The current delivery methods may be limited, including low delivery efficiency and difficulty in penetrating the blood-brain barrier. Developing more effective delivery systems is crucial for achieving successful treatment of neurological diseases. The issue of cell type specificity also needs to be considered. The nervous system is very complex, including various types of cells with different functions and characteristics. Gene editing technology needs to ensure that editing is only performed on target cells to avoid unexpected effects on other cell types. Although gene editing technology may be very useful in treatment, it also has safety issues. For example, unexpected splicing or addition events may trigger gene mutations unrelated to the disease, and even lead to other health issues. Therefore, ensuring the safety of gene editing technology is very important. In addition, when using gene editing technology to treat neurological diseases, ethical and moral issues also need to be considered (Shi et al., 2021). For example, for certain genetic diseases, gene editing may be necessary in the early stages of embryonic development, which raises a series of ethical and social value related issues. 3.3 Future development direction The future development direction of gene editing technology in the treatment of neurological diseases is very broad. Improving delivery systems is a key direction, and in response to the delivery challenges of current gene editing technology, researchers will continue to improve the efficiency and penetration of delivery systems to enhance the ability of editing tools to reach nerve cells. Possible methods include nanoparticles, viral vectors, etc. Further improvement is needed in the accuracy and specificity of gene editing techniques to ensure that editing is only performed in target cells and to minimize the impact on other cells. Researchers can explore new editing tools, editing techniques, and achieve their goals by improving Cas9 or developing more precise editing tools. Combination therapy strategies are also a promising direction, such as drug therapy, photogenetics, and electrical stimulation, to enhance treatment effectiveness. By comprehensively applying multiple treatment methods, interventions can be targeted at different aspects of the disease to improve the success rate of treatment (He et al., 2021). In addition, the development of genomics is crucial for understanding neurological diseases and developing new editing targets. By studying the genomic information of patients with neurological diseases, new targets and mutations related to the disease can be discovered, and gene editing techniques can be developed and applied for repair. The development of genomics will enhance the understanding of neurological diseases and provide better basis for personalized treatment. Before further development of gene editing technology in the treatment of neurological diseases, it is necessary to establish stricter clinical trials and regulatory frameworks to ensure the safety and effectiveness of treatment, and to address ethical and legal issues. Overall, the future development directions of gene editing technology in the treatment of neurological diseases include improving delivery systems, increasing the accuracy of editing techniques, combining treatment strategies, genomics research, and strengthening clinical applications and regulation. These developments will further promote the application and effectiveness of gene editing technology in the treatment of neurological diseases. 4 Summary and Outlook Gene editing technology, as an advanced biomedical tool, has shown great potential for application in the treatment of neurological diseases. Gene editing technology has been widely applied in cutting-edge research and clinical trials of neurological diseases, achieving some encouraging results. By precisely editing and repairing mutations in relevant genes, gene editing technology provides a new therapeutic approach for the treatment of congenital neurological diseases. In addition, gene editing can also be used to inhibit the occurrence of neurological tumors and inflammatory responses (Pazzaglia and Pioli, 2019), providing new strategies for the treatment of neurodegenerative diseases.

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