International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 42-47 http://medscipublisher.com/index.php/ijmms 44 1.2 Classification and characteristics of common neurological diseases Neurological diseases can be classified differently based on their characteristics. Neurodegenerative diseases are a common type of disease characterized by progressive degeneration or damage to the structure and function of the nervous system. These diseases are usually caused by genetic factors, which can lead to the death, loss of function, or degeneration of nerve cells, thereby affecting the normal functioning of the nervous system. Alzheimer's disease, Parkinson's disease, and Huntington's disease are several typical examples of neurodegenerative diseases. On the other hand, congenital neurological disorders refer to neurological abnormalities or defects that exist during embryonic development or birth. These diseases can be caused by genetic factors, environmental factors, or their interaction. Spinal meningocele, cerebellar hypoplasia, and phenylketonuria are several common examples of congenital neurological disorders (Li et al., 2022). These diseases have a significant impact on the lives and functions of patients. Neurodegenerative diseases and congenital nervous system diseases differ in their characteristics and pathogenesis, but their commonality is that they cause varying degrees of damage to the structure and function of the patient's nervous system, requiring attention to research and treatment efforts. The research and treatment of neurological diseases are of great significance. Early diagnosis and intervention are crucial for the prognosis and quality of life of patients. In recent years, with the development of gene editing technology, new opportunities and hope have been provided for the research and treatment of these diseases. Gene editing technology can help understand the pathogenesis of these diseases, develop personalized treatment plans, and provide new avenues for correcting neurological defects, such as locating and repairing mutations in related genes, or through gene therapy and other means. These advances bring hope for improving the prognosis and quality of life of patients. 2 The Application of Gene Editing Technology in the Treatment of Neurological Diseases 2.1 Application in the treatment of neurodegenerative diseases Gene editing technology has broad application prospects in the treatment of neurodegenerative diseases (Ekman et al., 2019). These technologies can be used to study the pathogenesis of diseases, develop personalized treatment strategies, and provide patients with new treatment options. Among them, CRISPR-Cas9 is a revolutionary gene editing technique that can be used to precisely modify the genome, such as knocking out or modifying specific genes. In neurodegenerative diseases, CRISPR-Cas9 can be used to evaluate gene function and repair genes with pathogenic mutations (Amin et al., 2018). Gene therapy is also a method of applying gene editing technology, which can restore the function of damaged nerve cells by introducing normal genes into the patient's cells. Gene editing technology can also be used to enhance the immune system's treatment of neurodegenerative diseases, such as by editing T cells to more effectively recognize and destroy harmful protein deposits. In addition, gene editing technology can also be used to create animal models (Figure 2), study the development process of human neurodegenerative diseases, and evaluate new treatment strategies (Ma et al., 2020). Figure 2 HD (Huntington's disease) gene edited (knocked in) pig model established using CRISPR-Cas9 technology Although gene editing technology has shown potential in the treatment of neurodegenerative diseases, challenges and ethical issues still need to be faced. Due to their safety and effectiveness, the research and application of these technologies require prospective clinical trials and regulation. In addition, it is necessary to weigh potential risks
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