International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 42-47 http://medscipublisher.com/index.php/ijmms 43 forward to the application prospects of gene editing technology in the treatment of neurological diseases. Although gene editing technology has enormous potential and advantages, it also faces many challenges, such as technical limitations and safety issues. This review promotes understanding and prospects for the application of gene editing technology in the treatment of neurological diseases. We hope that through this review, we can further promote the research and clinical application of gene editing technology, and provide more effective treatment plans for patients with neurological diseases. 1 Overview of Gene Editing and Neurological Diseases 1.1 Basic principles of gene editing Gene editing technology is a technique that can directly modify the genome of organisms and is widely used in disease research and treatment. In neurological diseases, gene editing technology has enormous potential to accurately locate and repair disease-related gene mutations, thereby changing the pathogenesis and progression of the disease, and bringing new treatment options to patients. Gene editing technology is mainly based on the CRISPR-Cas9 system, which is a natural defense mechanism derived from the bacterial immune system. The CRISPR-Cas9 system is composed of Cas9 protein and a directed RNA segment (sgRNA) (Bhattacharjee, 2022). Cas9 is an endonuclease used to cleave target DNA sequences, while sgRNA is an RNA molecule used to guide Cas9 protein to the target DNA sequence. The process of gene editing includes three main steps. Design and construct sgRNAs, by calculating the complementarity between target sequences in the genome and sgRNAs, suitable sgRNAs can be designed. Cas9 mediated target DNA cleavage, Cas9 protein and sgRNA form complexes, recognize and bind to specific positions in the target DNA sequence, and cleave the target DNA, resulting in double stranded breaks (Figure 1). DNA repair and genetic modification, cells use repair pathways such as non homologous end connections (NHEJ) and homologous recombination (HDR) to repair Cas9 induced DNA breaks. The NHEJ repair process may lead to insertion or deletion mutations, while the HDR repair process provides an opportunity for precise insertion, deletion, or repair of mutations. Gene editing technology has the advantages of high efficiency, speed, and precise control of target gene modifications. It can not only be used to repair mutations, but also to knock out or insert genes, thereby changing the function and expression level of genes. Therefore, gene editing technology has been widely applied in the construction of disease models, gene function research, and treatment and prevention of diseases. Figure 1 Cas protein mediated DNA disruption and identification
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