International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 14 used CRISPR/Cas9 technology to repair the mutated genes in a mouse model of LHON. This study demonstrates that gene editing technology can correct genetic eye disorders in mice, providing a promising approach for clinical treatment. Another important case involves the regeneration of retinal cells. Retinal cells are crucial cells in the visual process, and their damage can lead to blindness. Researchers used gene editing technology to activate regeneration-related genes in the mouse retina, successfully restoring the visual function of blind mice. This case provides strong support for applying gene editing technology to the treatment of hereditary blindness, especially in diseases related to the retina. 4.2 Discussion of treatment methods, results, and potential limitations Mouse gene editing therapy typically involves using CRISPR/Cas9 technology or other gene editing tools to repair or replace damaged genes in the patient's body. The application of these treatment methods in mouse models usually requires several steps. Researchers first identify the gene mutations causing blindness and then design CRISPR guide RNA to precisely locate and repair these mutations. This often involves in-depth molecular biology research and gene sequencing. Researchers introduce the CRISPR/Cas9 complex into mouse embryos to precisely cut and modify the damaged genes. This may require optimization to ensure efficient editing and minimal non-specific damage. Once the genes are edited, damaged cells can restore normal function through self-repair or regeneration. This may take time, and not all cells can successfully repair. Treatment outcomes typically require long-term tracking and evaluation in mouse models. These outcomes include the restoration of visual function (Figure 4), cellular pathological changes, and the repair of genetic mutations. Figure 4 Visual function recovery process However, mouse gene editing therapy also faces some potential limitations. Firstly, there are biological and genetic differences between mouse models and humans, so the efficacy of treatment needs validation in more suitable animal models before clinical translation. Secondly, the safety and precision of gene editing technology need further research to ensure no adverse consequences. Additionally, the cost and complexity of treatment are potential limitations that need to be addressed for widespread application.
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