International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 16 that research and treatments in this field are acceptable in terms of safety, ethics, and law. Furthermore, long-term monitoring and assessment are crucial steps to ensure patient safety and treatment effectiveness. While gene editing technology offers new hope for treating hereditary blindness, exploration of its potential must be approached with extreme caution to ensure the maximum benefit for patients. 6Summary Gene editing technology has a wide range of applications in the research and treatment of hereditary blindness in mouse models. This study discusses several aspects, including the significance of mouse models, the development and application of gene editing technology, specific applications of mouse gene editing in the treatment of hereditary blindness, and ethical and safety considerations. Mouse models play an irreplaceable role in the study of hereditary blindness. Mice share a similar genome with humans, grow rapidly, and possess rich genetic tools in a controllable experimental environment. This makes them ideal research subjects, aiding scientists in understanding the mechanisms of blindness and testing therapeutic methods. Gene editing technologies, especially CRISPR/Cas9, have made significant advancements, allowing precise modification of mouse genes to simulate genetic mutations associated with hereditary blindness. This provides powerful tools for disease modeling, validation of gene therapy strategies, drug screening, and efficacy testing. Implemented cases of gene editing therapy in mice bring new hope to the treatment of hereditary blindness. For instance, in LHON research, CRISPR/Cas9 successfully repaired mutated genes in blind mice, offering prospects for clinical treatment. Additionally, gene editing technology shows significant potential in retinal cell regeneration, contributing to the management of retina-related diseases. Despite facing challenges such as ensuring safety and treatment efficacy (Van Haasteren et al., 2020), mouse gene editing therapies hold immense clinical translational potential. These treatments enable personalized therapy, deepen our understanding of hereditary blindness in research, and lay the foundation for future clinical trials. In the treatment of hereditary blindness, ethical issues encompass genetic diversity, the hereditary transmission of genetic editing, non-therapeutic applications, and ethical review and regulation. Safety and potential risks of treatment involve targeting accuracy, cell and tissue toxicity, immune reactions, post-treatment effects, and the ethical and legal responsibilities of genetic editing. This study emphasizes the importance and potential of mouse gene editing technology in the research and treatment of hereditary blindness. Mouse models provide powerful tools, and gene editing technology brings new hope for treatment. However, caution and ethical considerations must be paramount to ensure that these treatment methods are both safe and ethically acceptable. Through comprehensive ethical frameworks and regulatory measures, it is expected that gene editing technology will soon see clinical applications in the treatment of hereditary blindness, providing more hope for patients (Doudna, 2020). In conclusion, mouse gene editing technology offers new prospects for the research and treatment of hereditary blindness, demonstrating its potential clinical translational value. Research in this field will continue to deepen, overcoming challenges and driving the application of gene editing technology in the treatment of hereditary blindness. With time, we can anticipate further breakthroughs in assisting blind patients in regaining their vision through these methods. References Böhm S., Splith V., Riedmayr L.M., Rötzer R.D., Gasparoni G., Nordström K.J.V., Wagner J.E., Hinrichsmeyer K.S., Walter J., Wahl-Schott C., Fenske S., Biel M., Michalakis S., and Becirovic E., 2020, A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation, Science advances, 6(34): eaba5614. https://doi.org/10.1126/sciadv.aba5614 PMid:32875106 PMCid:PMC7438099 Cremers F.P., Boon C.J., Bujakowska K., and Zeitz C., 2018, Special issue introduction: inherited retinal disease: novel candidate genes, genotype–phenotype correlations, and inheritance models, Genes, 9(4): 215. https://doi.org/10.3390/genes9040215 PMid:29659558 PMCid:PMC5924557
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