International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 9 Research Article Open Access Applications of Mouse Gene Editing Technology in the Treatment of Hereditary Blindness Zhenni Lu Hangzhou Luohuashi Biotechnology Co., Ltd, Hangzhou, 310000, China Corresponding author email: 1348509309@qq.com International Journal of Molecular Zoology, 2024, Vol.14, No.1 doi: 10.5376/ijmz.2024.14.0002 Received: 09 Nov., 2023 Accepted: 22 Dec., 2023 Published: 10 Jan., 2024 Copyright © 2024 Lu, This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Preferred citation for this article: Lu Z.N., 2024, Applications of mouse gene editing technology in the treatment of hereditary blindness, International Journal of Molecular Zoology, 14(1): 9-17 (doi: 10.5376/ijmz.2024.14.0002) Abstract This study explores the applications of mouse gene editing techniques in the research and treatment of hereditary blindness. Mouse models serve as ideal tools for biomedical research and possess significant advantages such as genetic similarity to humans, rapid growth and reproduction, abundant genetic tools, and controllable laboratory environments. Gene editing techniques, particularly CRISPR/Cas9, have made significant breakthroughs, enabling scientists to simulate genetic mutations related to inherited blindness, validate treatment strategies, conduct drug screening, and explore disease mechanisms. Implemented cases of mouse gene editing treatments, such as the restoration of Leber's hereditary optic neuropathy (LHON) and the regeneration of retinal cells, offer new hope for the treatment of hereditary blindness. While mouse gene editing treatments still face challenges, including ensuring safety and therapeutic efficacy, it holds enormous potential for clinical translation. Ethical considerations, targeting accuracy, cell toxicity, immune responses, and post-treatment effects are also crucial factors that need careful consideration. In conclusion, mouse gene editing techniques provide powerful tools for research in the treatment of hereditary blindness, offering new hope for future therapies. Keywords Mouse models; Gene editing techniques; Hereditary blindness; CRISPR/Cas9; Ethical considerations Hereditary blindness is a severe visual impairment typically caused by mutations in disease-associated genes, impacting the quality of life for millions of individuals globally. These genetic mutations can affect various aspects, including visual signal transduction, retinal structure and function, and optic nerve development, resulting in different types of hereditary blindness (Cremers et al., 2018). Patients may face the risk of blindness from birth or experience the onset of blindness in their youth or adulthood. Hereditary blindness imposes significant physical and psychological burdens on patients and their families, underscoring the crucial need for research and the development of therapeutic approaches. In recent years, the rapid advancement of gene editing technologies has provided new hope for the treatment of hereditary blindness. In this context, mouse models have become essential tools for studying the mechanisms and therapeutic strategies for hereditary blindness. Mice share a similar visual system structure and function with humans while having a shorter lifespan, facilitating experimental research (Liu et al., 2022). Through gene editing technologies, researchers can simulate pathogenic mutations causing hereditary blindness and test different treatment approaches, offering valuable insights for future therapeutic strategies. The objective of this study is to explore the application of mouse gene editing technology in the treatment of hereditary blindness. By reviewing the molecular basis of hereditary blindness, discussing the importance of mouse models, providing a detailed overview of the development and application of gene editing technology, examining the application of different gene editing techniques, and highlighting specific applications of mouse gene editing technology in the treatment of hereditary blindness (Ma and Liu, 2022). Finally, this study will pay attention to the ethical and safety considerations of gene editing technology, emphasizing the safety and potential risks of treatment. This research aims to provide readers with a comprehensive understanding of the latest developments and prospects of mouse gene editing technology in the treatment of hereditary blindness, offering robust support for future research and clinical applications.
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