Review and Progress

Applications of Mouse Gene Editing Technology in the Treatment of Hereditary Blindness  

Zhenni Lu
Hangzhou Luohuashi Biotechnology Co., Ltd, Hangzhou, 310000, China
Author    Correspondence author
International Journal of Molecular Zoology, 2024, Vol. 14, No. 1   doi: 10.5376/ijmz.2024.14.0002
Received: 26 Dec., 2023    Accepted: 03 Jan., 2024    Published: 17 Jan., 2024
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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, 13(1): 9-17 (doi: 10.5376/ijmz.2024.14.0002)


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.

Mouse models; Gene editing techniques; Hereditary blindness; CRISPR/Cas9; Ethical considerations
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