IJMZ_2024v14n1

International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 13 The advantages of CRISPR/Cas9 lie in its simplicity and efficiency, enabling researchers to precisely edit mouse genes to simulate and study gene mutations associated with hereditary blindness. 3.2 Discussion on the methods and results of applying CRISPR/Cas9 in mouse models Significant progress has been made in applying CRISPR/Cas9 technology in mouse models, providing robust support for the study and treatment of hereditary blindness. Mice are commonly used experimental animal models due to their biological similarities to humans, making the application of CRISPR/Cas9 particularly beneficial in mice. Through CRISPR/Cas9, researchers can accurately simulate gene mutations associated with hereditary blindness in mouse models. They can select target genes, design corresponding CRISPR guide RNAs, and introduce the Cas9 enzyme into mouse embryos. This approach has been successfully used to simulate various hereditary blindness diseases, such as retinitis pigmentosa, familial cataracts, and glaucoma (Liu et al., 2023). By observing the behavior and molecular changes in mice, researchers can gain in-depth insights into the pathogenesis of these diseases. Furthermore, CRISPR/Cas9 can be used to develop new therapeutic approaches. By repairing or replacing damaged genes, scientists can attempt to correct gene mutations associated with hereditary blindness. In mouse models, these therapeutic strategies have shown potential, although further research and optimization are still required. 3.3 Discussion on the application of other gene editing technologies In addition to CRISPR/Cas9, there are other gene editing technologies that can be applied to the study of hereditary blindness in mouse models. The most notable ones include zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). These technologies are also targeted, allowing researchers to edit mouse genes. ZFNs use zinc finger proteins to guide the cutting of specific DNA sequences, while TALENs use engineered proteins to achieve the same goal. Although their principles and application methods differ from CRISPR/Cas9, they still provide effective gene editing tools. In mouse models, these technologies have been used to simulate hereditary blindness and have demonstrated similar potential to CRISPR/Cas9. The choice of these methods depends on the specific needs and experimental conditions of researchers. Sometimes, different gene editing technologies can complement each other for a more comprehensive study. It should be noted that different gene editing technologies differ in efficiency, specificity, and complexity. Therefore, when choosing the most suitable technology, researchers need to balance these factors. In the context of studying hereditary blindness, gene editing technologies such as CRISPR/Cas9, ZFNs, and TALENs provide scientists with various tools to better understand the pathogenesis of hereditary blindness and explore new treatment strategies. The application of these technologies is expected to bring more hope to blind patients. Although further research and clinical validation are still needed, they have paved the way for the future treatment of hereditary blindness. 4 Mouse Gene Editing Technology in the Treatment of Hereditary Blindness 4.1 Introduction to implemented cases of mouse gene editing therapy In the treatment of hereditary blindness, mouse models have become an ideal choice for studying gene editing technology due to their unique advantages. There have been some exciting cases of mouse gene editing therapy, bringing new hope to the treatment of hereditary blindness. One notable case is the study on Leber's hereditary optic neuropathy (LHON) (Yu et al., 2018). LHON is a genetic eye disorder associated with mitochondrial dysfunction, leading to acute vision loss. Researchers successfully

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