International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 15 4.3 Emphasis on potential clinical translational value Although mouse gene editing therapy is still in its early stages, it holds tremendous potential for clinical translation. Mouse gene editing technology provides the possibility of personalized treatment. Since each patient's genetic mutations may differ, treatment plans can be customized to precisely repair their genes. Mouse gene editing therapy is not only used for treatment but also helps researchers better understand the pathological processes of hereditary blindness. This aids in the development of new treatment strategies. By validating treatment methods in mouse models, researchers can lay the foundation for future clinical trials, offering more treatment options for patients with hereditary blindness. While mouse gene editing therapy still faces multiple challenges before clinical application, these treatment methods open new avenues for the treatment of hereditary blindness, providing hope for patients and the potential for clinical application in the near future. Therefore, mouse gene editing technology holds significant clinical translational potential in the treatment of hereditary blindness. 5 Ethical and Safety Considerations of Gene Editing Technologies 5.1 Discussion on ethical issues of gene editing in the medical field The development of gene editing technologies in the medical field has raised numerous ethical concerns, particularly in the context of treating hereditary blindness. When employing gene editing therapies, especially using mouse models, there may be a need to select specific genotypes to create a "corrected" model. This raises questions about whether humans should intervene in genetic diversity, especially when blindness is associated with certain genetic traits. Additionally, it raises issues of social equity, questioning whether equal treatment opportunities should be provided to all patients (Khan, 2019). In the context of gene editing in mouse models, some edits may affect germ cells, leading to the genetic transmission of the edits. This raises an ethical dilemma about whether gene editing in human germ cells is acceptable, allowing edited changes to be passed on to future generations. This also involves the uncertainty of generational effects and long-term impacts. While gene editing is primarily discussed in the context of medical applications, there are non-therapeutic uses such as enhancing cognitive abilities or appearance. This raises ethical questions about whether humans should use this technology for self-improvement rather than just treating diseases. Ethical scrutiny and regulation of gene editing are crucial. Society needs to ensure that research and treatments are conducted within appropriate ethical frameworks to prevent misuse and potential risks. This requires the establishment of clear ethical guidelines and legal regulations to oversee and manage the application of gene editing technologies. 5.2 Emphasizing the safety of treatment and potential risks Beyond discussing the ethical issues of gene editing technology, researchers must also focus on the safety and potential risks of its therapeutic applications. A successful gene editing therapy must have extremely high targeting accuracy to avoid unnecessary interference with normal genes. Inaccurate edits may result in unintended genetic variations, possibly worsening diseases. When conducting gene editing therapy, appropriate carriers are needed to deliver editing tools, such as CRISPR/Cas9. This may lead to cell and tissue toxicity reactions, posing potential risks to the patient's physiological health. The patient's immune system may react to exogenous editing tools, potentially reducing the effectiveness of the treatment. Additionally, this may lead to the patient's immune system attacking edited cells, yielding undesired outcomes. Gene editing therapy may produce uncertain long-term effects that may not immediately manifest. Long-term monitoring and assessment are critical factors to ensure treatment safety and avoid unknown risks. If gene editing therapy results in genetic changes in patients, ethical and legal responsibility issues may arise. Clear definitions of responsibility are necessary to resolve disputes in uncertain situations. In summary, the application of gene editing technology in treating hereditary blindness presents numerous ethical issues and potential risks. A comprehensive ethical framework and regulatory measures must be taken to ensure
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