International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 1-7 http://medscipublisher.com/index.php/ijmms 5 challenges exist as the precise delivery of editing tools to specific tissues or organs in patients remains a challenge, necessitating effective delivery systems and strategies. In terms of ethical and moral considerations, gene editing involves the direct manipulation of human genes, sparking discussions on ethical and moral issues such as human genetic modification and genetic editing of embryos. The advantages of gene therapy include deliverability (Li et al., 2019). Gene therapy utilizes vectors (such as viruses or plasmids) to introduce normal genes into the patient's body, ensuring accurate delivery to the cells or tissues requiring treatment. With a variety of treatment strategies, gene therapy can intervene in genetic diseases by enhancing the expression of normal genes, suppressing the expression of harmful genes, or supplementing missing genes. Successful cases already exist; in treating certain genetic diseases, gene therapy has achieved significant success, such as in the treatment of some immunodeficiency disorders and retinal diseases. The limitations of gene therapy include clinical application restrictions. Currently, the clinical application of gene therapy is still subject to certain limitations, such as unstable treatment effects and the need for further validation of sustained efficacy. There are concerns about immune reactions and safety issues, the introduction of exogenous genes and vectors may trigger immune responses and safety issues, leading to treatment failure or adverse reactions. Cost is also a factor, the preparation and delivery costs of gene therapy are relatively high, limiting its feasibility for widespread application. Overall, both gene editing and gene therapy hold promise and face challenges as methods for treating genetic diseases. With continuous technological advancements and in-depth research on their safety and efficacy, these two approaches are expected to offer broader prospects for the treatment of genetic diseases in the future. 3.2 Criteria for selection and evaluation in different genetic diseases In selecting and assessing the application of gene editing and gene therapy methods for different genetic diseases, the following are common criteria. Regarding selection criteria, the first consideration is the characteristics of the disease, understanding the types of genetic mutations, the pathogenic mechanisms, and the severity of the disease. This helps determine whether gene editing or gene therapy can correct or alleviate the pathological processes caused by the genetic mutations. Additionally, considerations must include the accessibility of the target cells or tissues, determining whether gene editing or gene therapy techniques can effectively deliver to the specific cells or tissues requiring treatment. Assessing the therapeutic potential involves evaluating the efficacy and feasibility of gene editing or gene therapy methods in correcting genetic mutations, including theoretical effectiveness and pre-clinical study results. In the selection of competitive treatment methods, other possible treatments such as drug therapy or gene replacement therapy should be considered, evaluating the comparative advantages of gene editing or gene therapy against alternative approaches. In terms of evaluation criteria, the first consideration is efficacy and safety, assessing the method's impact on patients' disease symptoms, survival rates, and quality of life, as well as potential treatment risks and adverse reactions. The second consideration is persistence, examining whether the treatment effects can be sustained and whether repeated treatments are necessary. Feasibility and cost-effectiveness should also be taken into account, considering the feasibility of the treatment method, including the preparation and delivery of the treatment plan, and evaluating its cost-effectiveness compared to other treatment methods. Additionally, there is a need to assess the ethical and moral issues inherent in the treatment method (Wang et al., 2021), such as the uncertainty of potential genetic editing or modification impacts on future generations and the potential negative effects on specific ethnic or social groups. The above criteria are for reference only and are not exhaustive. In specific clinical applications, researchers and physicians need to comprehensively consider factors such as disease characteristics, treatment goals, safety, feasibility, and patient needs to make personalized choices and assessments. Additionally, ongoing clinical and scientific research is crucial for continually refining method selection and evaluation criteria.
RkJQdWJsaXNoZXIy MjQ4ODYzNQ==