International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 1-7 http://medscipublisher.com/index.php/ijmms 6 3.3 Complementarity in treating genetic diseases Gene editing and gene therapy complement each other in treating genetic diseases, offering mutual supplementation and synergistic effects at different levels. In terms of the scope of target diseases, gene editing and gene therapy exhibit complementarity in addressing different types of genetic mutations and disease mechanisms. Gene editing can precisely correct genetic mutations by modifying DNA sequences, particularly applicable to monogenic genetic diseases. On the other hand, gene therapy can intervene in various types of genetic diseases through methods such as delivering normal genes or suppressing the expression of harmful genes. Gene editing and gene therapy complement each other in terms of treatment timing. Gene editing typically requires intervention in the early stages of embryos or stem cells to correct genetic mutations that occur early in the patient's body. In contrast, gene therapy can be performed at any point after the patient's birth, even when the disease has already developed and symptoms have appeared. Gene editing and gene therapy possess complementary tissue and organ integration capabilities. Gene editing can achieve systemic efficacy by selectively editing problematic genes as needed. On the other hand, gene therapy can target specific tissues and organs, achieving targeted efficacy by selectively delivering corrective factors. Gene editing and gene therapy exhibit complementarity in terms of the persistence of treatment effects. Gene editing can maintain enduring genetic changes in cells and descendants, providing long-term therapeutic effects (Zhu et al., 2019). Meanwhile, gene therapy can achieve long-term therapeutic effects through sustained gene expression or maintaining stable levels of corrective factors. In summary, gene editing and gene therapy have complementary advantages in treating genetic diseases. Therefore, in specific clinical applications, a flexible combination of gene editing and gene therapy can be chosen based on factors such as the type of disease, treatment goals, and technical feasibility to maximize treatment effectiveness and provide personalized and comprehensive treatment plans for patients. 4 Summary and Outlook Gene editing and gene therapy represent cutting-edge technologies for the treatment of genetic diseases, holding revolutionary potential in genetic modification and intervention. Gene editing, through direct modification of DNA sequences, can precisely correct genetic mutations, potentially curing genetic diseases. Gene therapy, on the other hand, involves delivering normal genes into the patient's body or suppressing the expression of harmful genes to block or improve the course of the disease. Both technologies have significant and undeniable potential in the treatment of genetic diseases. In terms of clinical applications, gene editing and gene therapy have made significant strides. Clinical trials for some genetic diseases, such as cystic fibrosis and hereditary retinal disorders, have been conducted using gene editing or gene therapy, yielding promising results. These studies have laid the groundwork for further exploration of the clinical applications of gene editing and gene therapy. In the future, the application of gene editing and gene therapy in the treatment of genetic diseases will face critical challenges and directions for development. Firstly, there is a need to enhance the precision, efficiency, and safety of gene editing techniques, reducing non-specific editing and potential side effects. Simultaneously, improvements in gene delivery technologies are required to enhance the effective delivery and persistence of gene therapy. Additionally, a deeper understanding of genetic diseases, including disease mechanisms, pathogenic genes, and key factors in disease progression, is essential. This will contribute to better identifying treatment targets and selecting appropriate therapeutic strategies. In the realm of clinical trials and regulation, it is imperative to conduct more clinical trials to assess the efficacy and safety of gene editing and gene therapy. Simultaneously, the establishment of robust regulatory frameworks is essential to ensure the rationality and feasibility of treatments. Leveraging individual genomic information to achieve personalized treatment for genetic diseases is crucial. By integrating gene editing and gene therapy,
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