International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 1-7 http://medscipublisher.com/index.php/ijmms 1 Research Report Open Access Applications of Gene Editing and Gene Therapy in the Treatment of Genetic Disorders Xiaoping Cai Xiongcheng Jianmin Med. Ltd., Zhuji, 311800, China Corresponding email: 2985757244@qq.com International Journal of Molecular Medical Science, 2024, Vol.14, No.1 doi: 10.5376/ijmms.2024.14.0001 Received: 15 Nov., 2023 Accepted: 20 Dec., 2023 Published: 01 Jan., 2024 Copyright © 2024 Cai, 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: Cai X.P., 2024, Applications of gene editing and gene therapy in the treatment of genetic disorders, International Journal of Molecular Medical Science, 14(1): 1-7 (doi: 10.5376/ijmms.2024.14.0001) Abstract Gene editing and gene therapy are advanced biotechnologies that have gained significant attention in recent years, holding enormous potential for the treatment of genetic disorders. This review provides an overview of the applications of gene editing and gene therapy in the treatment of genetic diseases. It introduces the concept of gene editing and places a specific emphasis on the application of the CRISPR-Cas9 system in genetic disease research and therapy. Furthermore, the review outlines the principles and methods of gene therapy, including gene replacement, gene correction, and gene silencing, discussing their applications in the treatment of genetic disorders. By comparing the advantages and limitations of both gene editing and gene therapy strategies, including technical constraints, safety concerns, and ethical considerations, this review summarizes the current applications of gene editing and gene therapy in the treatment of genetic diseases and provides insights into future trends. The aim of this review is to offer a reference and guidance for research and clinical applications of gene editing and gene therapy in the field of genetic disease treatment. Keywords Gene editing; Gene therapy; Genetic diseases; CRISPR-Cas9; Gene correction Genetic diseases are diseases caused by abnormal gene or chromosome mutations, which impose a significant burden on both patients and society as a whole. Traditional treatment methods primarily focus on alleviating disease symptoms and managing associated complications, but they rarely have the ability to cure the disease itself. However, with the rapid development of gene editing and gene therapy technologies, new treatment strategies are emerging. Gene editing technology, especially the emergence of the CRISPR-Cas9 system, provides an unprecedented option for the treatment of genetic diseases (Hu, 2022). Gene editing allows for the precise modification of abnormal genes within a patient's body, effectively correcting genetic defects and restoring normal gene function. Compared to traditional gene therapy methods, gene editing offers higher precision, a wider range of applications, and greater treatment efficacy. Furthermore, gene editing can also be used to repair a patient's hematopoietic stem cells ex vivo, offering hope incurable by traditional methods. In addition to gene editing, gene therapy is also an important direction in the field of genetic disease treatment (Yang, 2020). Gene therapy uses delivery vectors to introduce normal genes into patients' bodies to repair or replace abnormal genes. This method has made some important breakthroughs in the treatment of monogenic genetic diseases, such as the cure of some rare genetic diseases through gene replacement therapy. In addition, gene therapy can also regulate gene expression through strategies such as stem cells and gene silencing, effectively controlling the development and progression of hereditary diseases. Although the application of gene editing and gene therapy has shown great potential and hope in the treatment of genetic diseases, it also faces some challenges. Technical limitations, security issues, and ethical and legal considerations are currently key issues that need to be addressed. Therefore, further research and standardized regulation are essential. This review will systematically review the application of gene editing and gene therapy in the treatment of genetic diseases, explore the principles, methods, and key application cases of these emerging technologies, and compare
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