International Journal of Molecular Medical Science, 2024, Vol.14, No.2, 81-89 http://medscipublisher.com/index.php/ijmms 81 Research Report Open Access The Progress and Challenges of CRISPR-Cas System from Laboratory to Clinical Practice Zhaohong Pu, Xiaoping Cai Xiongcheng Jianmin Med. Ltd., Zhuji, 311800, China Corresponding author email: 2985757244@qq.com International Journal of Molecular Medical Science, 2024, Vol.14, No.1 doi: 10.5376/ijmms.2024.14.0011 Received: 05 Mar., 2024 Accepted: 05 Apr., 2024 Published: 19 Apr., 2024 Copyright © 2024 Pu and 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: Pu Z.H., and Cai X.P., 2024, The progress and challenges of CRISPR-Cas system from laboratory to clinical practice, International Journal of Molecular Medical Science, 14(1): 81-89 (doi: 10.5376/ijmms.2024.14.0011) Abstract The CRISPR-Cas system, as an innovative and efficient gene editing technology, has provided new possibilities for genetic research and disease treatment. This technology draws inspiration from the natural defense mechanisms of microorganisms, enabling precise editing of genes by "cutting" and "pasting" DNA sequences. While it has found extensive applications in laboratory conditions and demonstrated promising results in clinical trials, its transition from the laboratory to clinical applications faces several key issues and challenges, including safety and side effects, ethical and moral considerations, as well as legal and regulatory issues. This review will focus on the progress of CRISPR-Cas system applications from the laboratory to clinical settings, particularly in the realms of gene therapy and disease research. It will extensively analyze and discuss challenges in aspects such as safety, precision, delivery efficiency, aiming to provide meaningful insights and guidance for future research and applications in this field. Keywords CRISPR-Cas system; Gene editing; Gene therapy; Safety; Ethical considerations With the rapid advancement of biotechnology, genome editing has become a prominent topic in contemporary scientific research. Among these techniques, the CRISPR-Cas system, owing to its distinctive efficiency and precision, has been widely employed in genetic editing studies. This method of altering the traits and functions of organisms through gene modification allows for a deeper understanding of the mysteries of life and, concurrently, also brings new possibilities for the treatment of diseases. However, the translation of CRISPR-Cas system from laboratory applications to clinical settings poses numerous challenges and issues in this process. The technical principles of the CRISPR-Cas system are derived from the immune systems of bacteria and archaea (Koonin and Makarova, 2019). Scientists have leveraged this principle to modify and optimize the CRISPR-Cas system, transforming it into a powerful tool for gene editing. Over the past few years, significant progress has been made in the application of the CRISPR-Cas system, not only extensively in laboratory settings but also initiating its use in clinical applications, offering new hope for the treatment of various diseases. However, as its scope of application expands, we also encounter numerous challenges, such as ensuring the precision of its editing and avoiding unnecessary side effects. The breakthrough applications of the CRISPR-Cas system in the laboratory have brought tremendous hope to the fields of gene editing and gene therapy. However, its use in clinical research still faces challenges related to safety and side effects, ethical and moral considerations, as well as legal and regulatory issues. Future directions for development include technological improvements and optimizations, interdisciplinary collaboration, knowledge sharing, and the application of the CRISPR-Cas system in other fields. By overcoming these challenges, the CRISPR-Cas system holds the potential to become a revolutionary gene therapy tool, bringing positive advancements to human health. Therefore, this review elaborates on the progress of the CRISPR-Cas system from the laboratory to clinical applications, discussing its transformative impact on gene editing, gene therapy, and disease research. Simultaneously, it delves into the current challenges and issues that need resolution, aiming to provide insights and guidance for the future applications of the CRISPR-Cas system.
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