International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 8-15 http://medscipublisher.com/index.php/ijmms 8 Review and Progress Open Access The Application Prospects of CRISPR-Cas9 Technology in Gene Therapys Jianer Wang Infinite (China) Co., Ltd., Zhuji, 31180, China Corresponding email: 1136425476@qq.com International Journal of Molecular Medical Science, 2024, Vol.14, No.1 doi: 10.5376/ijmms.2024.14.0002 Received: 01 Dec., 2024 Accepted: 07 Jan., 2024 Published: 19 Jan., 2024 Copyright © 2024 Wang, 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: Wang J.E., 2024, The application prospects of CRISPR-Cas9 technology in gene therapy, International Journal of Molecular Medical Science, 14(1): 8-15 (doi: 10.5376/ijmms.2024.14.0002) Abstract The CRISPR-Cas9 technology, as a revolutionary gene-editing tool, has achieved remarkable breakthroughs, significantly impacting the field of gene therapy. This review provides an overview of the fundamental principles and technological developments of CRISPR-Cas9, tracing the progress in gene therapy and extensively discussing its applications in the treatment of monogenic inherited diseases and the prospective role in cancer therapy. Additionally, the review delves into the potential applications of CRISPR-Cas9 technology in organ transplantation and regenerative medicine, elucidating the challenges and ethical considerations it faces. While summarizing the research progress of CRISPR-Cas9 technology, the review also looks ahead to the future prospects of CRISPR-Cas9 in gene therapy. This comprehensive review serves as a valuable reference for the innovation and significance of CRISPR-Cas9 technology in the field of gene therapy, providing insights to propel further development and application in this prominent area. Keywords CRISPR-Cas9 technology; Gene therapy; Genetic diseases; Cancer treatment; Organ transplantation Gene therapy, as a revolutionary medical strategy, is offering new hope for human health. With the continuous development and breakthroughs of gene editing technology, CRISPR-Cas9 technology has attracted widespread attention and research as an emerging gene editing tool (Zhang, 2021). The advent of CRISPR-Cas9 technology has brought about revolutionary changes in gene therapy. Its efficient, precise, and customizable characteristics make it an ideal gene editing tool, opening up novel prospects for gene therapy. Gene therapy aims to treat, prevent, or alleviate genetic and other diseases by directly repairing abnormal genes or inserting normal genes into the patient's body. However, gene therapy has long faced several challenges, such as the selection of effective vectors, the precision and safety of gene editing, among others. Traditional gene editing techniques, such as zinc finger nucleases and TALENs, have made significant progress, but they still have certain limitations in terms of precision, efficiency, and operability. In comparison, CRISPR-Cas9 technology has higher precision and manipulability, making gene therapy more feasible and practical. The CRISPR-Cas9 technology has opened a new era in the field of gene editing by harnessing the CRISPR RNA from the natural immune system of bacteria to guide the Cas9 nuclease in gene cutting and editing. The core of this technology lies in the design and synthesis of a short nucleotide sequence, allowing the CRISPR-Cas9 system to accurately recognize and target specific loci in the genome for editing. Compared to traditional gene editing tools, CRISPR-Cas9 technology offers higher manipulability and efficiency, enabling the editing and repair of target genes within a relatively short timeframe. This review aims to comprehensively explore the prospects of CRISPR-Cas9 technology in gene therapy. Challenges faced by CRISPR-Cas9 technology, such as issues related to technical accuracy, non-specific cleavage, and ethical concerns, are discussed. The paper summarizes current research progress and anticipates the future potential of CRISPR-Cas9 technology in gene therapy. It is believed that CRISPR-Cas9 technology will catalyze a revolution in the field of gene therapy, bringing about significant changes and hope for human health. Through this review, we hope to provide a deeper understanding of the importance and innovativeness of CRISPR-Cas9 technology in the field of gene therapy, serving as a reference to further drive the development and application of CRISPR-Cas9 technology.
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