International Journal of Molecular Medical Science, 2024, Vol.14, No.2, 81-89 http://medscipublisher.com/index.php/ijmms 83 present in human genetic diseases and investigate the impact of these mutations on the occurrence and development of diseases in model organisms. The establishment of such disease models helps researchers gain a better understanding of the pathogenic mechanisms underlying diseases and provides new perspectives and methods for the treatment and prevention of diseases. The CRISPR-Cas system can also be utilized to study the functions and regulatory mechanisms of disease-related genes. Through genome editing techniques, researchers can precisely knock out or modify genes associated with specific diseases, and then observe the impact of these genetic alterations on the occurrence and development of diseases. This research can unveil the functions and regulatory mechanisms of disease-related genes, providing new targets and strategies for the treatment and prevention of diseases. Furthermore, the CRISPR-Cas system can be employed for the screening and validation of potential therapeutic targets and drugs. Using genome editing techniques, researchers can knock out or modify genes associated with diseases and observe the impact of these genetic alterations on the therapeutic efficacy in disease models. This screening and validation process can assist researchers in identifying potential therapeutic targets and drugs, expediting the development and clinical application of new medications. 1.3 Potential applications of gene therapy Gene therapy is still in the research and development stage, with many technical and security issues still need to be addressed. Challenges related to ethics and legal aspects also confront the application of gene therapy. The CRISPR-Cas system presents new possibilities for gene therapy, but further research and exploration are needed for its practical application. The CRISPR-Cas system can be used for genome editing, precisely modifying the genome of organisms. This technology enables researchers to study the functions and regulatory mechanisms of specific genes, as well as the effects of gene mutations on organisms. Through the CRISPR-Cas system, researchers can knock out or modify specific genes and observe the impact of these genetic changes on organisms, thereby revealing the functions and regulatory mechanisms of genes. The CRISPR-Cas system can also be employed for genome-wide screening, involving the knockout or modification of a large number of genes to study their functions and interactions in organisms. This screening method helps researchers identify genes associated with specific biological processes or diseases and reveals the mechanisms of action of these genes in organisms. Furthermore, the CRISPR-Cas system can be utilized for genome regulation studies. Through the CRISPR-Cas system, researchers can precisely regulate the expression levels of genes, thereby studying gene regulatory networks and signaling pathways. This technology helps researchers understand the mechanisms of gene regulation and the relationship between abnormal gene expression and diseases. Regarding the potential applications of gene therapy, the CRISPR-Cas system offers a precise method for gene editing, providing new possibilities for gene therapy. Gene therapy is a strategy for treating diseases by repairing or replacing defective genes within a patient. The CRISPR-Cas system can be used to repair mutated genes within patients or restore normal gene function by knocking out or modifying abnormal genes. This ability for precise gene editing makes gene therapy more accurate and effective, offering new hope for the treatment of many genetic diseases. 2 Progress of CRISPR-Cas System in Clinical Research 2.1 Clinical trials in gene therapy The CRISPR-Cas system has made significant strides in clinical research, particularly in the field of gene therapy. Although still in the early stages, there are ongoing clinical trials to assess the safety and efficacy of the CRISPR-Cas system in treating human diseases. With continuous technological development and refinement, gene therapy holds the potential to become a crucial treatment method, bringing new hope to patients with various genetic diseases.
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