International Journal of Molecular Medical Science, 2024, Vol.14, No.2, 81-89 http://medscipublisher.com/index.php/ijmms 82 1 Progress of the CRISPR-Cas System in Basic Research 1.1 Genome editing and functional studies The CRISPR-Cas system has made significant strides in basic research, particularly in the realms of genome editing and functional studies. Its application in basic research provides an efficient and precise tool for genome editing and functional studies. Through this technology, researchers can elucidate the functions and regulatory mechanisms of genes, laying crucial foundations for the understanding of biology and the treatment of diseases. Genome editing is one of the most crucial applications of the CRISPR-Cas system. By introducing the CRISPR-Cas system, researchers can precisely edit target genes, including additions, deletions, and modifications. This precise genome editing technology provides a powerful tool for elucidating the roles of genes in organism development, physiological functions, and disease occurrence. The fundamental principle of the CRISPR-Cas system involves the pairing of guide RNA (gRNA) with the CRISPR characteristic sequences located near some CRISPR-associated genes that encode a series of Cas proteins, collectively known as the CRISPR-Cas system (Figure 1). This system directs Cas proteins to the target DNA sequence, and through the nucleolytic activity of Cas proteins, the target DNA is cleaved. During the cleavage process, the cell's self-repair mechanisms intervene, leading to genome editing. This genome editing technology can be employed to study the function of genes, such as investigating their role in organisms by knocking out specific genes (Manghwar et al., 2019). Figure 1 CRISPR-Cas system In addition to gene knockout, the CRISPR-Cas system can also achieve gene addition and modification. By introducing exogenous DNA fragments and the CRISPR-Cas system, researchers can add specific genes to the target genome, thereby enabling functional studies of genes. Furthermore, the CRISPR-Cas system can facilitate gene modification, such as studying gene function and regulatory mechanisms through point mutations or insertions. The advancement of genome editing technology provides a powerful tool for gene function studies. Through the CRISPR-Cas system, researchers can precisely edit target genes, elucidating the roles of genes in organism development, physiological functions, and disease occurrence. The application of this technology has made significant breakthroughs in various organisms, including model organisms such as mice, fruit flies, nematodes, and zebrafish. 1.2 Establishment and study of disease models In addition to its applications in genome editing and functional studies, the CRISPR-Cas system has made significant strides in the establishment and study of disease models. Through this technology, researchers can simulate mutations associated with genetic diseases and investigate the impact of these mutations on the occurrence and development of diseases. Moreover, the CRISPR-Cas system can reveal the functions and regulatory mechanisms of disease-related genes, aiding in the screening and validation of potential therapeutic targets and drugs. These advancements provide new perspectives and approaches for the treatment and prevention of diseases. The CRISPR-Cas system can be employed to establish disease models, particularly models for genetic diseases (Garcia-Robledo et al., 2020). By introducing the CRISPR-Cas system, researchers can simulate mutations
RkJQdWJsaXNoZXIy MjQ4ODYzNQ==