Advances in Clinical Research on CRISPR-Cas9 for the Treatment of Common Monogenic Diseases
DOI:
https://doi.org/10.61173/jk7w2946Keywords:
CRISPR-Cas9, monogenic diseases, gene therapy, precision medicine, cancer immunotherapy, long COVIDAbstract
CRISPR-Cas9 gene editing technology has achieved milestone breakthroughs in the treatment of monogenic hereditary diseases. We systematically review the latest clinical research progress of this technology in treating common monogenic disorders, summarizing key efficacy data, safety outcomes, and current major obstacles. On this basis, we analyze the shift in application logic when extending from monogenic diseases to specific cancers and long COVID syndrome, that is, moving from defect repair to engineering modification and immunomodulation. For cancer, we discuss the prospects of CRISPR in CAR-T enhancement and oncogene disruption, as well as challenges such as tumor heterogeneity and delivery bottlenecks. For long COVID, we explore its value as a research tool and the prospects for direct therapy. Finally, we propose strategies to address common challenges including delivery optimization, off-target detection standardization, and ethical regulation. The success of CRISPR-Cas9 in monogenic diseases represents the prelude to precision medicine, whereas conquering complex diseases will require systematic leaps in target discovery, delivery technology, and safety profiles.
References
[1] Ganger, S., Harale, G., & Majumdar, P. (2023). Clustered regularly interspaced short palindromic repeats/CRISPR- associated (CRISPR/Cas) systems: Discovery, structure, classification, and general mechanism. In CRISPR/Cas-Mediated Dean&Francis ISSN 2959-409X Genome Editing in Plants (pp. 65-97). Apple Academic Press.
[2] Li, J., Wu, S., Zhang, K., Sun, X., Lin, W., Wang, C., & Lin, S. (2024). Clustered regularly interspaced short palindromic repeat/crispr-associated protein and its utility all at sea: Status, challenges, and prospects. Microorganisms, 12(1), 118.
[3] Stranneheim, H., & Wedell, A. (2016). Exome and genome sequencing: a revolution for the discovery and diagnosis of monogenic disorders. Journal of internal medicine, 279(1), 3-15.
[4] Randolph, T. R. (2019). Hemoglobinopathies (structural defects in hemoglobin). Rodak's Hematology-E-Book: Rodak's Hematology-E-Book, 394.
[5] Janoudi, T., Jagdale, M., Wu, M., Gorlla, S., Zhang, P., Shao, Y., ... & Chang, K. H. (2025). Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies. Blood Advances, 9(4), 808-813.
[6] Tebbi, C. K. (2022). Sickle cell disease, a review. Hemato, 3(2), 341-366.
[7] Zheng, G., & Orkin, S. H. (2024). Transcriptional repressor BCL11A in erythroid cells. Transcription factors in blood cell development, 199-215.
[8] Kovacs, K. D., Ciulla, T. A., & Kiss, S. (2022). Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques. Expert Opinion on Biological Therapy, 22(9), 1193-1208.
[9] Huang, J., Li, J., Xu, X., & Li, K. (2025). Adeno-associated virus vectors in retinal gene therapy: challenges, innovations, and future directions. Biomolecules, 15(7), 940.
[10] Kingdom, R., & Wright, C. F. (2022). Incomplete penetrance and variable expressivity: from clinical studies to population cohorts. Frontiers in Genetics, 13, 920390.
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