Development and Application of CRISPR Technology in the Treatment of Autoimmune Diseases

Authors

  • Xiang Zhang

DOI:

https://doi.org/10.61173/t6mbry57

Keywords:

CRISPR, Cas9, autoimmune diseases, systemic lupus erythematosus, rheumatoid arthritis

Abstract

Autoimmune diseases (ADs) result from the immune system’s misidentification of self-antigens, leading to harmful responses and pathological states. The CRISPR/Cas9 system, a groundbreaking gene-editing technology, offers promising avenues for addressing ADs. Drawing inspiration from prokaryotic immune mechanisms, CRISPR/Cas9 precisely targets and edits foreign DNA, providing unprecedented genome-editing capabilities. Manipulating immune cell genetics or regulating relevant protein expression with CRISPR/Cas9 shows significant therapeutic potential in managing ADs. Recent research and clinical studies emphasize the valuable role of CRISPR/Cas9 in understanding the underlying pathogenic mechanisms of ADs, especially concerning non-viral triggers. Through strategic and targeted approaches, substantial progress is anticipated in comprehending and managing ADs. CRISPR/Cas9 emerges as a safe and efficient tool for tackling a wide range of ADs with diverse etiologies, offering transformative advancements in treatment strategies. This technology holds immense promise in reshaping the landscape of autoimmune disease management, providing hope for improved outcomes and enhanced quality of life for affected individuals.

References

[1] Rodríguez‑Rodríguez, D.R., Ramírez‑Solís, R., et al. Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review). International Journal of Molecular Medicine, 2019, 43, 1559-1574.

[2] Mohammadzadeh I, Qujeq D, Yousefi T, et al. CRISPR/Cas9 gene editing: A new therapeutic approach in the treatment of infection and autoimmunity. IUBMB Life. 2020; 72: 1603–1621.

[3] Martin Jinek et al., A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity.Science. 2012, 337,816-821.

[4] Salas-Mckee, J., Kong, W., Gladney, W. L., Jadlowsky, J. K., Plesa, G., Davis, M. M., & Fraietta, J. A. CRISPR/Cas9-based genome editing in the era of CAR T cell immunotherapy. Human Vaccines & Immunotherapeutics, 2019, 15(5), 1126–1132.

[5] Qi L S, Larson M H, Gilbert L A, et al. Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression. Cell, 2013, 152(5): 1173-1183.

[6] Yu Zhang et al.,Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.Sci. Adv. 2020, 6,eaay6812.

[7] Wu, J., Yang, S., Yu, D. et al. CRISPR/cas9 mediated knockout of an intergenic variant rs6927172 identified IL-20RA as a new risk gene for multiple autoimmune diseases. Genes Immun 2019, 20, 103–111.

[8] Yang, J., McGovern, A., Martin, P. et al. Analysis of chromatin organization and gene expression in T cells identifies functional genes for rheumatoid arthritis. Nat Commun. 2020, 11, 4402.

[9] Echuan Tan et al. Dual-responsive nanocarriers for efficient cytosolic protein delivery and CRISPR-Cas9 gene therapy of inflammatory skin disorders. Sci. Adv. 2024, 10,eadl4336.

[10] Fan, Y., Qin, G., He, K., Gong, Y., Li, W., & Wu, G. Expression of immune-related genes in rheumatoid arthritis and a two-sample Mendelian randomization study of immune cells. Chinese Journal of Tissue Engineering, 2024, 28(27), 4312- 4318.

[11] Mateu-Arrom, L., & Puig, L. Genetic and epigenetic mechanisms of psoriasis. Genes, 2023, 14(8), 1619.

[12] Hou, G., Zhou, T., Xu, N., et al. Integrative Functional Genomics Identifies Systemic Lupus Erythematosus Causal Genetic Variant in the IRF5. 2022

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Published

2024-06-06