Exploring the Potential of CRISPR/Cas9 Technology in Autoimmune Disease Management
DOI:
https://doi.org/10.54097/12q0tr03Keywords:
CRISPR/Cas9, autoimmune disease, detection, treatment.Abstract
Autoimmune diseases are a group of diseases caused by the immune system mistakenly attacking its own tissues and cells. Autoimmune diseases are marked by the binding of autoantibodies to self-antigens, where the immune system mistakenly recognizes its own proteins, DNA or RNA as foreign substances and produces autoantibodies. There is a large inter-individual variation in the detection of autoimmune diseases, and in terms of treatment it is still only possible to alleviate the disease. But the development of gene editing technology, especially the CRISPR system, has made it possible to treat autoimmune diseases by gene editing using the CRISPR/Cas system. This research focuses on the application of CRISPR/Cas9 technology for the detection and treatment of autoimmune diseases. Relying on the high affinity of the dCas9 protein-sgRNA complex for specific sequences, abnormal nucleic acid sequences can be detected, and the use of the Cas12 system with exogenous ssDNA as a signal can make the results more obvious. In terms of therapy, according to previous experimental reports, the CRISPR/Cas9 system shows its potential in treating autoimmune diseases in three ways: directly knocking down the relevant disease-causing genes, modulating enhancers to activate or silence them by using CRISPR/dCas technology, and editing CAR T-cells to attack B-cells and plasma blasts by using CRISPR/dCas technology. This research will provide an in-depth discussion of these three aspects and their rationale, and make a discussion based on the current challenges of treating autoimmune diseases.
Downloads
References
Baker M, Perazella M A. NSAIDs in CKD: Are They Safe? Am J Kidney Dis, 2020, 76 (4): 546-557.
Niazi S K. Anti-Idiotypic mRNA Vaccine to Treat Autoimmune Disorders. Vaccines, 2024, 12 (1): 9.
Ding J, Frantzeskos A, Orozco G. Functional interrogation of autoimmune disease genetics using CRISPR/Cas9 technologies and massively parallel reporter assays. Seminars in Immunopathology, 2022, 44 (1): 137-147.
Uddin F, Rudin C M, Sen T. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future. Front Oncol, 2020, 10: 1387.
Chen M, Mao A, Xu M, et al. CRISPR-Cas9 for cancer therapy: Opportunities and challenges. Cancer Letters, 2019, 447: 48-55.
Xue H Y, Ji L J, Gao A M, et al. CRISPR-Cas9 for medical genetic screens: applications and future perspectives. J Med Genet, 2016, 53 (2): 91-97.
O'Connell M R, Oakes B L, Sternberg S H, et al. Programmable RNA recognition and cleavage by CRISPR/Cas9. Nature, 2014, 516 (7530): 263-266.
Gray P E, David C. Inborn Errors of Immunity and Autoimmune Disease. J Allergy Clin Immunol Pract, 2023, 11 (6): 1602-1622.
Guk K, Keem J O, Hwang S G, et al. A facile, rapid and sensitive detection of MRSA using a CRISPR-mediated DNA FISH method, antibody-like dCas9/sgRNA complex. Biosens Bioelectron, 2017, 95: 67-71.
Li S Y, Cheng Q X, Liu J K, et al. CRISPR-Cas12a has both cis- and trans-cleavage activities on single-stranded DNA. Cell Res, 2018, 28 (4): 491-493.
Chen Y, Song J, Ruan Q, et al. Single-Cell Sequencing Methodologies: From Transcriptome to Multi-Dimensional Measurement. Small Methods, 2021, 5 (6): e2100111.
Singh M, Bindal G, Misra C S, Rath D. The era of Cas12 and Cas13 CRISPR-based disease diagnosis. Crit Rev Microbiol, 2022, 48 (6): 714-729.
Pickar-Oliver A, Gersbach C A. The next generation of CRISPR-Cas technologies and applications. Nat Rev Mol Cell Biol, 2019, 20 (8): 490-507.
Lee M H, Shin J I, Yang J W, et al. Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases: A Comprehensive Review. Int J Mol Sci, 2022, 23 (3): 1337.
Zhu Q, Wang J, Zhang L, et al. LCK rs10914542-G allele associates with type 1 diabetes in children via T cell hyporesponsiveness. Pediatr Res, 2019, 86 (3): 311-315.
Tang Y, Luo X, Cui H, et al. MicroRNA-146A contributes to abnormal activation of the type I interferon pathway in human lupus by targeting the key signaling proteins. Arthritis Rheum, 2009, 60 (4): 1065-1075.
Zhu X, Zhang Y, Yin Z, et al. Three-Dimensional Chromosomal Landscape Revealing miR-146a Dysfunctional Enhancer in Lupus and Establishing a CRISPR-Mediated Approach to Inhibit the Interferon Pathway. Arthritis Rheumatol, 2024, 76 (3): 384-395.
Mackensen A, Müller F, Mougiakakos D, et al. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nat Med, 2022, 28 (10): 2124-2132.
Downloads
Published
Issue
Section
License
Copyright (c) 2024 Highlights in Science, Engineering and Technology

This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.







