Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases: A Comprehensive Review

Lee, Min Ho and Shin, Jae Il and Yang, Jae Won and Lee, Keum Hwa and Cha, Do Hyeon and Hong, Jun Beom and Park, Yeoeun and Choi, Eugene and Tizaoui, Kalthoum and Koyanagi, Ai and Jacob, Louis and Park, Seoyeon and Kim, Ji Hong and Smith, Lee (2022) Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases: A Comprehensive Review. International Journal of Molecular Sciences, 23 (3). p. 1337. ISSN 1422-0067

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Official URL: https://doi.org/10.3390/ijms23031337

Abstract

Autoimmune diseases are disorders that destruct or disrupt the body’s own tissues by its own immune system. Several studies have revealed that polymorphisms of multiple genes are involved in autoimmune diseases. Meanwhile, gene therapy has become a promising approach in autoimmune diseases, and clustered regularly interspaced palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) has become one of the most prominent methods. It has been shown that CRISPR-Cas9 can be applied to knock out proprotein convertase subtilisin/kexin type 9 (PCSK9) or block PCSK9, resulting in lowering low-density lipoprotein cholesterol. In other studies, it can be used to treat rare diseases such as ornithine transcarbamylase (OTC) deficiency and hereditary tyrosinemia. However, few studies on the treatment of autoimmune disease using CRISPR-Cas9 have been reported so far. In this review, we highlight the current and potential use of CRISPR-Cas9 in the management of autoimmune diseases. We summarize the potential target genes for immunomodulation using CRISPR-Cas9 in autoimmune diseases including rheumatoid arthritis (RA), inflammatory bowel diseases (IBD), systemic lupus erythematosus (SLE), multiple sclerosis (MS), type 1 diabetes mellitus (DM), psoriasis, and type 1 coeliac disease. This article will give a new perspective on understanding the use of CRISPR-Cas9 in autoimmune diseases not only through animal models but also in human models. Emerging approaches to investigate the potential target genes for CRISPR-Cas9 treatment may be promising for the tailored immunomodulation of some autoimmune diseases in the near future.

Item Type: Journal Article
Keywords: CRISPR-Cas9, Genome Editing, autoimmune diseases
Faculty: Faculty of Science & Engineering
SWORD Depositor: Symplectic User
Depositing User: Symplectic User
Date Deposited: 04 Jan 2022 09:50
Last Modified: 16 Mar 2022 16:21
URI: https://arro.anglia.ac.uk/id/eprint/707221

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