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

Authors
 Min Ho Lee  ;  Jae Il Shin  ;  Jae Won Yang  ;  Keum Hwa Lee  ;  Do Hyeon Cha  ;  Jun Beom Hong  ;  Yeoeun Park  ;  Eugene Choi  ;  Kalthoum Tizaoui  ;  Ai Koyanagi  ;  Louis Jacob  ;  Seoyeon Park  ;  Ji Hong Kim  ;  Lee Smith 
Citation
 INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, Vol.23(3) : 1337, 2022-02 
Journal Title
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
ISSN
 1661-6596 
Issue Date
2022-02
MeSH
Animals ; Autoimmune Diseases / genetics* ; CRISPR-Cas Systems / genetics* ; Gene Editing / methods ; Humans ; RNA, Guide / genetics
Keywords
CRISPR-Cas9 ; autoimmune diseases ; genome editing
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.
Files in This Item:
T202200624.pdf Download
DOI
10.3390/ijms23031337
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Ji Hong(김지홍) ORCID logo https://orcid.org/0000-0001-5352-5423
Shin, Jae Il(신재일) ORCID logo https://orcid.org/0000-0003-2326-1820
Lee, Keum Hwa(이금화) ORCID logo https://orcid.org/0000-0002-1511-9587
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/188095
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