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Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

Authors
 Jung Min Lim  ;  Hyongbum Henry Kim 
Citation
 YONSEI MEDICAL JOURNAL, Vol.63(2) : 105-113, 2022-02 
Journal Title
YONSEI MEDICAL JOURNAL
ISSN
 0513-5796 
Issue Date
2022-02
MeSH
Animals ; CRISPR-Cas Systems* / genetics ; Gene Editing* ; Genome ; Humans
Keywords
CRISPR-Associated Protein 9 ; CRISPR-Cas systems ; Genome editing ; gene editing ; genetic therapy
Abstract
Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.
Files in This Item:
T202202166.pdf Download
DOI
10.3349/ymj.2022.63.2.105
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pharmacology (약리학교실) > 1. Journal Papers
Yonsei Authors
Kim, Hyongbum(김형범) ORCID logo https://orcid.org/0000-0002-4693-738X
Lim, Jung Min(임정민) ORCID logo https://orcid.org/0000-0002-0223-5933
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/189287
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