Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

• Published in: Yonsei medical journal Vol. 63; no. 2; pp. 105 - 113
• Main Authors: Lim, Jung Min, Kim, Hyongbum Henry
• Format: Journal Article
• Language: English
• Published: Korea (South) Yonsei University College of Medicine 01.02.2022; 연세대학교의과대학
• Subjects: Animals; CRISPR-Cas Systems - genetics; Gene Editing; Genome; Humans; Review; 의학일반; CRISPR-Associated Protein 9; Genome editing; genetic therapy; gene editing; CRISPR-Cas systems
• ISSN: 0513-5796; 1976-2437; 1976-2437
• DOI: 10.3349/ymj.2022.63.2.105

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.