Yonsei Med J.  2022 Feb;63(2):105-113. 10.3349/ymj.2022.63.2.105.

Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

Affiliations
  • 1Department of Pharmacology, Yonsei University College of Medicine, Seoul, Korea.
  • 2Brain Korea 21 Plus Project for Medical Sciences, Yonsei University College of Medicine, Seoul, Korea.
  • 3Severance Biomedical Science Institute, Yonsei University College of Medicine, Seoul, Korea.
  • 4Institute for Immunology and Immunological Diseases, Yonsei University College of Medicine, Seoul, Korea.
  • 5Graduate Program of NanoScience and Technology, Yonsei University, Seoul, Korea.
  • 6Center for Nanomedicine, Institute for Basic Science (IBS), Seoul, Korea.
  • 7Graduate Program of Nano Biomedical Engineering (NanoBME), Advanced Science Institute, Yonsei University, Seoul, Korea.

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.

Keyword

Genome editing; gene editing; CRISPR-Cas systems; CRISPR-Associated Protein 9; genetic therapy
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