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Korean Society for Biochemistry and Molecular Biology (생화학분자생물학회)
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Applications of CRISPR technologies to the development of gene and cell therapy

  • Chul-Sung Park (Department of Biomedical Science, Graduate School of Biomedical Science and Engineering, Hanyang University) ;
  • Omer Habib (R&D, RedGene Inc.) ;
  • Younsu Lee (R&D, RedGene Inc.) ;
  • Junho K. Hur (Department of Biomedical Science, Graduate School of Biomedical Science and Engineering, Hanyang University)
  • Received : 2023.11.14
  • Accepted : 2023.12.26
  • Published : 2024.01.31
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Abstract

Advancements in gene and cell therapy have resulted in novel therapeutics for diseases previously considered incurable or challenging to treat. Among the various contributing technologies, genome editing stands out as one of the most crucial for the progress in gene and cell therapy. The discovery of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and the subsequent evolution of genetic engineering technology have markedly expanded the field of target-specific gene editing. Originally studied in the immune systems of bacteria and archaea, the CRISPR system has demonstrated wide applicability to effective genome editing of various biological systems including human cells. The development of CRISPR-based base editing has enabled directional cytosine-to-thymine and adenine-to-guanine substitutions of select DNA bases at the target locus. Subsequent advances in prime editing further elevated the flexibility of the edit multiple consecutive bases to desired sequences. The recent CRISPR technologies also have been actively utilized for the development of in vivo and ex vivo gene and cell therapies. We anticipate that the medical applications of CRISPR will rapidly progress to provide unprecedented possibilities to develop novel therapeutics towards various diseases.

Keywords

Acknowledgement

This research was supported by the National Research Foundation of Korea (NRF) grants funded by the Korea government (MSIT) (grant numbers: 2021M3A9I4024452, 2023R1A2C1005623, 2023R1A6C101A009, RS-2023-00261114) and the Korea Health Industry Development Institute (KHIDI) grant funded by the Ministry of Health & Welfare (grant number: HI22C0636) and to J.K.H.

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