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Biomedical Application of Gene Editing

유전자 교정 기술의 생의학적 응용

  • Ju-Chan, Park (College of Pharmacy, Seoul National University) ;
  • Hyeon-Ki, Jang (Division of Chemical Engineering and Bioengineering, College of Art Culture and Engineering)
  • Received : 2022.11.09
  • Accepted : 2022.12.14
  • Published : 2022.12.31

Abstract

The CRISPR system has revolutionized gene editing field. Cas9-mediated gene editing such as Indel induction or HDR enable targeted gene disruption or precise correction of mutation. Moreover, CRISPR-based new editing tools have been developed such as base editors. In this review, we focus on gene editing in human pluripotent stem cells, which is principal technique for gene correction therapy and disease modeling. Pluripotent stem cell-specific drug YM155 enabled selection of target gene-edited pluripotent stem cells. Also, we discussed base editing for treatment of congenital retina disease. Adenine base editor delivery as RNP form provide an approach for genetic disease treatment with safe and precise in vivo gene correction.

Keywords

Acknowledgement

이 논문은 2022년도 한국연구재단의 기초연구사업의 지원을 받아 수행된 연구임(No. NRF-2022R1C1C2011617). 본문에 삽입된 그림은 BioRender.Com을 이용하여 작성되었음.

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