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유전자 교정 기술의 생의학적 응용

Biomedical Application of Gene Editing

장현기|박주찬
산업기술연구소 (Institute of Industrial Technology)|2022.12|산업기술연구 (Journal of Industrial Technology)|제42권 제1호|pp. 29-36|DOI 10.22805/JIT.2022.42.1.029

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키워드

Gene therapyGene editingCRISPRDisease modeling
주제분류공학일반

초록

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.

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.

참고문헌 (45)

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관련 논문AI추천

CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells강기호, 김은지, 주지현대한내과학회 · The Korean Journal of Internal Medicine2017 · 제32권 제1호Evolution of CRISPR towards accurate and efficient mammal genome engineering김경미, 류석민, 허준석생화학분자생물학회 · BMB Reports2019 · 제52권 제8호Applications of CRISPR technologies to the development of gene and cell therapyChul-Sung Park, Junho K. Hur, Omer Habib, Younsu Lee생화학분자생물학회 · BMB Reports2024 · 제57권 제1호Therapeutic Applications of Genome Editing Tools for Genetic DiseasesJu-Chan Park, Sangsu Bae대한근전도전기진단의학회 · 대한근전도 전기진단의학회지2025 · 제27권 제1호Basic Principles and Clinical Applications of CRISPR-Based Genome Editing김형범, 임정민연세대학교의과대학 · Yonsei Medical Journal2022 · 제63권 제2호Recent advances in genome engineering by CRISPR technology이승환, Yeounsun Oh, Youngsik Lee생화학분자생물학회 · BMB Reports2024 · 제57권 제1호

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