Gene editing with ‘pencil’ rather than ‘scissors’ in human pluripotent stem cells

Stem Cell Research & Therapy

Review

Seung-Yeon Lee¹  Ju-Chan Park¹  Dayeon Kim¹  Mihn Jeong Park¹  Hyuk-Jin Cha¹  Keun-Tae Kim¹  Hyeon-Ki Jang² 

[1] College of Pharmacy, Seoul National University, 1 Gwanak-ro, Gwanak-gu, 08826, Seoul, Republic of Korea;Division of Chemical Engineering and Bioengineering, College of Art Culture and Engineering, Kangwon National University, Chuncheon, South Korea;

关键词: Human pluripotent stem cells;    Disease modeling;    Ex vivo therapy;    Isogenic pair;    Base editors;    Prime editor;    Base substitution;    Cas9;   

DOI  :  10.1186/s13287-023-03394-5

 received in 2023-02-17, accepted in 2023-06-02,  发布年份 2023

来源: Springer

【 摘 要 】

Owing to the advances in genome editing technologies, research on human pluripotent stem cells (hPSCs) have recently undergone breakthroughs that enable precise alteration of desired nucleotide bases in hPSCs for the creation of isogenic disease models or for autologous ex vivo cell therapy. As pathogenic variants largely consist of point mutations, precise substitution of mutated bases in hPSCs allows researchers study disease mechanisms with “disease-in-a-dish” and provide functionally repaired cells to patients for cell therapy. To this end, in addition to utilizing the conventional homologous directed repair system in the knock-in strategy based on endonuclease activity of Cas9 (i.e., ‘scissors’ like gene editing), diverse toolkits for editing the desirable bases (i.e., ‘pencils’ like gene editing) that avoid the accidental insertion and deletion (indel) mutations as well as large harmful deletions have been developed. In this review, we summarize the recent progress in genome editing methodologies and employment of hPSCs for future translational applications.

【 授权许可】

CC BY   
© The Author(s) 2023

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