Induced Pluripotency and Gene Editing in Disease Modelling: Perspectives and Challenges

International Journal of Molecular Sciences

Yu Fen Samantha Seah²  Chadi A. EL Farran²  Tushar Warrier²  Jian Xu³  Yuin-Han Loh²  Wenbin Deng¹ 

[1] Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, SingaporeEpigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore;Department of Biological Sciences, National University of Singapore, Singapore 117543;

关键词: iPSCs;    CRISPR;    gene editing;    disease modeling;   

DOI  :  10.3390/ijms161226119

来源: mdpi

【 摘 要 】

Embryonic stem cells (ESCs) are chiefly characterized by their ability to self-renew and to differentiate into any cell type derived from the three main germ layers. It was demonstrated that somatic cells could be reprogrammed to form induced pluripotent stem cells (iPSCs) via various strategies. Gene editing is a technique that can be used to make targeted changes in the genome, and the efficiency of this process has been significantly enhanced by recent advancements. The use of engineered endonucleases, such as homing endonucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and Cas9 of the CRISPR system, has significantly enhanced the efficiency of gene editing. The combination of somatic cell reprogramming with gene editing enables us to model human diseases in vitro, in a manner considered superior to animal disease models. In this review, we discuss the various strategies of reprogramming and gene targeting with an emphasis on the current advancements and challenges of using these techniques to model human diseases.

【 授权许可】

CC BY   
© 2015 by the authors; licensee MDPI, Basel, Switzerland.