title: Induced Pluripotency and Gene Editing in Disease Modelling: Perspectives and Challenges.
abstract: 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.
authors.affiliation: Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore. yfseah@imcb.a-star.edu.sg.
authors.forename: Yu Fen Samantha
authors.initials: YF
authors.lastname: Seah
authors.affiliation: Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore. chadi@u.nus.edu.|Department of Biological Sciences, National University of Singapore, Singapore 117543. chadi@u.nus.edu.
authors.forename: Chadi A
authors.initials: CA
authors.lastname: El Farran
authors.affiliation: Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore. e0011350@u.nus.edu.|Department of Biological Sciences, National University of Singapore, Singapore 117543. e0011350@u.nus.edu.
authors.forename: Tushar
authors.initials: T
authors.lastname: Warrier
authors.affiliation: Department of Biological Sciences, National University of Singapore, Singapore 117543. dbsxj@nus.edu.sg.|NUS Centre for BioImaging Sciences, National University of Singapore, Singapore 117543. dbsxj@nus.edu.sg.
authors.forename: Jian
authors.initials: J
authors.lastname: Xu
authors.affiliation: Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore. yhloh@imcb.a-star.edu.sg.|Department of Biological Sciences, National University of Singapore, Singapore 117543. yhloh@imcb.a-star.edu.sg.
authors.forename: Yuin-Han
authors.initials: YH
authors.lastname: Loh
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D065150:Cellular Reprogramming; D018390:Gene Targeting; D005818:Genetic Engineering; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000378:metabolism*; D008954:Models, Biological; D019076:Transgenes
keywords: CRISPR; disease modeling; gene editing; iPSCs