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Title: Induced pluripotency and gene editing in disease modelling: Perspectives and challenges
Authors: Seah, Y.F.S
El Farran, C.A
Warrier, T
Xu, J 
Loh, Y.-H 
Keywords: alkaline phosphatase
alpha 1 antitrypsin
caspase 9
leucine rich repeat kinase 2
octamer transcription factor 4
stage specific embryo antigen 1
transcription activator like effector nuclease
transcription factor Sox2
transforming growth factor beta
zinc finger protein
Alzheimer disease
clustered regularly interspaced short palindromic repeat
Crigler Najjar syndrome
DNA methylation
embryonic stem cell
fragile X syndrome
gene editing
gene targeting
glycogen storage disease type 1
herpes simplex
herpes zoster
nuclear reprogramming
pancreas adenocarcinoma
pluripotent stem cell
Prader Willi syndrome
Williams Beuren syndrome
biological model
CRISPR Cas system
genetic engineering
induced pluripotent stem cell
nuclear reprogramming
Cellular Reprogramming
CRISPR-Cas Systems
Gene Targeting
Genetic Engineering
Induced Pluripotent Stem Cells
Models, Biological
Issue Date: 2015
Citation: Seah, Y.F.S, El Farran, C.A, Warrier, T, Xu, J, Loh, Y.-H (2015). Induced pluripotency and gene editing in disease modelling: Perspectives and challenges. International Journal of Molecular Sciences 16 (12) : 28614-28634. ScholarBank@NUS Repository.
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. © 2015 by the authors; licensee MDPI, Basel, Switzerland.
Source Title: International Journal of Molecular Sciences
ISSN: 1661-6596
DOI: 10.3390/ijms161226119
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