Genome-editing Tools for Stem Cell Biology
Overview
General Medicine
Affiliations
Human pluripotent stem cells provide a versatile platform for regenerative studies, drug testing and disease modeling. That the expression of only four transcription factors, Oct4, Klf4, Sox2 and c-Myc (OKSM), is sufficient for generation of induced pluripotent stem cells (iPSCs) from differentiated somatic cells has revolutionized the field and also highlighted the importance of OKSM as targets for genome editing. A number of novel genome-editing systems have been developed recently. In this review, we focus on successful applications of several such systems for generation of iPSCs. In particular, we discuss genome-editing systems based on zinc-finger fusion proteins (ZFs), transcription activator-like effectors (TALEs) and an RNA-guided DNA-specific nuclease, Cas9, derived from the bacterial defense system against viruses that utilizes clustered regularly interspaced short palindromic repeats (CRISPR).
Gutierrez-Guerrero A, Abrey Recalde M, Mangeot P, Costa C, Bernadin O, Perian S Front Genome Ed. 2021; 3:604371.
PMID: 34713246 PMC: 8525375. DOI: 10.3389/fgeed.2021.604371.
Zhang Y, Arango G, Li F, Xiao X, Putatunda R, Yu J BMC Med Genomics. 2018; 11(1):78.
PMID: 30200981 PMC: 6131778. DOI: 10.1186/s12920-018-0394-2.
Modeling rare diseases with induced pluripotent stem cell technology.
Anderson R, Francis K Mol Cell Probes. 2018; 40:52-59.
PMID: 29307697 PMC: 6033695. DOI: 10.1016/j.mcp.2018.01.001.
Induced Pluripotent Stem Cells in Huntington's Disease Research: Progress and Opportunity.
Tousley A, Kegel-Gleason K J Huntingtons Dis. 2016; 5(2):99-131.
PMID: 27372054 PMC: 4942721. DOI: 10.3233/JHD-160199.
Gwiazda K, Grier A, Sahni J, Burleigh S, Martin U, Yang J Mol Ther. 2016; 24(9):1570-80.
PMID: 27203437 PMC: 5113096. DOI: 10.1038/mt.2016.105.