A broad overview and review of CRISPR-Cas technology and stem cells

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Title: A broad overview and review of CRISPR-Cas technology and stem cells
Author(s): Waddington, SN
Privolizzi, R
Karda, R
O'Neill, HC
Item Type: Journal Article
Abstract: The pinnacle of four decades of research, induced pluripotent stem cells (iPSCs), and genome editing with the advent of clustered, regularly interspaced, short palindromic repeats (CRISPR) now promise to take drug development and regenerative medicine to new levels and to enable the interrogation of disease mechanisms with a hitherto unimaginable level of model fidelity. Autumn 2014 witnessed the first patient receiving iPSCs differentiated into retinal pigmented epithelium to treat macular degeneration. Technologies such as 3D bioprinting may now exploit these advances to manufacture organs in a dish. As enticing as these prospects are, these technologies demand a deeper understanding, which will lead to improvements in their safety and efficacy. For example, precise and more efficient reprogramming for iPSC production is a requisite for wider clinical adoption. Improving awareness of the roles of long non-coding RNAs (lncRNAs) and microRNAs (miRNAs) and genomic epigenetic status will contribute to the achievement of these aims. Similarly, increased efficiency, avoidance of off-target effects, and expansion of available target sequences are critical to the uptake of genome editing technology. In this review, we survey the historical development of genetic manipulation and stem cells. We explore the potential of genetic manipulation of iPSCs for in vitro disease modeling, generation of new animal models, and clinical applicability. We highlight the aspects that define CRISPR-Cas as a breakthrough technology, look at gene correction, and consider some important ethical and societal implications of this approach.
Publication Date: 11-Feb-2016
Date of Acceptance: 1-Feb-2016
URI: http://hdl.handle.net/10044/1/58374
DOI: https://dx.doi.org/10.1007/s40778-016-0037-5
ISSN: 2198-7866
Publisher: Springer Verlag
Start Page: 9
End Page: 20
Journal / Book Title: Current Stem Cell Reports
Volume: 2
Issue: 1
Copyright Statement: © The Author(s) 2016. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
Keywords: CRISPR-Cas
Gene therapy
Genome engineering
Human Genome Editing
iPSCs
CRISPR-Cas
Gene therapy
Genome engineering
Human Genome Editing
iPSCs
Publication Status: Published
Conference Place: Switzerland
Appears in Collections:National Heart and Lung Institute
Faculty of Medicine



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