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Int. J. Mol. Sci. 2018, 19(9), 2721; https://doi.org/10.3390/ijms19092721

In Vivo Genome Editing as a Therapeutic Approach

1
Disease Modeling and Therapeutics Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore
2
Department of Biological Sciences, National University of Singapore, Singapore 117543, Singapore
These authors contributed equally to this work.
*
Author to whom correspondence should be addressed.
Received: 2 August 2018 / Revised: 8 September 2018 / Accepted: 10 September 2018 / Published: 12 September 2018
(This article belongs to the Special Issue Genome Editing 2018)
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Abstract

Genome editing has been well established as a genome engineering tool that enables researchers to establish causal linkages between genetic mutation and biological phenotypes, providing further understanding of the genetic manifestation of many debilitating diseases. More recently, the paradigm of genome editing technologies has evolved to include the correction of mutations that cause diseases via the use of nucleases such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALENs), and more recently, Cas9 nuclease. With the aim of reversing disease phenotypes, which arise from somatic gene mutations, current research focuses on the clinical translatability of correcting human genetic diseases in vivo, to provide long-term therapeutic benefits and potentially circumvent the limitations of in vivo cell replacement therapy. In this review, in addition to providing an overview of the various genome editing techniques available, we have also summarized several in vivo genome engineering strategies that have successfully demonstrated disease correction via in vivo genome editing. The various benefits and challenges faced in applying in vivo genome editing in humans will also be discussed. View Full-Text
Keywords: in vivo; genome editing; correcting genetic mutations; ZFN; TALENs; Cas9 in vivo; genome editing; correcting genetic mutations; ZFN; TALENs; Cas9
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
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Ho, B.X.; Loh, S.J.H.; Chan, W.K.; Soh, B.S. In Vivo Genome Editing as a Therapeutic Approach. Int. J. Mol. Sci. 2018, 19, 2721.

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