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Title: In vivo genome editing as a therapeutic approach
Authors: Ho B.X.
Loh S.J.H.
Chan W.K. 
Soh B.S. 
Keywords: Cas9
Correcting genetic mutations
Genome editing
In vivo
Issue Date: 2018
Publisher: MDPI AG
Citation: Ho B.X., Loh S.J.H., Chan W.K., Soh B.S. (2018). In vivo genome editing as a therapeutic approach. International Journal of Molecular Sciences 19 (9) : 2721. ScholarBank@NUS Repository.
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. © 2018 by the authors. Licensee MDPI, Basel, Switzerland.
Source Title: International Journal of Molecular Sciences
ISSN: 16616596
DOI: 10.3390/ijms19092721
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