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Int. J. Mol. Sci. 2016, 17(5), 626; doi:10.3390/ijms17050626

In Vivo Delivery Systems for Therapeutic Genome Editing

1
Institute for Advancing Translational Medicine in Bone & Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China
2
Central Laboratory, Zheng Zhou Hospital of Traditional Chinese Medicine, Zhengzhou 450000, China
These authors contributed equally to this work.
*
Authors to whom correspondence should be addressed.
Academic Editor: Izuho Hatada
Received: 15 February 2016 / Revised: 11 April 2016 / Accepted: 14 April 2016 / Published: 27 April 2016
(This article belongs to the Special Issue Genome Editing)
View Full-Text   |   Download PDF [2240 KB, uploaded 27 April 2016]   |  

Abstract

Therapeutic genome editing technology has been widely used as a powerful tool for directly correcting genetic mutations in target pathological tissues and cells to cure of diseases. The modification of specific genomic sequences can be achieved by utilizing programmable nucleases, such as Meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly-interspaced short palindromic repeat-associated nuclease Cas9 (CRISPR/Cas9). However, given the properties, such as large size, negative charge, low membrane penetrating ability, as well as weak tolerance for serum, and low endosomal escape, of these nucleases genome editing cannot be successfully applied unless in vivo delivery of related programmable nucleases into target organisms or cells is achieved. Here, we look back at delivery strategies having been used in the in vivo delivery of three main genome editing nucleases, followed by methodologies currently undergoing testing in clinical trials, and potential delivery strategies provided by analyzing characteristics of nucleases and commonly used vectors. View Full-Text
Keywords: in vivo delivery systems; vectors; genome editing; programmable nucleases in vivo delivery systems; vectors; genome editing; programmable nucleases
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Wang, L.; Li, F.; Dang, L.; Liang, C.; Wang, C.; He, B.; Liu, J.; Li, D.; Wu, X.; Xu, X.; Lu, A.; Zhang, G. In Vivo Delivery Systems for Therapeutic Genome Editing. Int. J. Mol. Sci. 2016, 17, 626.

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