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In Vivo Delivery Systems for Therapeutic Genome Editing

by Luyao Wang 1,†, Fangfei Li 1,†, Lei Dang 1,†, Chao Liang 1, Chao Wang 1, Bing He 1, Jin Liu 1, Defang Li 1, Xiaohao Wu 1, Xuegong Xu 2,*, Aiping Lu 1,* and Ge Zhang 1,*
Institute for Advancing Translational Medicine in Bone & Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China
Central Laboratory, Zheng Zhou Hospital of Traditional Chinese Medicine, Zhengzhou 450000, China
Authors to whom correspondence should be addressed.
These authors contributed equally to this work.
Academic Editor: Izuho Hatada
Int. J. Mol. Sci. 2016, 17(5), 626;
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)
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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MDPI and ACS Style

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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