Document Type

Journal Article

Department/Unit

School of Chinese Medicine

Language

English

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.

Keywords

in vivo delivery systems, vectors, genome editing, programmable nucleases

Publication Date

4-2016

Source Publication Title

International Journal of Molecular Sciences

Volume

17

Issue

5

Start Page

626

Publisher

MDPI

Peer Reviewed

1

Copyright

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).

Funder

This study was supported by the Hong Kong General Research Fund (HKBU12102914 to Ge Zhang).

DOI

10.3390/ijms17050626

Link to Publisher's Edition

http://dx.doi.org/10.3390/ijms17050626

ISSN (print)

16616596

ISSN (electronic)

14220067

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