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Issue 7, 2010
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Therapeutic targeting in the silent era: advances in non-viral siRNA delivery

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Abstract

Gene silencing using RNA-interference, first described in mammalian systems almost a decade ago, is revolutionizing therapeutic target validation efforts both in vitro and in vivo. Moreover, the potential for using short interfering RNA (siRNA) as a therapy in its own right is also progressing at a significant pace. However, the widespread use of such approaches is contingent on having appropriate systems to achieve clinically appropriate, safe, and efficient delivery of siRNA. There are many physicochemical and biological barriers to such delivery, and a growing emphasis on the design and characterisation of non-viral technologies that will overcome these barriers and expedite targeted delivery. This review discusses the considerations and challenges associated with use of siRNA-based therapeutics, including stability and off-target effects. Speculation is made on the properties of an ideal delivery system and the non-viral delivery approaches used to date, both in vitro and in vivo, are classified and discussed. Moreover, the ability of cyclodextrin-based delivery vectors to fulfil many of the criteria of an ideal delivery construct is also elaborated.

Graphical abstract: Therapeutic targeting in the silent era: advances in non-viral siRNA delivery

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Article information


Submitted
18 Dec 2008
Accepted
01 Mar 2010
First published
29 Apr 2010

Mol. BioSyst., 2010,6, 1143-1161
Article type
Review Article

Therapeutic targeting in the silent era: advances in non-viral siRNA delivery

J. Guo, K. A. Fisher, R. Darcy, J. F. Cryan and C. O’Driscoll, Mol. BioSyst., 2010, 6, 1143
DOI: 10.1039/C001050M

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