Cyclodextrins for non-viral gene and siRNA delivery

dc.contributor.authorO'Mahony, Aoife M.
dc.contributor.authorO'Driscoll, Caitríona M.
dc.contributor.authorO'Neill, Martin J.
dc.contributor.authorGodinho, Bruno M. D. C.
dc.contributor.authorDarcy, Raphael
dc.contributor.authorCryan, John F.
dc.contributor.funderScience Foundation Irelanden
dc.contributor.funderIrish Research Council for Science Engineering and Technologyen
dc.contributor.funderIrish Drug Delivery Networken
dc.date.accessioned2013-01-09T12:00:54Z
dc.date.available2013-01-09T12:00:54Z
dc.date.issued2013-01
dc.date.updated2013-01-04T14:55:58Z
dc.description.abstractConsiderable research is focused on the development of non-viral vectors for gene and RNA interference therapies, with significant advancements in this field over the past number of years. Cationic lipids and polymers have been extensively investigated for these purposes, but there still remains a need for alternative vectors. Cyclodextrins (CDs) are cyclic oligosaccharides derived from starch and are well characterised pharmaceutical excipients. They offer many advantages as potential non-viral vectors for gene and siRNA delivery, in particular the ease with which they can be chemically modified and their limited toxicity. In recent years, there has been a surge in the number of publications concerning CDs in this field. In this paper, we will review the two main approaches to the use of CDs for gene and siRNA delivery. In the first instance, CDs are used as a scaffold, to which various chemical groups can be grafted, yielding monodisperse functionalised CDs which can self-assemble in the presence of oligonucleotides. CDs are particularly amenable to chemical modification and this approach enables specific and precise design of CD vectors for targeting to various cell and tissue types. In the second approach, CDs can be included as a component of a delivery system, for example, as part of a polymer backbone, appended to a dendrimeric vector, or in polyrotaxane systems. Here, the inclusion of CDs facilitates post-modification of the vector through the formation of inclusion complexes with adamantane and, in some instances, reduces toxicity of the vector. Lastly, we will consider the development of in vivo CD vectors for therapeutic use and other novel applications including siRNA delivery in neurons and the CNS.en
dc.description.sponsorshipScience Foundation Ireland (grant no. 07/SRC/B1154); Irish Research Council for Science Engineering and Technology (Embark initiative)en
dc.description.statusPeer revieweden
dc.description.versionAccepted Versionen
dc.format.mimetypeapplication/pdfen
dc.identifier.citationO'Mahony A.M., O Neill M.J., Godinho B.M.D.C., Darcy, R., Cryan, J.F.,O'Driscoll, C.M. (2013) 'Cyclodextrins for Non-Viral Gene and siRNA Delivery'. Pharmaceutical Nanotechnology, 1(1) pp. 6-14. doi: 10.2174/2211738511301010006en
dc.identifier.doi10.2174/2211738511301010006
dc.identifier.endpage14en
dc.identifier.issn2211-7385
dc.identifier.issn2211-7393
dc.identifier.issued1en
dc.identifier.journaltitlePharmaceutical Nanotechnologyen
dc.identifier.startpage6en
dc.identifier.urihttps://hdl.handle.net/10468/872
dc.identifier.volume1en
dc.language.isoenen
dc.publisherBentham Science Publishersen
dc.relation.urihttp://www.benthamscience.com/pnt/index.htm
dc.rightsCopyright © 2013 Bentham Science Publishers. All Rights Reserved.en
dc.subjectsiRNAen
dc.subjectClick chemistryen
dc.subjectGenesen
dc.subjectNon-viral deliveryen
dc.subjectPre-clinicalen
dc.subject.lcshCyclodextrinsen
dc.titleCyclodextrins for non-viral gene and siRNA deliveryen
dc.typeArticle (peer-reviewed)en
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