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Molecules 2012, 17(2), 1138-1148; doi:10.3390/molecules17021138
Article

Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

1, 2, 2, 2, 2, 2, 3, 3, 3, 1 and 4,*
1 School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey TW20 0EX, UK 2 Research Division, Weill Cornell Medical College in Qatar, Doha, P.O. Box 24144, Qatar 3 Department of Chemistry, Norwegian University of Science and Technology (NTNU), 7491 Trondheim, Norway 4 Pre-Medical Unit, Weill Cornell Medical College in Qatar, Doha, P.O. Box 24144, Qatar
* Author to whom correspondence should be addressed.
Received: 25 October 2011 / Revised: 22 December 2011 / Accepted: 4 January 2012 / Published: 24 January 2012
(This article belongs to the Special Issue Carotenoids)
Download PDF [1278 KB, 18 June 2014; original version 18 June 2014]

Abstract

Duchenne Muscular Dystrophy (DMD) is a common, inherited, incurable, fatal muscle wasting disease caused by deletions that disrupt the reading frame of the DMD gene such that no functional dystrophin protein is produced. Antisense oligonucleotide (AO)-directed exon skipping restores the reading frame of the DMD gene, and truncated, yet functional dystrophin protein is expressed. The aim of this study was to assess the efficiency of two novel rigid, cationic carotenoid lipids, C30-20 and C20-20, in the delivery of a phosphorodiamidate morpholino (PMO) AO, specifically designed for the targeted skipping of exon 45 of DMD mRNA in normal human skeletal muscle primary cells (hSkMCs). The cationic carotenoid lipid/PMO-AO lipoplexes yielded significant exon 45 skipping relative to a known commercial lipid, 1,2-dimyristoyl-sn-glycero-3-ethylphosphocholine (EPC).
Keywords: cationic lipid; carotenoid; antisense oligonucleotide; exon skipping; Duchenne muscular dystrophy cationic lipid; carotenoid; antisense oligonucleotide; exon skipping; Duchenne muscular dystrophy
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.

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MDPI and ACS Style

Popplewell, L.J.; Abu-Dayya, A.; Khanna, T.; Flinterman, M.; Khalique, N.A.; Raju, L.; Øpstad, C.L.; Sliwka, H.-R.; Partali, V.; Dickson, G.; Pungente, M.D. Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy. Molecules 2012, 17, 1138-1148.

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