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,* email
Received: 25 October 2011; in revised form: 22 December 2011 / Accepted: 4 January 2012 / Published: 24 January 2012
(This article belongs to the Special Issue Carotenoids)
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.
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
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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.

AMA Style

Popplewell LJ, Abu-Dayya A, Khanna T, Flinterman M, Khalique NA, Raju L, Øpstad CL, Sliwka H-R, Partali V, Dickson G, Pungente MD. Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy. Molecules. 2012; 17(2):1138-1148.

Chicago/Turabian Style

Popplewell, Linda J.; Abu-Dayya, Aseel; Khanna, Tushar; Flinterman, Marcella; Khalique, Nada Abdul; Raju, Liji; Øpstad, Christer L.; Sliwka, Hans-Richard; Partali, Vassilia; Dickson, George; Pungente, Michael D. 2012. "Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy." Molecules 17, no. 2: 1138-1148.

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