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Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in duchenne muscular dystrophy

Popplewell, Linda; Abu-Dayya, Aseel; Khanna, Tushar; Flinterman, Marcella; Abdul Khalique, nada; Raju, Liji; Øpstad, Christer Lorentz; Sliwka, Hans-Richard; Partali, Vassilia; Dickson, George; Pungente, Michael
Journal article, Peer reviewed
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URI
http://hdl.handle.net/11250/2359499
Date
2012
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  • Institutt for kjemi [805]
  • Publikasjoner fra CRIStin - NTNU [20804]
Original version
Molecules 2012, 17(2):1138-1148   10.3390/molecules17021138
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).
Publisher
MDPI
Journal
Molecules

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