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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Date
2012Metadata
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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).