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

Linda J Popplewell, Aseel Abu-Dayya, Tushar Khanna, Marcella Flinterman, Nada Abdul Khalique, Liji Raju, Christer L Øpstad, Hans-Richard Sliwka, Vassilia Partali, George Dickson, Michael D Pungente

Research output: Contribution to journalArticlepeer-review

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).

Original languageEnglish
Pages (from-to)1138-1148
Number of pages11
JournalMolecules
Volume17
Issue number2
DOIs
Publication statusPublished - 24 Jan 2012
Externally publishedYes

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