Popplewell, Linda J, Abu-Dayya, Aseel, Khana, Tushar, Flinterman, Marcella, Khalique, Nada Abdul, Raju, Liji, Opstad, Christer L, Sliwka, Hans-Richard, Partali, Vassilia, Dickson, George and Pungente, Michael D (2012) Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in duchenne muscular dystrophy. Molecules, 17 (2).
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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).
This is a Submitted version This version's date is: 2012 This item is not peer reviewed
https://repository.royalholloway.ac.uk/items/ef69817b-9c4c-9251-e13d-20def2e5a97d/4/
Deposited by Research Information System (atira) on 03-Jul-2014 in Royal Holloway Research Online.Last modified on 03-Jul-2014