原创力文档novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in duchenne muscular dystrophy新型阳离子类胡萝卜素脂质作为外显子不交付向量的反义寡核苷酸在杜氏肌萎缩症.pdfVIP
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novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in duchenne muscular dystrophy新型阳离子类胡萝卜素脂质作为外显子不交付向量的反义寡核苷酸在杜氏肌萎缩症
Molecules 2012, 17, 1138-1148; doi:10.3390/molecule
OPEN ACCESS
molecules
ISSN 1420-3049
/journal/molecules
Article
Novel Cationic Carotenoid Lipids as Delivery Vectors of
Antisense Oligonucleotides for Exon Skipping in Duchenne
Muscular Dystrophy
1 2 2 2
Linda J. Popplewell , Aseel Abu-Dayya , Tushar Khanna , Marcella Flinterman ,
Nada Abdul Khalique 2, Liji Raju 2, Christer L. Øpstad 3, Hans-Richard Sliwka 3,
Vassilia Partali 3, George Dickson 1 and Michael D. Pungente 4,*
1 School of Biological Sciences, Royal Holloway-University of London, Egham, Surrey TW20 0EX,
UK
2 Research Division, Weill Cornell Medical College in Qatar, Doha, P.O. Box 24144, Qatar
3 Department of Chemistry, Norwegian University of Science and Technology (NTNU), 7491
Trondheim, Norway
4 Pre-Medical Unit, Weill Cornell Medical College in Qatar, Doha, P.O. Box 24144, Qatar
* Author to whom correspondence should be addressed; E-Mail: mdp2001@;
Tel.: +974-4492-8216; Fax: +974-4492-8222.
Received: 25 October 2011; in revised form: 22 December 2011 / Accepted: 4 January 2012 /
Published: 24 January 2012
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 t
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