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Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

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 fram...

Ausführliche Beschreibung

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Bibliografische Detailangaben
Hauptverfasser: Vassilia Partali, Michael D. Pungente, Aseel Abu-Dayya, Tushar Khanna, Marcella Flinterman, Nada Abdul Khalique, George Dickson, Liji Raju, Hans-Richard Sliwka, Christer L. Øpstad, Linda J. Popplewell
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI AG 2012-01-01
Schriftenreihe:Molecules
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Online-Zugang:http://www.mdpi.com/1420-3049/17/2/1138/
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