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...

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發表在:Molecules
Main Authors: Popplewell, Linda J., Abu-Dayya, Aseel, Khanna, Tushar, Flinterman, Marcella, Khalique, Nada Abdul, Raju, Liji, Øpstad, Christer L., Sliwka, Hans-Richard, Partali, Vassilia, Dickson, George, Pungente, Michael D.
格式: Artigo
語言:Inglês
出版: MDPI 2012
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6268324/
https://ncbi.nlm.nih.gov/pubmed/22274137
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/molecules17021138
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