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

Pub. Date : 2012 Jan 24

PMID : 22274137






1 Functional Relationships(s)
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Protein Name
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1 Antisense oligonucleotide (AO)-directed exon skipping restores the reading frame of the DMD gene, and truncated, yet functional dystrophin protein is expressed. Oligonucleotides, Antisense dystrophin Homo sapiens