Title : Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

Pub. Date : 2008 Dec 15

PMID : 18784278






1 Functional Relationships(s)
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1 Antisense oligonucleotides (AOs) have the potential to induce functional dystrophin protein expression via exon skipping by restoring in-frame transcripts in the majority of patients suffering from Duchenne muscular dystrophy (DMD). Oligonucleotides, Antisense dystrophin Homo sapiens