Muscle binding peptides found by phage display as delivery agent for antisense oligonucleotides

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Abstract summaryDuchenne muscular dystrophy (DMD) is a severe muscle wasting disease, caused by frame shifting mutations in the DMD gene resulting in non-functional dystrophins. Antisense oligonucleotide (AON) mediated exon skipping to restore the reading frame, is a promising therapeutic approach. To further increase the efficiency of this technique and allow systemic application, AON uptake by muscles has to be improved. Using phage display we have searched for muscle homing peptides, which resulted in two promising peptides.

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