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Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

27 Jun 2019

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphorodiamidate morpholino backbone (PMO) of ASOs generated the peptide-conjugated PMOs (PPMOs) that exhibit a...

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PMID: 31257147
DOI: 10.1016/j.ebiom.2019.06.036

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