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Article

Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.

Nucleic acid therapeutics - 1 Feb 2014

Jirka Silvana M G, Heemskerk Hans, Tanganyika-de Winter Christa L, Muilwijk Daan, Pang Kar Him, de Visser Peter C, Janson Anneke, Karnaoukh Tatyana G, Vermue Rick, 't Hoen Peter A C, van Deutekom Judith C T, Aguilera Begoña, Aartsma-Rus Annemieke

Abstract excerpt

Antisense oligonucleotide (AON)-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy that is currently being tested in various clinical trials. This approach is based on restoring the open reading frame of dystrophin transcripts resulting in shorter but partially functional dystrophin proteins as found in patients with Becker muscular dystrophy. After systemic administration,...

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Share to Dystrophin
Share to Mice, Inbred mdx

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Dystrophy, Animal

Share to Muscular Dystrophy, Duchenne

Share to Myocardium

Share to Oligonucleotides, Antisense

Share to Peptide Library

Share to Peptide Nucleic Acids

Source

Identifiers

PMID: 24320790
DOI: 10.1089/nat.2013.0448

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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