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Peptide Conjugation of 2′- O -methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice

✍ Scribed by 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

Book ID: 125514512
Publisher: Mary Ann Liebert
Year: 2014
Tongue: English
Weight: 917 KB
Volume: 24
Category: Article
ISSN: 2159-3337
DOI: 10.1089/nat.2013.0448

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In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

✍ Hans A. Heemskerk; Christa L. de Winter; Sjef J. de Kimpe; Petra van Kuik-Romeij 📂 Article 📅 2009 🏛 John Wiley and Sons 🌐 English ⚖ 409 KB

## Abstract ## Background Antisense‐mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne