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T.P.2.05 Direct comparison of 2′O-methyl and PMO AONs for exon skipping in preclinical model in DMD

✍ Scribed by H.A. Heemskerk; C.L. de Winter; S.J. de Kimpe; P. van Kuik-Romeijn; N. Heuvelmans; G.J. Platenburg; G.J.B. van Ommen; J.C.T. van Deutekom; A. Aartsma-Rus


Book ID
116793533
Publisher
Elsevier Science
Year
2008
Tongue
English
Weight
49 KB
Volume
18
Category
Article
ISSN
0960-8966

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In vivo comparison of 2′-O-methyl phosph
✍ 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