2′-O-Lysylaminohexyl Oligonucleotides: Modifications for Antisense and siRNA
✍ Scribed by Johannes Winkler; Matthias Gilbert; Aneta Kocourková; Martina Stessl; Christian R. Noe
- Publisher
- John Wiley and Sons
- Year
- 2008
- Tongue
- English
- Weight
- 393 KB
- Volume
- 3
- Category
- Article
- ISSN
- 1860-7179
No coin nor oath required. For personal study only.
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## 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