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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

✍ Scribed by Bennett, C. Frank; Swayze, Eric E.

Book ID
115544090
Publisher
Annual Reviews
Year
2010
Tongue
English
Weight
776 KB
Volume
50
Category
Article
ISSN
0362-1642

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## Abstract ## Background The activity of synthetic antisense oligonucleotides (splicomers) designed to block pre‐mRNA splicing at specific exons has been demonstrated in a number of model systems, including constitutively spliced exons in mouse dystrophin RNA. Splicomer reagents directed to Duche