Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries
Open Access
- 1 January 2007
- journal article
- Published by Springer Nature in BMC Molecular Biology
- Vol. 8 (1) , 57
- https://doi.org/10.1186/1471-2199-8-57
Abstract
Antisense oligonucleotides (AOs) can interfere with exon recognition and intron removal during pre-mRNA processing, and induce excision of a targeted exon from the mature gene transcript. AOs have been used in vitro and in vivo to redirect dystrophin pre-mRNA processing in human and animal cells. Targeted exon skipping of selected exons in the dystrophin gene transcript can remove nonsense or frame-shifting mutations that would otherwise have lead to Duchenne Muscular Dystrophy, the most common childhood form of muscle wasting.Keywords
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