Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
β Scribed by McClorey, G; Moulton, H M; Iversen, P L; Fletcher, S; Wilton, S D
- Book ID
- 110039850
- Publisher
- Nature Publishing Group
- Year
- 2006
- Tongue
- English
- Weight
- 362 KB
- Volume
- 13
- Category
- Article
- ISSN
- 0969-7128
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## Abstract ## Background Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by dystrophin gene mutations that preclude synthesis of a functional protein. One potential treatment of the disorder has utilised antisense oligoribonucleotides (AOs) to induce removal of diseaseβassoci
## Abstract ## Background The activity of synthetic antisense oligonucleotides (splicomers) designed to block preβmRNA 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