Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle
β Scribed by K.E. Wells; S. Fletcher; C.J. Mann; S.D. Wilton; D.J. Wells
- Book ID
- 117105740
- Publisher
- Elsevier Science
- Year
- 2003
- Tongue
- English
- Weight
- 336 KB
- Volume
- 552
- Category
- Article
- ISSN
- 0014-5793
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## Abstract ## Background Duchenne and Becker muscular dystrophies are allelic disorders arising from mutations in the dystrophin gene. Duchenne muscular dystrophy is characterised by an absence of functional protein, while Becker muscular dystrophy is usually caused by inβframe deletions allowing
## 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