✦ LIBER ✦
Exon skipping and reading through stop codons: two research approaches for a therapy of Duchenne muscular dystrophy. Monaco, 17–18 January, 2004
✍ Scribed by Guenter Scheuerbrandt
- Publisher
- John Wiley and Sons
- Year
- 2005
- Tongue
- English
- Weight
- 85 KB
- Volume
- 7
- Category
- Article
- ISSN
- 1099-498X
- DOI
- 10.1002/jgm.680
No coin nor oath required. For personal study only.
✦ Synopsis
Exon skipping to change Duchenne into Becker dystrophy
The exon-skipping technique tries to change a Duchenne into a Becker mutation. If a deletion or a point mutation disturbs the reading frame, and thus causes Duchenne dystrophy, the reading frame can be restored by removing one or more exons from the mRNA directly before or after the mutation.
Exons can be eliminated from the mRNA using antisense oligoribonucleotides. These are RNA structures consisting of about 20 nucleotides whose