Skipping along: an exon skipping therapy shows promise for Duchenne muscular dystrophy
β Scribed by AK Hawkins
- Book ID
- 110889309
- Publisher
- John Wiley and Sons
- Year
- 2011
- Tongue
- English
- Weight
- 511 KB
- Volume
- 80
- Category
- Article
- ISSN
- 0009-9163
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Antisense-mediated exon skipping aiming for reading frame restoration is currently a promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach is mutation specific, but as the majority of DMD patients have deletions that cluster in hotspot regions, the skipping of a smal
## Abstract ## Background Antisenseβmediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne
## 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
## Abstract ## Background Targeted splice modulation of preβmRNA transcripts by antisense oligonucleotides (AOs) can correct the function of aberrant diseaseβrelated genes. Duchenne muscular dystrophy (DMD) arises as a result of mutations that interrupt the openβreading frame in the DMD gene encod