- Abstract:
-
BACKGROUND: Targeted splice modulation of pre-mRNA transcripts by antisense oligonucleotides (AOs) can correct the function of aberrant disease-related genes. Duchenne muscular dystrophy (DMD) arises as a result of mutations that interrupt the open-reading frame in the DMD gene encoding dystrophin such that dystrophin protein is absent, leading to fatal muscle degeneration. AOs have been shown to correct this dystrophin defect via exon skipping to yield functional dystrophin protein in animal...
Expand abstract - Publisher copy:
- 10.1002/jgm.1446
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- Journal:
- The journal of gene medicine
- Volume:
- 12
- Issue:
- 4
- Pages:
- 354-364
- Publication date:
- 2010-04-05
- DOI:
- EISSN:
-
1521-2254
- ISSN:
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1099-498X
- URN:
-
uuid:40628566-1f80-4a57-8fba-1d2ef6c43c76
- Source identifiers:
-
115563
- Local pid:
- pubs:115563
- Language:
- English
- Keywords:
- Copyright date:
- 2010
Journal article
In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
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