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In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

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...

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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:
1099-498X
URN:
uuid:40628566-1f80-4a57-8fba-1d2ef6c43c76
Source identifiers:
115563
Local pid:
pubs:115563

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