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Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.

Abstract:

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. In most cases, the open-reading frame is disrupted which results in the absence of functional protein. Antisense-mediated exon skipping is one of the most promising approaches for the treatment of DMD and has recently been shown to correct the reading frame and restore dystrophin expression in vitro and in vivo. Specific exon skipping can be achieved using synthetic oligonucleotide...

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Publication status:
Published

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Publisher copy:
10.1038/mt.2009.113

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
Journal:
Molecular therapy : the journal of the American Society of Gene Therapy
Volume:
17
Issue:
7
Pages:
1234-1240
Publication date:
2009-07-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
URN:
uuid:5b2e90e1-249d-4c08-a190-aa5ce096a1fd
Source identifiers:
106373
Local pid:
pubs:106373

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