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Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy

Abstract:

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. Antisense-mediated exon skipping is one of the most promising approaches for the treatment of DMD but still faces personalized medicine challenges as different mutations found in DMD patients require skipping of different exons. However, 70% of DMD patients harbor dystrophin gene deletions in a mutation-rich area or "hot-spot" in the central genomic region. In this study, we have d...

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Publisher copy:
10.1038/mt.2012.26

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Journal:
Molecular Therapy
Volume:
20
Issue:
6
Pages:
1212-1221
Publication date:
2012-06-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
URN:
uuid:80c66d5b-8db3-4043-bf49-c1ba01cf063b
Source identifiers:
340197
Local pid:
pubs:340197
Language:
English

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