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Journal article

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.

Abstract:

As a target for gene therapy, Duchenne muscular dystrophy (DMD) presents many obstacles but also an unparalleled prospect for correction by alternative splicing. The majority of mutations in the dystrophin gene occur in the region encoding the spectrin-like central rod domain, which is largely dispensable. Thus, splicing around mutations can generate a shortened but in-frame transcript, permitting translation of a partially functional dystrophin protein. We have tested this idea in vivo in th...

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Publication status:
Published

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Publisher copy:
10.1038/nm897

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Institution:
University of Oxford
Department:
Oxford, MSD, NDORMS
Role:
Author
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Journal:
Nature medicine
Volume:
9
Issue:
8
Pages:
1009-1014
Publication date:
2003-08-05
DOI:
EISSN:
1546-170X
ISSN:
1078-8956
URN:
uuid:6f49047a-e3af-4918-bd78-4ed82c3edc1f
Source identifiers:
223488
Local pid:
pubs:223488

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