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AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy

Abstract:

In the context of future adeno-associated viral (AAV)-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We showed that AAV genomes encoding non-therapeutic U7 were lost from mdx dystrophic muscles within 3 weeks after intramuscular injection. In contrast, AAV genomes encoding U7ex23 restoring expression of a slightly shortened dystrophin were maintained endorsing ...

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Publisher copy:
10.1038/mt.2013.121

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
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Journal:
Molecular Therapy
Volume:
21
Issue:
8
Pages:
1551-1558
Publication date:
2013-08-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
URN:
uuid:77d0dc56-47b1-42f8-be1b-171e1111922c
Source identifiers:
419341
Local pid:
pubs:419341
Language:
English

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