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Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.

Abstract:

Recombinant adeno-associated virus (rAAV) vectors have been shown to permit very efficient widespread transgene expression in skeletal muscle after systemic delivery, making these increasingly attractive as vectors for Duchenne muscular dystrophy (DMD) gene therapy. DMD is a severe muscle-wasting disorder caused by DMD gene mutations leading to complete loss of dystrophin protein. One of the major issues associated with delivery of the DMD gene, as a therapeutic approach for DMD, is its large...

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Publication status:
Published

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Publisher copy:
10.1089/hum.2013.164

Authors


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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Popplewell, L More by this author
Athanasopoulos, T More by this author
Dickson, G More by this author
Journal:
Human gene therapy
Volume:
25
Issue:
2
Pages:
98-108
Publication date:
2014-02-05
DOI:
EISSN:
1557-7422
ISSN:
1043-0342
URN:
uuid:2d01f8af-2641-4b9d-97ba-6a8b05e837d8
Source identifiers:
442063
Local pid:
pubs:442063

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