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Journal article

Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Abstract:

Duchenne muscular dystrophy is a severe X-linked inherited muscle wasting disorder caused by mutations in the dystrophin gene. Adeno-associated virus (AAV) vectors have been extensively used to deliver genes efficiently for dystrophin expression in skeletal muscles. To overcome limited packaging capacity of AAV vectors (<5 kb), truncated recombinant microdystrophin genes with deletions of most of rod and carboxyl-terminal (CT) domains of dystrophin have been developed. We have previously s...

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Publication status:
Published

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Publisher copy:
10.1089/hum.2011.020

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
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Journal:
Human gene therapy
Volume:
22
Issue:
11
Pages:
1379-1388
Publication date:
2011-11-05
DOI:
EISSN:
1557-7422
ISSN:
1043-0342
URN:
uuid:7550a780-d95b-4181-b5f2-057ed7ac71e6
Source identifiers:
248753
Local pid:
pubs:248753

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