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Journal article

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy

Abstract:

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of dystrophin. Several therapeutic modalities are currently in clinical trials but none will achieve maximum functional rescue and full disease correction. Therefore, we explored the potential of combining the benefits of dystrophin with increases of utrophin, an autosomal paralogue of dystrophin. Utrophin and dystrophin can be co-expressed and co-localized at the same muscle membrane. Wild-type (wt) levels of...

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Publication status:
Published
Peer review status:
Peer reviewed

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Files:
Publisher copy:
10.1093/hmg/ddz049

Authors


More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
Role:
Author
More from this funder
Name:
Medical Research Council
Grant:
MR/N010698/1
Publisher:
Oxford University Press
Journal:
Human Molecular Genetics More from this journal
Volume:
28
Issue:
13
Pages:
2189–2200
Publication date:
2019-03-05
Acceptance date:
2019-02-26
DOI:
EISSN:
1460-2083
ISSN:
0964-6906
Pmid:
30990876
Language:
English
Subjects:
Pubs id:
pubs:992687
UUID:
uuid:8ad70f11-4483-4a16-98a4-20da2a9e22de
Local pid:
pubs:992687
Source identifiers:
992687
Deposit date:
2019-06-06

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