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Journal article

Micro-utrophin improves cardiac and skeletal muscle function of severely affected D2/mdx mice

Abstract:

Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment except palliative care, although personalized treatments such as exon skipping, stop codon read-through, and viral-based gene therapies are making progress. Patients present with skeletal muscle pathology, but most also show cardiomyopathy by the age...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's Version

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Publisher copy:
10.1016/j.omtm.2018.10.005

Authors


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Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Physiology Anatomy and Genetics
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Publisher:
Elsevier Publisher's website
Journal:
Molecular Therapy - Methods and Clinical Development Journal website
Volume:
11
Pages:
92-105
Publication date:
2018-10-16
Acceptance date:
2018-10-08
DOI:
EISSN:
2329-0501
Pubs id:
pubs:940623
URN:
uri:870cd85e-faa0-432f-a83a-90e989c5bce5
UUID:
uuid:870cd85e-faa0-432f-a83a-90e989c5bce5
Local pid:
pubs:940623

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