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Thesis

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy

Abstract:

Duchenne muscular dystrophy (DMD) is a fatal, muscle-wasting disorder due to mutations/deletions in the dystrophin gene. Whilst improvements in palliative care have increased the life expectancy of patients, cardiomyopathy and respiratory complications are still the leading causes of death. A potential therapy for the treatment of DMD is antisense oligonucleotides (AOs), which modulate dystrophin pre-mRNA splicing to restore the dystrophin reading frame and generate a truncated functional ...

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Research group:
Wood
Oxford college:
St Edmund Hall
Role:
Author
More by this author
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author

Contributors

Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Supervisor
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Supervisor
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Funding agency for:
Betts, C
Grant:
AVRTDF01
Publication date:
2014
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
Oxford University, UK
Language:
English
Keywords:
Subjects:
UUID:
uuid:545d586a-ad7b-4089-8537-b2677957b874
Local pid:
ora:9894
Deposit date:
2015-02-03

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