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Thesis

Oligonucleotide-based therapies for neuromuscular disease

Abstract:

Genetic neuromuscular diseases remain essentially untreatable. Duchenne muscular dystrophy (DMD) is one such example and another is C9ORF72-related frontotemporal dementia/amyotrophic lateral sclerosis (c9FTD/ALS). Both these conditions may, however, be amenable to treatment with antisense oligonucleotide (ASO) compounds. Therapeutic ASO development for DMD is already in the clinical trial phase, while the applicability of ASO therapies in c9FTD/ALS remains to be clarified. No rel...

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Research group:
Matthew Wood, Kevin Talbot
Oxford college:
Green Templeton College
Role:
Author
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Division:
MPLS
Department:
Doctoral Training Centre - MPLS
Role:
Author

Contributors

Division:
MPLS
Department:
Doctoral Training Centre - MPLS
Role:
Supervisor
Division:
MPLS
Department:
Doctoral Training Centre - MPLS
Role:
Supervisor
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Funding agency for:
Douglas, A
Grant:
AVRVTB0
Publication date:
2015
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
Oxford University, UK

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