Optimization of splice switching therapies to treat Duchenne muscular dystrophy [ 2013 - 2015 ]

Also known as: Enhanced exon skipping to treat Duchenne muscular dystrophy

Research Grant

[Cite as]

Researchers: Prof Stephen Wilton (Principal investigator) ,  Dr Roberto Barrero Prof Matthew Bellgard Prof Sue Fletcher

Brief description Duchenne muscular dystrophy, the most common and serious form of childhood muscle wasting, is caused by mutations in the dystrophin gene that block synthesis of the normal product. Antisense oligomers have been used in clinical trials to remove the disease-causing part of the message and rescue expression. Clinical trials have demonstrated proof-of-concept, although individual responses varied. This application seeks to improve the therapeutic potential of these compounds.

Funding Amount $AUD 448,827.57

Funding Scheme Project Grants

Notes Standard Project Grant

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