Optimization of splice switching therapies to treat Duchenne muscular dystrophy

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1043758

Funding Status
Closed

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Funded Activity Summary

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.

Funded Activity Details

Start Date: 01-01-2013

End Date: 01-01-2015

Funding Scheme: Project Grants

Funding Amount: $448,827.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Biomolecular Modelling and Design

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

Duchenne muscular dystrophy | antisense technology | computer-based decision support | gene expression | splicing | therapy

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