Applying gene therapy towards Limb Girdle muscular dystrophy 2I and other human muscle diseases.

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1036656

Funding Status
Closed

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Funded Activity Summary

Therapeutic replacement of small, normal sections of the dystrophin gene can prevent muscle wasting in young dystrophic mice with mutations in dystrophin. This project attempts to apply the same principle to treat another inherited muscle disorder, caused by mutations in the FKRP gene. This approach can also potentially be used to enhance muscle regeneration and treat age related muscle atrophy, or synergistically applied with other therapies that target specific genetic mutations.

Funded Activity Details

Start Date: 01-01-2012

End Date: 01-01-2016

Funding Scheme: Early Career Fellowships

Funding Amount: $347,264.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Gene and Molecular Therapy

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

adeno-associated virus | gene delivery | gene expression | gene therapy | muscle disease | muscle regeneration

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