Developing bone marrow transplant and novel therapeutic vectors to treat Friedreich ataxia

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1048795

Funding Status
Closed

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Funded Activity Summary

We aim to develop effective therapies for the neuromuscular disease Friedreich ataxia (FRDA). The neurodegeneration inherent to FRDA slowly robs a person of the ability to move freely and care for themselves. It needs life-long medical support and there is no cure. FRDA lowers frataxin, a critical mitochondrial protein. Evidence indicates increasing frataxin can be beneficial. Using disease models, we will determine if increasing frataxin via bone marrow transplant or gene therapy improves FRDA.

Funded Activity Details

Start Date: 01-01-2013

End Date: 01-01-2015

Funding Scheme: Project Grants

Funding Amount: $598,163.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Neurogenetics

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

Friedreich ataxia | bone marrow transplantation | gene therapy | lentiviral vector | neurodegenerative disorders

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