Developing bone marrow transplant and novel therapeutic vectors to treat Friedreich ataxia [ 2013 - 2015 ]

Research Grant

Researchers: Prof Martin Delatycki (Principal investigator) ,  A/Pr Alice Pebay ,  Dr Marguerite Evans-Galea ,  Prof Beverly Davidson

Brief description 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.

Funding Amount $AUD 598,163.79

Funding Scheme Project Grants

Notes Standard Project Grant