Abstract
Friedreich's ataxia (FA) is the most common recessive ataxia, affecting 1|[ndash]|2 in 50,000 Caucasians, and there is currently no effective cure or treatment. FA results from a deficiency in frataxin, a mitochondrial protein, caused by repeat expansion in intron 1 of the FRDA gene. The main areas affected are the central nervous system (particularly the spinocerebellar system) and the cardiac tissue. Therapies aimed at alleviating the neurological degeneration have proved unsuccessful to date, although some treatments based upon administration of antioxidants have been used to treat the cardiac condition. We are interested in developing a functional complementation gene therapy strategy to treat the neurological disorder and correct mitochondrial dysfunction.
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