Investigating Friedreich Ataxia cardiomyopathy and ophthalmopathy using induced pluripotent stem cells

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There is no effective treatment for Friedreich ataxia (FRDA). Patients, usually diagnosed as children, will end up in wheelchairs; many have difficulty with movement, speech, develop eye problems and diabetes. Most die at a young age from heart failure. In this project we will study stem cells from patients with FRDA. In the laboratory, these stem cells will be turned into heart and eye cells to study why patients with FRDA develop symptoms and to help develop new drugs to provide effective treatment.