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Friedreich's ataxia: An effective gene therapy in an animal model

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The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy of gene therapy for treating the heart disease associated with Friedreich's ataxia, a rare hereditary neuro-degenerative disorder.

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