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New cellular model to follow FA development in time-resolved way

Friedreich's ataxia (FRDA) is a recessive autosomal ataxia caused by reduced levels of frataxin, an essential mitochondrial protein highly conserved from bacteria to primates. The exact role of frataxin and its primary function remain unclear although this information would be very valuable to design a therapeutic approach for FRDA. A main difficulty encountered so far has been that of establishing a clear temporal relationship between the different observations that could allow a distinction between causes and secondary effects and provide a clear link between aging and disease development. To approach this problem, we developed a cellular model in which we can switch off/on in a time-controlled way the frataxin gene partially mimicking what happens in the disease.

Read More: A new cellular model to follow Friedreich's ataxia development in a time-resolved way




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