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Mesenchymal stem cells restore frataxin expression and increase hydrogen peroxide scavenging enzymes in friedreich ataxia fibroblasts

Dramatic advances in recent decades in understanding the genetics of Friedreich ataxia (FRDA)--a GAA triplet expansion causing greatly reduced expression of the mitochondrial protein frataxin--have thus far yielded no therapeutic dividend, since there remain no effective treatments that prevent or even slow the inevitable progressive disability in affected individuals.

Mesenchymal stem cells restore frataxin expression and increase hydrogen peroxide scavenging enzymes in friedreich ataxia fibroblasts


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Jen Farmer

Jen Farmer

Executive Director

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