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FARAFARA Cure FA

Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's Ataxia

Inherited deficiency in the mitochondrial protein frataxin causes the rare disease Friedreich's ataxia (FA), for which there is no successful treatment. We identified a redox deficiency in FA cells and used this to model the disease. We screened a 1600-compound library to identify existing drugs which could be of therapeutic benefit. We identified the topical anesthetic dyclonine as protective. Dyclonine increased FXN transcript and frataxin protein dose-dependently in FA cells and brains of animal models.

Read More: Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's Ataxia


About the Author

Jen Farmer

Jen Farmer

Executive Director

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