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FARAFARA Cure FA

Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen.

Pharmacological high-throughput screening (HTS) represents a powerful strategy for drug discovery in genetic diseases, particularly when the full spectrum of pathological dysfunctions remains unclear, such as in Friedreich ataxia (FRDA). FRDA, the most common recessive ataxia, results from a generalized deficiency of mitochondrial and cytosolic iron-sulfur cluster (ISC) proteins activity, due to a partial loss of frataxin function, a mitochondrial protein proposed to function as an iron-chaperone for ISC biosynthesis. In the absence of measurable catalytic function for frataxin, a cell-based assay is required for HTS assay.

Read More: Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen.

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Jen Farmer

Jen Farmer

Executive Director

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