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FARAFARA Cure FA

Development of Frataxin Gene Expression Measures for the Evaluation of Experimental Treatments in Friedreich's Ataxia

BACKGROUND:

Friedreich ataxia is a progressive neurodegenerative disorder caused by GAA triplet repeat expansions or point mutations in the FXN gene and, ultimately, a deficiency in the levels of functional frataxin protein. Heterozygous carriers of the expansion express approximately 50% of normal frataxin levels yet manifest no clinical symptoms, suggesting that therapeutic approaches that increase frataxin may be effective even if frataxin is raised only to carrier levels. Small molecule HDAC inhibitor compounds increase frataxin mRNA and protein levels, and have beneficial effects in animal models of FRDA.

Read More: Development of Frataxin Gene Expression Measures for the Evaluation of Experimental Treatments in Friedreich's Ataxia


About the Author

Jen Farmer

Jen Farmer

Executive Director

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