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FARAFARA Cure FA

Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy

Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by homozygous expansion of a GAA·TTC trinucleotide repeat within the first intron of the FXN gene, leading to reduced FXN transcription and decreased levels of frataxin protein. Recent advances in FRDA research have revealed the presence of several epigenetic modifications that are either directly or indirectly involved in this FXN gene silencing. Although epigenetic marks may be inherited from one generation to the next, modifications of DNA and histones can be reversed, indicating that they are suitable targets for epigenetic-based therapy.

Read More: Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy


About the Author

Jen Farmer

Jen Farmer

Executive Director

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