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FARAFARA Cure FA

A Molecule That Binds GAA-TCC Triplet Repeat DNA Increases Frataxin Protein in Patient Cells

Scientists at The Scripps Research Institute, The California Institute of Technology (Caltech) and Texas A & M University Health Science Center have reported that a synthetic molecule that binds the expanded GAA-TCC repeat sequences in the gene associated with Friedreich's ataxia (frataxin) increases expression of this gene in a cell line derived from a Friedreich's patient, with a corresponding increase in frataxin protein. This paper, published in the Proceeding of the National Academy of Sciences of the USA, represents a first step towards development of gene-targeted therapeutics for FRDA. The next steps in assessing this molecule as a potential therapeutic will be to ask whether the molecule functions in neuronal cells, and whether the molecule can access the organs affected in Friedreich's ataxia — the brain and heart. These questions are being explored in animal studies, being conducted both at Caltech and in collaboration with scientists in Brussels, Belgium. We will also want to know if the molecule increases frataxin expression in the brain and heart in a mouse model for FRDA. If the answers to these questions are positive, then other animal studies will be needed to assess the safety of this molecule prior to any thought of clinical trials. So although encouraging, this work is a first step in the long road toward development of a therapy that increases frataxin protein in our patients. This is very important research because there is a belief that by increasing frataxin protein in patients with Friedreich's ataxia that it may slow or stop progression of the disorder. The article, "DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA-TTC repeats in Friedreich's ataxia," can be found online at http://www.pnas.org or (Burnett et al., Proceedings of the National Academy of Sciences of the USA, volume 103, pages 11497-11502, 2006).


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Jen Farmer

Jen Farmer

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