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DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA·TTC repeats in Friedreich's ataxia

The DNA abnormality found in 98% of Friedreich's ataxia (FRDA) patients is the unstable hyperexpansion of a GAA·TTC triplet repeat in the first intron of the frataxin gene. Expanded GAA·TTC repeats result in decreased transcription and reduced levels of frataxin protein in affected individuals....

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Bibliografische gegevens
Hoofdauteurs: Burnett, Ryan, Melander, Christian, Puckett, James W., Son, Leslie S., Wells, Robert D., Dervan, Peter B., Gottesfeld, Joel M.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: National Academy of Sciences 2006
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1544198/
https://ncbi.nlm.nih.gov/pubmed/16857735
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0604939103
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