Long intronic GAA•TTC repeats induce epigenetic changes and reporter gene silencing in a molecular model of Friedreich ataxia

Friedreich ataxia (FRDA) is caused by hyperexpansion of GAA•TTC repeats located in the first intron of the FXN gene, which inhibits transcription leading to the deficiency of frataxin. The FXN gene is an excellent target for therapeutic intervention since (i) 98% of patients carry the same type of m...

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Autors principals: Soragni, E., Herman, D., Dent, S. Y. R., Gottesfeld, J. M., Wells, R. D., Napierala, M.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2008
Matèries:
Gene Regulation, Chromatin and Epigenetics
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2577344/
https://ncbi.nlm.nih.gov/pubmed/18820300
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkn604
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