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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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Detalles Bibliográficos
Autores principales: Soragni, E., Herman, D., Dent, S. Y. R., Gottesfeld, J. M., Wells, R. D., Napierala, M.
Formato: Texto
Lenguaje:English
Publicado: Oxford University Press 2008
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2577344/
https://www.ncbi.nlm.nih.gov/pubmed/18820300
http://dx.doi.org/10.1093/nar/gkn604