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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...
Autores principales: | Soragni, E., Herman, D., Dent, S. Y. R., Gottesfeld, J. M., Wells, R. D., Napierala, M. |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2008
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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 |
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