Activating frataxin expression by repeat-targeted nucleic acids

Friedreich's ataxia is an incurable genetic disorder caused by a mutant expansion of the trinucleotide GAA within an intronic FXN RNA. This expansion leads to reduced expression of frataxin (FXN) protein and evidence suggests that transcriptional repression is caused by an R-loop that forms bet...

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Detalles Bibliográficos
Autores principales: Li, Liande, Matsui, Masayuki, Corey, David R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4742999/
https://www.ncbi.nlm.nih.gov/pubmed/26842135
http://dx.doi.org/10.1038/ncomms10606

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4742999/
https://www.ncbi.nlm.nih.gov/pubmed/26842135
http://dx.doi.org/10.1038/ncomms10606

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