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Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome
Fragile X syndrome (FXS) is a common cause of intellectual disability that is most often due to a CGG-repeat expansion mutation in the FMR1 gene that triggers epigenetic gene silencing. Epigenetic modifying drugs can only transiently and modestly induce FMR1 reactivation in the presence of the elong...
Autores principales: | Xie, Nina, Gong, He, Suhl, Joshua A., Chopra, Pankaj, Wang, Tao, Warren, Stephen T. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5074572/ https://www.ncbi.nlm.nih.gov/pubmed/27768763 http://dx.doi.org/10.1371/journal.pone.0165499 |
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