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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...

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Detalles Bibliográficos
Autores principales: Xie, Nina, Gong, He, Suhl, Joshua A., Chopra, Pankaj, Wang, Tao, Warren, Stephen T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
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