Rescue of Fragile X syndrome neurons by DNA methylation editing of the FMR1 gene

Fragile X syndrome (FXS), the most common genetic form of intellectual disability in males, is caused by silencing of the FMR1 gene by hypermethylation of the CGG expansion mutation in the 5’UTR region of FMR1 in FXS patients. Here, we applied recently developed DNA methylation editing tools to reve...

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Gepubliceerd in:Cell
Hoofdauteurs: Liu, X Shawn, Wu, Hao, Krzisch, Marine, Wu, Xuebing, Graef, John, Muffat, Julien, Hnisz, Denes, Li, Charles H., Yuan, Bingbing, Xu, Chuanyun, Li, Yun, Vershkov, Dan, Cacace, Angela, Young, Richard A., Jaenisch, Rudolf
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2018
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6375087/
https://ncbi.nlm.nih.gov/pubmed/29456084
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.cell.2018.01.012
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