Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene
Abstract
Fragile X syndrome (FXS), the most common genetic form of intellectual disability in males, is caused by silencing of the FMR1 gene associated with hypermethylation of the CGG expansion mutation in the 5' UTR of FMR1 in FXS patients. Here, we applied recently developed DNA methylation editing tools to reverse this hypermethylation event. Targeted demethylation of the CGG expansion by dCas9-Tet1/single guide RNA (sgRNA) switched the...
Paper Details
Title
Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene
Published Date
Feb 1, 2018
Journal
Volume
172
Issue
5
Pages
979 - 992.e6
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