Targeted Reactivation of FMR1 Transcription in Fragile X Syndrome Embryonic Stem Cells

Targeted Reactivation of FMR1 Transcription in Fragile X Syndrome Embryonic Stem Cells

Fragile X Syndrome (FXS) is the most common inherited cause of intellectual disability and autism. It results from expansion of a CGG nucleotide repeat in the 5′ untranslated region (UTR) of FMR1. Large expansions elicit repeat and promoter hyper-methylation, heterochromatin formation, FMR1 transcri...

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Bibliographic Details
Main Authors: Jill M. Haenfler, Geena Skariah, Caitlin M. Rodriguez, Andre Monteiro da Rocha, Jack M. Parent, Gary D. Smith, Peter K. Todd
Format: Article
Language:English
Published: Frontiers Media S.A. 2018-08-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Fragile X Syndrome
human embryonic stem cells
CRISPR-dCas9
transcriptional activation
VP-192
nucleotide repeat expansion
Online Access:https://www.frontiersin.org/article/10.3389/fnmol.2018.00282/full

Internet

https://www.frontiersin.org/article/10.3389/fnmol.2018.00282/full

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