Ontology highlight
ABSTRACT:
SUBMITTER: Xie N
PROVIDER: S-EPMC5074572 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Xie Nina N Gong He H Suhl Joshua A JA Chopra Pankaj P Wang Tao T Warren Stephen T ST
PloS one 20161021 10
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 elongated CGG repeat. As a proof-of-principle, we excised the expanded CGG-repeat in both somatic cell hybrids containing the human fragile X chromosome and human FXS iPS cells using the CRISPR/Cas9 genome ...[more]