As a proof-of-principle, the scientists 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 editing. Their data demonstrate the excision of the expanded CGG-repeat from the fragile X chromosome can result in FMR1 reactivation.
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As a proof-of-principle, the scientists 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 editing. Their data demonstrate the excision of the expanded CGG-repeat from the fragile X chromosome can result in FMR1 reactivation.