The epigenome editing framework provides an engineering approach to explore chromatin-based gene expression mechanisms. However, therapeutic utility of epigenetic editing-based systems has been lacking. A report in Cell (Liu et. al., 2018) shows that epigenetic editors can revert abnormal heterochromatin formation at the gene promoter leading to restoration of FMR1 gene expression, functionally rescuing fragile X syndrome (FXS), an otherwise unamenable genetic disorder.

中文翻译：

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表观遗传编辑：为营救目的

表观基因组编辑框架为探索基于染色质的基因表达机制提供了一种工程方法。然而，缺乏基于表观遗传编辑的系统的治疗用途。Cell上的一篇报道（Liu等人，2018）表明，表观遗传编辑器可以还原基因启动子处的异常异染色质形成，从而导致FMR1基因表达的恢复，在功能上挽救脆弱的X综合征（FXS），这是否则无法解决的遗传疾病。