Gene Expression Restored Despite ‘OFF’ State, Opening New Path for Neurodegenerative Disease Treatment

St. Jude Children’s Research Hospital successfully reactivated the frataxin gene, which was suppressed by hundreds of repetitive sequences, using a synthetic regulator. The researchers confirmed that the synthetic regulator “SynGR1” induces gene expression by recruiting the active protein BRD4, even when repressive signals increase. The researchers presented a new possibility for treating neurodegenerative diseases that could overcome the complexity created by combinations of about 20 types of histone modifications.