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\u6458\u8981\uff1aCurrent genome-editing systems generally rely on inducing DNA double-strand breaks (DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by DSBs can have deleterious effects. CRISPR\/Cas9 system has recently been repurposed to enable target gene activation, allowing regulation of endogenous gene expression without creating DSBs. However, in\u00a0vivo<\/em> implementation of this gain-of-function system has proven difficult. Here, we report a robust system for in\u00a0vivo<\/em> activation of endogenous target genes through trans<\/em>-epigenetic remodeling. The system relies on recruitment of Cas9 and transcriptional activation complexes to target loci by modified single guide RNAs. As proof-of-concept, we used this technology to treat mouse models of diabetes, muscular dystrophy, and acute kidney disease. Results demonstrate that CRISPR\/Cas9-mediated target gene activation can be achieved in\u00a0vivo<\/em>, leading to measurable phenotypes and amelioration of disease symptoms. This establishes new avenues for developing targeted epigenetic therapies against human diseases.<\/p>\n \u5168\u6587PDF\uff1acell-epigenetics-modification<\/a><\/p>\n <\/p>\n","protected":false},"excerpt":{"rendered":"