In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation
Liao, Hsin-Kai; Hatanaka, Fumiyuki; Araoka, Toshikazu; Reddy, Pradeep; Wu, Min-Zu; Sui, Yinghui; Yamauchi, Takayoshi; Sakurai, Masahiro; O'Keefe, David D.; Nunez-Delicado, Estrella; Guillen, Pedro; Campistol, Josep M.; Wu, Cheng-Jang; Lu, Li-Fan; Rodriguez
CELL
2017
VL / 171 - BP / 1495 - EP / +
abstract
Current 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 vivo implementation of this gain-of-function system has proven difficult. Here, we report a robust system for in vivo activation of endogenous target genes through trans-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 vivo, leading to measurable phenotypes and amelioration of disease symptoms. This establishes new avenues for developing targeted epigenetic therapies against human diseases.
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820 InfluRatio
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Green accepted, Bronze
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Biology & Biochemistry
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57 Wikipedia
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721 Policy
Molecular Biology & Genetics
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3 Twitter
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57 Wikipedia
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721 Policy
Among papers in Biology & Biochemistry
Among papers in Molecular Biology & Genetics
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