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Durable and efficient gene silencing in vivo by hit-and-run epigenome editing - Nature

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Durable and efficient gene silencing in vivo by hit-and-run epigenome editing - Nature

Permanent epigenetic silencing using programmable editors equipped with transcriptional repressors holds great promise for the treatment of human diseases1–3. However, to unlock its full therapeutic potential, an experimental confirmation of durable epigenetic silencing after the delivery of transient delivery of editors in vivo is needed. To this end, here we targeted Pcsk9, a gene expressed in hepatocytes that is involved in cholesterol homeostasis.

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Durable and efficient gene silencing in vivo by hit-and-run epigenome editing - Nature

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Durable and efficient gene silencing in vivo by hit-and-run epigenome editing - Nature

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