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A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels

10 Feb 2025

Abstract excerpt

Epigenetic editing holds the promise of durable therapeutic effects by silencing disease-causing genes without changing the underlying DNA sequence. In this study, we designed an epigenetic editor to target human PCSK9 and thereby induce DNA methylation at this locus. A single administration of lipid nanoparticles encapsulating mRNA encoding this epigenetic editor was sufficient to drive near-complete silencing...

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PMID: 39930141
DOI: 10.1038/s41591-025-03508-x

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