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

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Tác giả: Sahar Abubucker, Scott Clarkson, Gabriel K El Sebae, Taesun Eom, Ari E Friedland, Cristiana Giancarlo, Erica M Hildebrand, Aron B Jaffe, Kenneth Kelly, Chih-Wei Ko, Samantha J Linder, Songlei Liu, Liam Lofgren, Angelo Lombardo, Morgan L Maeder, Padma Malyala, Salette Martinez, Mary S Morrison, Vic E Myer, Shefal Parikh, Ricardo N Ramirez, Shrijal S Shah, Frederic Tremblay, Sarah B Voytek, Christine Waters, Yuki Weber, Shane H Wright, Qiang Xiong

Ngôn ngữ: eng

Ký hiệu phân loại: 636.0885 Animal husbandry

Thông tin xuất bản: United States : Nature medicine , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 51240

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 of human PCSK9 in transgenic mice. Silencing was durable for at least 1 year and was fully maintained after partial hepatectomy-induced liver regeneration. In addition, we showed reversibility of epigenetic editing in mice with previously silenced PCSK9 upon treatment with a targeted epigenetic activator designed to demethylate the PCSK9 locus. Notably, in cynomolgus monkeys, a single administration of the epigenetic editor potently and durably decreased circulating PCSK9 protein levels by approximately 90% with concomitant reduction in low-density lipoprotein cholesterol levels by approximately 70%. These findings demonstrate the therapeutic potential of durable and reversible epigenetic editing in vivo and support the development of epigenetic editor-based treatment for hypercholesterolemia.
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