Epigenetic editing of PCSK9 for a durable reduction in cholesterol

IF 50 1区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Nature Medicine Pub Date : 2025-02-25 DOI:10.1038/s41591-025-03583-0
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Abstract

A sustained decrease in low-density lipoprotein cholesterol (LDL-C) is key to reducing the risk of atherosclerotic cardiovascular disease; however, achieving this goal can be hampered by low adherence to standard of care and the sub-maximal efficacy of currently available long-acting therapeutics. We developed an epigenetic editor that targets PCSK9 and robustly and durably decreases LDL-C.

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对PCSK9进行表观遗传编辑以持久降低胆固醇
持续降低低密度脂蛋白胆固醇(LDL-C)是降低动脉粥样硬化性心血管疾病风险的关键;然而,实现这一目标可能会受到对标准护理的低依从性和目前可用的长效治疗药物的次最大疗效的阻碍。我们开发了一种以PCSK9为靶点的表观遗传编辑器,可以稳健而持久地降低LDL-C。
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来源期刊
Nature Medicine
Nature Medicine 医学-生化与分子生物学
CiteScore
100.90
自引率
0.70%
发文量
525
审稿时长
1 months
期刊介绍: Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.
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