Insights into a PCSK9 structural groove: a harbinger of new drugs to reduce LDL-cholesterol

Nature Structural & Molecular Biology, Published online: 5 October 2017; doi:10.1038/nsmb.3471

PCSK9 enhances LDL cholesterol (LDL-c) levels by escorting the liver LDL receptor (LDLR) to endosomes and lysosomes for degradation. PCSK9 monoclonal antibodies and RNA-antisense formulations are effective in reducing LDL cholesterol in patients. The recent structural identification of a novel pocket in PCSK9 paves the way to the future development of orally active small-molecule hypocholesterolemic drugs.

中文翻译：

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洞悉PCSK9结构槽：减少LDL胆固醇的新药预兆

洞悉PCSK9结构槽：减少LDL胆固醇的新药预兆

《自然结构与分子生物学》，在线发布：2017年10月5日；doi：10.1038 / nsmb.3471

PCSK9通过将肝脏LDL受体（LDLR）护送到内体和溶酶体进行降解，从而提高了LDL胆固醇（LDL-c）的水平。PCSK9单克隆抗体和RNA反义制剂可有效降低患者的LDL胆固醇。PCSK9中一个新口袋的最新结构鉴定为口服活性小分子降胆固醇药的未来开发铺平了道路。