给予高密度脂蛋白模拟物CER - 001治疗遗传性卵磷脂胆固醇酰基转移酶缺乏症。 - Suppr

Administration of the High-Density Lipoprotein Mimetic CER-001 for Inherited Lecithin-Cholesterol Acyltransferase Deficiency.

作者信息

Faguer Stanislas, Colombat Magali, Chauveau Dominique, Bernadet-Monrozies Pauline, Beq Audrey, Delas Audrey, Soler Vincent, Labadens Isabelle, Huart Antoine, Benlian Pascale, Schanstra Joost P

机构信息

Département de Néphrologie et Transplantation d'organes, Centre de Référence des Maladies Rénales Rares, Centre Hospitalier Universitaire de Toulouse, Institut National de la Science et de la Recherche Médicale, INSERM U1297-Institut des Maladies Métaboliques et Cardiovasculaires, and Université Paul Sabatier-Toulouse III, Toulouse, France.

Service d'Anatomo-Pathologie, Institut Universitaire du Cancer de Toulouse-Oncopole, Centre Hospitalier Universitaire de Toulouse, Toulouse, France.

出版信息

Ann Intern Med. 2021 Jul;174(7):1022-1025. doi: 10.7326/L20-1300. Epub 2021 Mar 2.

DOI:10.7326/L20-1300

PMID:33646847

Abstract

摘要

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Administration of the High-Density Lipoprotein Mimetic CER-001 for Inherited Lecithin-Cholesterol Acyltransferase Deficiency.给予高密度脂蛋白模拟物CER - 001治疗遗传性卵磷脂胆固醇酰基转移酶缺乏症。

Ann Intern Med. 2021 Jul;174(7):1022-1025. doi: 10.7326/L20-1300. Epub 2021 Mar 2.

CER-001 ameliorates lipid profile and kidney disease in a mouse model of familial LCAT deficiency.CER-001 改善家族性 LCAT 缺陷小鼠模型的血脂谱和肾脏疾病。

Metabolism. 2021 Mar;116:154464. doi: 10.1016/j.metabol.2020.154464. Epub 2020 Dec 9.

The HDL mimetic CER-001 remodels plasma lipoproteins and reduces kidney lipid deposits in inherited lecithin:cholesterol acyltransferase deficiency.HDL 模拟物 CER-001 重塑血浆脂蛋白，并减少遗传性卵磷脂：胆固醇酰基转移酶缺乏症中的肾脏脂质沉积。

J Intern Med. 2022 Mar;291(3):364-370. doi: 10.1111/joim.13404. Epub 2021 Nov 11.

Lipoprotein subfractions highly associated with renal damage in familial lecithin:cholesterol acyltransferase deficiency.载脂蛋白亚类与家族性卵磷脂：胆固醇脂酰转移酶缺乏症所致的肾脏损害密切相关。

Arterioscler Thromb Vasc Biol. 2014 Aug;34(8):1756-62. doi: 10.1161/ATVBAHA.114.303420. Epub 2014 May 29.

Possible induction of renal dysfunction in patients with lecithin:cholesterol acyltransferase deficiency by oxidized phosphatidylcholine in glomeruli.卵磷脂：胆固醇酰基转移酶缺乏患者肾小球中氧化磷脂酰胆碱可能诱发肾功能障碍。

Arterioscler Thromb Vasc Biol. 1999 Mar;19(3):794-801. doi: 10.1161/01.atv.19.3.794.

[Glomerulopathy associated with lecithin-cholesterol-acyltransferase deficiency: A case report and literature review].[卵磷脂胆固醇酰基转移酶缺乏相关的肾小球病：一例报告及文献复习]

Ann Pathol. 2019 Apr;39(2):172-176. doi: 10.1016/j.annpat.2018.09.005. Epub 2018 Dec 12.

Familial lecithin:cholesterol acyltransferase deficiency: First-in-human treatment with enzyme replacement.家族性卵磷脂：胆固醇酰基转移酶缺乏症：酶替代疗法的首次人体治疗。

J Clin Lipidol. 2016 Mar-Apr;10(2):356-67. doi: 10.1016/j.jacl.2015.12.007. Epub 2015 Dec 23.

Familial Lecithin Cholesterol Acyl Transferase Deficiency with Chronic Kidney Disease.家族性卵磷脂胆固醇酰基转移酶缺乏症合并慢性肾脏病

J Assoc Physicians India. 2016 Oct;64(10):90-91.

Effect of Serial Infusions of CER-001, a Pre-β High-Density Lipoprotein Mimetic, on Coronary Atherosclerosis in Patients Following Acute Coronary Syndromes in the CER-001 Atherosclerosis Regression Acute Coronary Syndrome Trial: A Randomized Clinical Trial.CER-001 动脉粥样硬化消退急性冠脉综合征试验中 CER-001，一种前β高密度脂蛋白类似物，对急性冠脉综合征后患者冠状动脉粥样硬化的连续输注的影响：一项随机临床试验。

JAMA Cardiol. 2018 Sep 1;3(9):815-822. doi: 10.1001/jamacardio.2018.2121.

[Familial LCAT deficiency].[家族性卵磷脂胆固醇酰基转移酶缺乏症]

Nihon Rinsho. 1994 Dec;52(12):3210-5.

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Quo Vadis after AEGIS: New Opportunities for Therapies Targeted at Reverse Cholesterol Transport?在AEGIS之后何去何从：针对逆向胆固醇转运的治疗新机遇？

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HDL and chronic kidney disease.高密度脂蛋白与慢性肾脏病

Atheroscler Plus. 2023 Apr 18;52:9-17. doi: 10.1016/j.athplu.2023.04.001. eCollection 2023 Jun.

Very low HDL levels: clinical assessment and management.极低的高密度脂蛋白水平：临床评估和管理。

Arch Endocrinol Metab. 2023 Jan 18;67(1):3-18. doi: 10.20945/2359-3997000000585.

Two novel variants in the lecithin:cholesterol acyltransferase gene resulted in classic LCAT deficiency.卵磷脂胆固醇酰基转移酶基因中的两个新变异导致了典型的卵磷脂胆固醇酰基转移酶缺乏症。

Atheroscler Plus. 2022 Jun 2;49:28-31. doi: 10.1016/j.athplu.2022.05.005. eCollection 2022 Aug.

High-density lipoprotein revisited: biological functions and clinical relevance.高密度脂蛋白再探：生物学功能与临床相关性。

Eur Heart J. 2023 Apr 21;44(16):1394-1407. doi: 10.1093/eurheartj/ehac605.

Apolipoprotein-A-I for severe COVID-19-induced hyperinflammatory states: A prospective case study.载脂蛋白A-I用于治疗重症新型冠状病毒肺炎所致的高炎症状态：一项前瞻性病例研究。

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First Recombinant High-Density Lipoprotein Particles Administration in a Severe ICU COVID-19 Patient, a Multi-Omics Exploratory Investigation.首例重组高密度脂蛋白颗粒用于重症监护病房（ICU）的新冠肺炎患者：一项多组学探索性研究

Biomedicines. 2022 Mar 23;10(4):754. doi: 10.3390/biomedicines10040754.

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Curr Atheroscler Rep. 2022 May;24(5):365-377. doi: 10.1007/s11883-022-01012-y. Epub 2022 Mar 10.

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Administration of the High-Density Lipoprotein Mimetic CER-001 for Inherited Lecithin-Cholesterol Acyltransferase Deficiency.

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