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Emerging Therapies for Familial Lecithin-Cholesterol Acyltransferase Deficiency: A Role for Plasma Exchange.

作者信息

Ratnayake Aruni, Turri Marta, Calabresi Laura, Pavanello Chiara, McLean Adam, Tanna Anisha, Cegla Jaimini, Jones Ben, Duncan Neill

机构信息

Imperial College Renal and Transplant Center, Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK.

Centro Grossi Paoletti, Dipartimento di Scienze Farmacologiche Biomolecolari, Università degli Studi di Milano, Italy.

出版信息

Kidney Int Rep. 2024 Apr 16;9(7):2299-2302. doi: 10.1016/j.ekir.2024.04.026. eCollection 2024 Jul.

DOI:10.1016/j.ekir.2024.04.026
PMID:39081770
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11284401/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a978/11284401/fec9fa31be9a/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a978/11284401/445e3973b0c5/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a978/11284401/fec9fa31be9a/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a978/11284401/445e3973b0c5/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a978/11284401/fec9fa31be9a/gr2.jpg

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J Clin Lipidol. 2024 Jul-Aug;18(4):e636-e643. doi: 10.1016/j.jacl.2024.03.002. Epub 2024 Mar 13.
2
First-in-human autologous implantation of genetically modified adipocytes expressing LCAT for the treatment of familial LCAT deficiency.首次将表达卵磷脂胆固醇酰基转移酶（LCAT）的基因修饰脂肪细胞自体植入人体用于治疗家族性LCAT缺乏症。
Heliyon. 2022 Nov 1;8(11):e11271. doi: 10.1016/j.heliyon.2022.e11271. eCollection 2022 Nov.
3
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.
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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.
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Sequential kidney-liver transplantation from the same living donor for lecithin cholesterol acyl transferase deficiency.来自同一活体供体的序贯肝肾移植治疗卵磷脂胆固醇酰基转移酶缺乏症。
Clin Transplant. 2016 Oct;30(10):1370-1374. doi: 10.1111/ctr.12826. Epub 2016 Sep 24.
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PLoS One. 2016 Feb 26;11(2):e0150083. doi: 10.1371/journal.pone.0150083. eCollection 2016.
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Clin Biochem. 2015 May;48(7-8):472-5. doi: 10.1016/j.clinbiochem.2014.08.014. Epub 2014 Aug 27.
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