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The heat shock protein amplifier arimoclomol improves refolding maturation and lysosomal activity of glucocerebrosidase

机译：热激蛋白放大器阿莫洛莫可改善葡萄糖脑苷脂酶的重折叠成熟和溶酶体活性

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BackgroundGaucher Disease is caused by mutations of the GBA gene which encodes the lysosomal enzyme acid beta-glucosidase (GCase). GBA mutations commonly affect GCase function by perturbing its protein homeostasis rather than its catalytic activity. Heat shock proteins are well known cytoprotective molecules with functions in protein homeostasis and lysosomal function and their manipulation has been suggested as a potential therapeutic strategy for GD. The investigational drug arimoclomol, which is in phase II/III clinical trials, is a well-characterized HSP amplifier and has been extensively clinically tested. Importantly, arimoclomol efficiently crosses the blood-brain-barrier presenting an opportunity to target the neurological manifestations of GD, which remains without a disease-modifying therapy.

机译：背景高雪氏病是由编码溶酶体酶酸性β-葡萄糖苷酶（GCase）的GBA基因突变引起的。 GBA突变通常通过干扰其蛋白质稳态而不是其催化活性来影响GCase的功能。热休克蛋白是众所周知的细胞保护分子，其具有蛋白质稳态和溶酶体功能，并且已建议将其操纵作为GD的潜在治疗策略。处于II / III期临床试验中的研究药物阿莫洛莫是一种特征明确的HSP放大器，并已进行了广泛的临床测试。重要的是，arimoclomol有效地穿过了血脑屏障，为靶向GD的神经系统表现提供了机会，而GD无需进行任何改善疾病的疗法。

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期刊名称 EBioMedicine
作者
Cathrine K. Fog; Paola Zago; Erika Malini; Lukasz M. Solanko; Paolo Peruzzo; Claus Bornaes; Raffaella Magnoni; Arnela Mehmedbasic; Nikolaj H.T. Petersen; Bruno Bembi; Johannes F.M.G. Aerts; Andrea Dardis; Thomas Kirkegaard;
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年(卷),期 2018(38),-1
年度 2018
页码 142–153
总页数 12
原文格式 PDF
正文语种
中图分类
关键词
Gaucher disease GBA Heat shock response Arimoclomol Heat shock proteins HSP HSP70 Proteostasis GCase Lysosomes Lysosomal storage diseases Sphingolipidoses;

机译：高雪氏病;GBA;热休克反应;扑热息痛;热休克蛋白;HSP;HSP70;蛋白稳定;GCase;溶酶体;溶酶体贮积病;鞘脂;

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专利

1. The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase [J] . Cathrine K. Fog, Paola Zago, Erika Malini, EBioMedicine . 2018,第4期

机译：热激蛋白放大器阿莫洛莫改善葡萄糖脑苷脂酶的重折叠，成熟和溶酶体活性
2. Arimoclomol, a clinical candidate for neuronopathic Gaucher disease, increases glucocerebrosidase activity through amplification of heat shock proteins [J] . Kirkegaard Thomas, Fog-Tonnesen Cathrine Kolster, Zago Paola, Molecular genetics and metabolism . 2018,第2期

机译：神经治疗Gaucher病的临床候选者Arimoclomol通过扩增热休克蛋白来增加葡萄糖酶活性
3. Brain-penetrant heat shock protein amplifier arimoclomol enhances GCase activity in in vitro Gaucher disease models [J] . Aubryanna Hettinghouse, Chuan-ju Liu EBioMedicine . 2018,第152期

机译：脑渗透热休克蛋白放大器阿莫洛莫可增强体外高雪氏病模型中的GCase活性
4. Analysis of Small Heat Shock Protein Gene Family Expression (RNA-Seq) during the Tomato Fruit Maturation [C] . D.P. Arce, F.J. Krsliccvic, M.R. Berlolaccini Latin American Congress on Biomedical Engineering . 2015

机译：番茄果实成熟过程中小热休克蛋白基因家族表达（RNA-SEQ）分析
5. The expression of heat shock protein 70, heat shock protein 90 and heat shock factor 1 within the bovine midcycle corpus luteum after stimulation with temperature in vitro and PGF2alpha in vitro and in vivo. [D] . Kopka, Melissa N. 2006

机译：热激蛋白70，热激蛋白90和热激因子1在体外温度和体外及体内PGF2α刺激后在牛黄体中体中的表达。
6. Brain-penetrant heat shock protein amplifier arimoclomol enhances GCase activity in in vitro Gaucher disease models [O] . Aubryanna Hettinghouse, Chuan-ju Liu 2018

机译：脑渗透热休克蛋白放大器阿莫洛莫可增强体外高雪氏病模型中的GCase活性
7. The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase [O] . Cathrine K. Fog, Paola Zago, Erika Malini, 2018

机译：热休克蛋白放大器Arimoclomol可提高葡萄糖纤维素酶的重折叠，成熟和溶酶体活性

The heat shock protein amplifier arimoclomol improves refolding maturation and lysosomal activity of glucocerebrosidase

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