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突变型甲羟戊酸激酶活性的温度依赖性作为高IgD和周期性发热综合征的致病因素

Temperature dependence of mutant mevalonate kinase activity as a pathogenic factor in hyper-IgD and periodic fever syndrome.

作者信息

Houten Sander M, Frenkel Joost, Rijkers Ger T, Wanders Ronald J A, Kuis Wietse, Waterham Hans R

机构信息

Departments of Paediatrics/Emma Children's Hospital and Clinical Chemistry, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands.

出版信息

Hum Mol Genet. 2002 Dec 1;11(25):3115-24. doi: 10.1093/hmg/11.25.3115.

DOI:10.1093/hmg/11.25.3115
PMID:12444096
Abstract

Hyper-IgD and periodic fever syndrome (HIDS) and mevalonic aciduria are autosomal recessive disorders characterized by recurrent episodes of fever and generalized inflammation. Both syndromes are caused by specific mutations in the gene encoding mevalonate kinase (MK), resulting in a depressed enzymatic activity mainly due to reduced protein levels. We studied the effect of temperature on the activity of wild-type and several mutant MKs in fibroblasts. All fibroblast cell lines from HIDS patients and harbouring the common V377I MVK allele displayed substantially higher MK activities at 30 degrees C as compared to 37 degrees C. As shown by temperature inactivation experiments this resulted in a protein nearly as stable as in control cell lines, indicating that primarily the maturation of the protein is affected. Accordingly, when HIDS cell lines were cultured at 39 degrees C, MK activity decreased further. This triggered a compensatory increase in 3-hydroxy-3-methylglutaryl-CoA reductase activity, indicating that MK becomes progressively rate-limiting. A similar phenomenon occurs in vivo. MK activity in peripheral blood mononuclear cells drops 2-8-fold when HIDS patients experience febrile attacks. Our results suggest that minor elevations in temperature can set off a chain of events with MK becoming progressively rate-limiting, leading to a temporary deficiency of isoprenoid end-products, which induces inflammation and fever.

摘要

高IgD血症和周期性发热综合征(HIDS)以及甲羟戊酸尿症是常染色体隐性疾病,其特征为反复发热发作和全身性炎症。这两种综合征均由编码甲羟戊酸激酶(MK)的基因中的特定突变引起,主要由于蛋白质水平降低导致酶活性下降。我们研究了温度对成纤维细胞中野生型和几种突变型MK活性的影响。所有来自HIDS患者且携带常见V377I MVK等位基因的成纤维细胞系在30℃时的MK活性显著高于37℃时。如温度失活实验所示,这导致蛋白质几乎与对照细胞系中的蛋白质一样稳定,表明主要是蛋白质的成熟受到影响。因此,当HIDS细胞系在39℃培养时,MK活性进一步降低。这引发了3-羟基-3-甲基戊二酰辅酶A还原酶活性的代偿性增加,表明MK逐渐成为限速因素。类似现象在体内也会发生。当HIDS患者经历发热发作时,外周血单核细胞中的MK活性会下降2至8倍。我们的结果表明,温度的轻微升高可引发一系列事件,MK逐渐成为限速因素,导致类异戊二烯终产物暂时缺乏,从而诱发炎症和发热。

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