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散发性克雅氏病患者嫁入格斯特曼-施特劳斯勒-谢因克家族:朊病毒传播的微嵌合体研究。

Sporadic Creutzfeldt-Jakob Disease in a Woman Married Into a Gerstmann-Sträussler-Scheinker Family: An Investigation of Prions Transmission via Microchimerism.

机构信息

Danish Reference Center for Prion Diseases, Department of Pathology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.

Department of Neurology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.

出版信息

J Neuropathol Exp Neurol. 2018 Aug 1;77(8):673-684. doi: 10.1093/jnen/nly043.

Abstract

This is the first report of presumed sporadic Creutzfeldt-Jakob disease (sCJD) and Gerstmann-Sträussler-Scheinker disease (GSS) with the prion protein gene c.305C>T mutation (p.P102L) occurring in one family. The father and son were affected with GSS and the mother had a rapidly progressive form of CJD. Diagnosis of genetic, variant, and iatrogenic CJD was ruled out based on the mother's clinical history, genetic tests, and biochemical investigations, all of which supported the diagnosis of sCJD. However, given the low incidence of sCJD and GSS, their co-occurrence in one family is extraordinary and challenging. Thus, a hypothesis for the transmission of infectious prion proteins (PrPSc) via microchimerism was proposed and investigated. DNA from 15 different brain regions and plasma samples of the CJD patient was subjected to PCR and shallow sequencing for detection of a male sex-determining chromosome Y (chr. Y). However, no trace of chr. Y was found. A long CJD incubation period or presumed small concentrations of chr. Y may explain the obtained results. Further studies of CJD and GSS animal models with controlled genetic and proteomic features are needed to determine whether maternal CJD triggered via microchimerism by a GSS fetus might present a new PrPSc transmission route.

摘要

这是首例家族性朊蛋白基因 c.305C>T 突变(p.P102L)所致散发性 Creutzfeldt-Jakob 病(sCJD)和 Gerstmann-Sträussler-Scheinker 病(GSS)的报告。父亲和儿子患有 GSS,母亲患有快速进展型 CJD。根据母亲的临床病史、基因检测和生化研究,排除了遗传、变异和医源性 CJD 的诊断,所有这些都支持 sCJD 的诊断。然而,鉴于 sCJD 和 GSS 的发病率较低,它们在一个家庭中的同时发生是罕见且具有挑战性的。因此,提出并研究了通过微嵌合体传播传染性朊病毒蛋白(PrPSc)的假设。对 CJD 患者的 15 个不同脑区的 DNA 和血浆样本进行 PCR 和浅层测序,以检测男性性别决定染色体 Y(chr. Y)。然而,没有发现 chr. Y 的痕迹。较长的 CJD 潜伏期或假定的 chr. Y 浓度低可能解释了获得的结果。需要对具有受控遗传和蛋白质组学特征的 CJD 和 GSS 动物模型进行进一步研究,以确定母体 CJD 是否可能通过 GSS 胎儿的微嵌合体引发新的 PrPSc 传播途径。

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