Fondazione IRCCS Istituto Neurologico Carlo Besta, Division of Neurology 5-Neuropathology, Milan.
Fondazione IRCCS Istituto Neurologico Carlo Besta, Division of Neurology 5-Neuropathology, Milan, Italy.
Eur J Histochem. 2021 Oct 15;65(s1):3298. doi: 10.4081/ejh.2021.3298.
Sporadic Creutzfeldt-Jakob disease (sCJD) is a rare and fatal neurodegenerative disorder with an incidence of 1.5 to 2 cases per million population/year. The disease is caused by a proteinaceous infectious agent, named prion (or PrPSc), which arises from the conformational conversion of the cellular prion protein (PrPC). Once formed, PrPSc interacts with the normally folded PrPC coercing it to undergo similar structural rearrangement. The disease is highly heterogeneous from a clinical and neuropathological point of view. The origin of this variability lies in the aberrant structures acquired by PrPSc. At least six different sCJD phenotypes have been described and each of them is thought to be caused by a peculiar PrPSc strain. Definitive sCJD diagnosis requires brain analysis with the aim of identifying intracerebral accumulation of PrPSc which currently represents the only reliable biomarker of the disease. Clinical diagnosis of sCJD is very challenging and is based on the combination of several clinical, instrumental and laboratory tests representing surrogate disease biomarkers. Thanks to the advent of the ultrasensitive Real-Time Quaking-Induced Conversion (RT-QuIC) assay, PrPSc was found in several peripheral tissues of sCJD patients, sometimes even before the clinical onset of the disease. This discovery represents an important step forward for the clinical diagnosis of sCJD. In this manuscript, we present an overview of the current applications and future perspectives of RT-QuIC in the field of sCJD diagnosis.
散发性克雅氏病(sCJD)是一种罕见且致命的神经退行性疾病,发病率为每百万人口每年 1.5 至 2 例。该疾病是由一种名为朊病毒(或 PrPSc)的蛋白质传染性病原体引起的,它源自细胞朊蛋白(PrPC)的构象转换。一旦形成,PrPSc 与正常折叠的 PrPC 相互作用,迫使它发生类似的结构重排。从临床和神经病理学的角度来看,该疾病具有高度异质性。这种可变性的起源在于 PrPSc 获得的异常结构。已经描述了至少六种不同的 sCJD 表型,每种表型都被认为是由一种特殊的 PrPSc 株引起的。明确的 sCJD 诊断需要对大脑进行分析,目的是识别脑内 PrPSc 的积累,这目前是该疾病唯一可靠的生物标志物。sCJD 的临床诊断极具挑战性,基于几项临床、仪器和实验室测试的组合,这些测试代表了替代疾病生物标志物。得益于超灵敏实时震颤诱导转换(RT-QuIC)检测的出现,在 sCJD 患者的几种外周组织中发现了 PrPSc,甚至在疾病临床发作之前。这一发现代表了 sCJD 临床诊断的重要一步。在本文中,我们概述了 RT-QuIC 在 sCJD 诊断领域的当前应用和未来前景。