Llorens Franc, Thüne Katrin, Schmitz Matthias, Ansoleaga Belén, Frau-Méndez Margalida A, Cramm Maria, Tahir Waqas, Gotzmann Nadine, Berjaoui Sara, Carmona Margarita, Silva Christopher J, Fernandez-Vega Ivan, José Zarranz Juan, Zerr Inga, Ferrer Isidro
Department of Neurology, University Medical Center Göttingen, and German Center for Neurodegenerative Diseases (DZNE)-site Göttingen, Göttingen 37075, Germany.
Institute of Neuropathology, Service of Pathological Anatomy, Bellvitge University Hospital, University of Barcelona, Bellvitge Biomedical Research Institute (IDIBELL), Hospitalet de Llobregat, and Biomedical Research Center of Neurodegenerative Diseases (CIBERNED) Hospitalet del Llobregat 08907, Spain.
Hum Mol Genet. 2016 Jun 15;25(12):2417-2436. doi: 10.1093/hmg/ddw108. Epub 2016 Apr 7.
Fatal familial insomnia is a rare disease caused by a D178N mutation in combination with methionine (Met) at codon 129 in the mutated allele of PRNP (D178N-129M haplotype). FFI is manifested by sleep disturbances with insomnia, autonomic disorders and spontaneous and evoked myoclonus, among other symptoms. This study describes new neuropathological and biochemical observations in a series of eight patients with FFI. The mediodorsal and anterior nuclei of the thalamus have severe neuronal loss and marked astrocytic gliosis in every case, whereas the entorhinal cortex is variably affected. Spongiform degeneration only occurs in the entorhinal cortex. Synaptic and fine granular proteinase K digestion (PrPres) immunoreactivity is found in the entorhinal cortex but not in the thalamus. Interleukin 6, interleukin 10 receptor alpha subunit, colony stimulating factor 3 receptor and toll-like receptor 7 mRNA expression increases in the thalamus in FFI. PrPc levels are significantly decreased in the thalamus, entorhinal cortex and cerebellum in FFI. This is accompanied by a particular PrPc and PrPres band profile. Altered PrP solubility consistent with significantly reduced PrP levels in the cytoplasmic fraction and increased PrP levels in the insoluble fraction are identified in FFI cases. Amyloid-like deposits are only seen in the entorhinal cortex. The RT-QuIC assay reveals that all the FFI samples of the entorhinal cortex are positive, whereas the thalamus is positive only in three cases and the cerebellum in two cases. The present findings unveil particular neuropathological and neuroinflammatory profiles in FFI and novel characteristics of natural prion protein in FFI, altered PrPres and Scrapie PrP (abnormal and pathogenic PrP) patterns and region-dependent putative capacity of PrP seeding.
致死性家族性失眠症是一种罕见疾病,由PRNP突变等位基因中的D178N突变与密码子129处的甲硫氨酸(Met)相结合(D178N - 129M单倍型)引起。FFI的表现为睡眠障碍伴失眠、自主神经功能紊乱以及自发和诱发性肌阵挛等症状。本研究描述了一系列8例FFI患者的新神经病理学和生化观察结果。丘脑的内侧背核和前核在每个病例中都有严重的神经元丢失和明显的星形胶质细胞增生,而内嗅皮质受到的影响各不相同。海绵状变性仅发生在内嗅皮质。在内嗅皮质中发现了突触和细颗粒蛋白酶K消化(PrPres)免疫反应性,而在丘脑中未发现。在FFI患者的丘脑中,白细胞介素6、白细胞介素10受体α亚基、集落刺激因子3受体和Toll样受体7的mRNA表达增加。在FFI患者中,丘脑、内嗅皮质和小脑中的PrPc水平显著降低。这伴随着特定的PrPc和PrPres条带图谱。在FFI病例中,发现PrP溶解度改变,与细胞质部分中PrP水平显著降低以及不溶性部分中PrP水平增加一致。淀粉样沉积物仅在内嗅皮质中可见。实时QuIC分析显示,所有内嗅皮质的FFI样本均为阳性,而丘脑仅在3例中为阳性,小脑在2例中为阳性。本研究结果揭示了FFI中特定的神经病理学和神经炎症特征以及FFI中天然朊病毒蛋白的新特性、PrPres和瘙痒病PrP(异常和致病性PrP)模式的改变以及PrP播种的区域依赖性假定能力。