Pasanen Petra, Myllykangas Liisa, Siitonen Maija, Raunio Anna, Kaakkola Seppo, Lyytinen Jukka, Tienari Pentti J, Pöyhönen Minna, Paetau Anders
Folkhälsan Institute of Genetics, Biomedicum Helsinki, Finland; Department of Medical Biochemistry and Genetics, University of Turku, Turku, Finland.
Folkhälsan Institute of Genetics, Biomedicum Helsinki, Finland; Department of Pathology, University of Helsinki and HUSLAB, Helsinki, Finland.
Neurobiol Aging. 2014 Sep;35(9):2180.e1-5. doi: 10.1016/j.neurobiolaging.2014.03.024. Epub 2014 Mar 26.
We describe the clinical, neuropathological, and genetic features of a Finnish patient with a novel α-synuclein (SNCA) mutation A53E. The patient was clinically diagnosed with atypical Parkinson's disease (PD) with age of onset at 36 years. In the neuropathological analysis performed at the age of 60 years, highly abundant SNCA pathology was observed throughout the brain and spinal cord showing features of multiple system atrophy and PD. Neuronal and glial (including oligodendroglial) SNCA inclusions and neurites were found to be particularly prominent in the putamen, caudatus, amygdala, temporal and insular cortices, gyrus cinguli, and hippocampus CA2-3 region. These areas as well as the substantia nigra and locus coeruleus showed neuronal loss and gliosis. We also found TDP-43 positive but mostly SNCA negative perinuclear inclusions in the dentate fascia of the hippocampus. The A53E mutation was found in 2 other relatives who had parkinsonism. Our results suggest that the novel SNCA A53E substitution is a causative mutation resulting clinically in parkinsonism and pathologically in severe multiple system atrophy- and PD-type phenotype.
我们描述了一名患有新型α-突触核蛋白(SNCA)A53E突变的芬兰患者的临床、神经病理学和遗传学特征。该患者临床诊断为非典型帕金森病(PD),发病年龄为36岁。在60岁时进行的神经病理学分析中,在整个大脑和脊髓中观察到高度丰富的SNCA病理学表现,呈现多系统萎缩和PD的特征。在壳核、尾状核、杏仁核、颞叶和岛叶皮质、扣带回和海马CA2-3区,神经元和胶质细胞(包括少突胶质细胞)中的SNCA包涵体和神经突尤为突出。这些区域以及黑质和蓝斑显示神经元丢失和胶质增生。我们还在海马齿状筋膜中发现了TDP-43阳性但大多为SNCA阴性的核周包涵体。在另外两名患有帕金森症的亲属中发现了A53E突变。我们的结果表明,新型SNCA A53E替代是一种致病突变,临床上导致帕金森症,病理上导致严重的多系统萎缩和PD型表型。