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一个家族性阿尔茨海默病中新型致病性 PSEN1 突变:表型和神经病理学特征。

A novel pathogenic PSEN1 mutation in a family with Alzheimer's disease: phenotypical and neuropathological features.

机构信息

Centro Regionale di Neurogenetica, ASP Catanzaro, Lamezia Terme (CZ), Italy.

出版信息

J Alzheimers Dis. 2011;25(3):425-31. doi: 10.3233/JAD-2011-110185.

Abstract

We report a novel presenilin1 (PSEN1) gene mutation (I143 V) in a four-generation family with Alzheimer's disease. Clinical, molecular, and neuropathological examinations were performed on index patient; thirteen affected subjects were also identified. The index patient presented at 55 with personality changes, apathy, reduction of verbal fluency, and temporal and spatial disorientation. At 68, she showed visual hallucinations; blurred language, and rigidity. She became bedridden and died at 75. A novel mutation at codon 143 was found in PSEN1 gene, changing isoleucine to valine. The brain showed severe atrophy of the frontal and temporal lobes. Parenchymal amyloid-β (Aβ) deposits were abundant, diffuse to grey structures and contained Aβ42, but very few Aβ40. Amyloid angiopathy was absent. Neurofibrillary changes were severe. Our study confirms that PSEN1 mutations can be associated with unusual phenotypes. The peculiarity of the age at onset (not very early), the long course, and the frontal involvement, together with the rather complete absence of Aβ40 and of amyloid angiopathy, widen the spectrum of PSEN1-linked phenotypes.

摘要

我们报道了一个四代阿尔茨海默病家系中存在早老素 1(PSEN1)基因的一个新突变(I143V)。对先证者进行了临床、分子和神经病理学检查;还确定了 13 名受影响的受试者。先证者 55 岁时出现人格改变、冷漠、言语流畅性降低和时空定向障碍。68 岁时,她出现了视幻觉、语言模糊和僵硬。她卧床不起,75 岁时去世。在 PSEN1 基因中发现了一个新的 143 密码子突变,导致异亮氨酸变为缬氨酸。大脑显示额颞叶严重萎缩。实质淀粉样β(Aβ)沉积物丰富,弥漫于灰质结构,含有 Aβ42,但 Aβ40 很少。淀粉样血管病不存在。神经纤维改变严重。我们的研究证实 PSEN1 突变可与不常见的表型相关。发病年龄(不是非常早)、病程长和额叶受累的特点,以及 Aβ40 和淀粉样血管病的完全缺失,拓宽了 PSEN1 相关表型的范围。

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