PSEN1/SLC20A2双突变导致早发性阿尔茨海默病和原发性家族性脑钙化合并发病。

IF 1.6 4区 医学 Q3 CLINICAL NEUROLOGY
Neurogenetics Pub Date : 2023-07-01 Epub Date: 2023-06-21 DOI:10.1007/s10048-023-00723-x
Sophie Hebestreit, Janine Schwahn, Vesile Sandikci, Mate E Maros, Ivan Valkadinov, Rüstem Yilmaz, Lukas Eckrich, Seyed Babak Loghmani, Hendrik Lesch, Julian Conrad, Holger Wenz, Anne Ebert, David Brenner, Jochen H Weishaupt
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引用次数: 0

摘要

原发性家族性脑钙化(PFBC;以前的Fahr病)和早发性阿尔茨海默病(EOAD)可能有部分重叠的致病原理。尽管杂合子功能丧失突变c.1523 + 1G > 在一名患有不对称震颤、早发性痴呆和脑钙化的患者中检测到PFBC相关基因SLC20A2中的T,CSFβ-淀粉样蛋白参数和FBB-PET提示皮质β-淀粉状蛋白病理。外显子组序列的遗传再分析揭示了可能致病的错义突变c.235G > PSEN1中的A/p.A79T。SLC20A2突变与两名30岁以下儿童的轻度钙化分离。因此,我们描述了遗传性PFBC和遗传性EOAD的随机极不可能合并发病。临床综合征表明这两种突变具有相加作用而非协同作用。MRI数据显示,PFBC钙化的形成早在疾病可能发作的几十年前。我们的报告进一步证明了神经心理学和淀粉样蛋白PET在鉴别诊断中的价值。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

PSEN1/SLC20A2 double mutation causes early-onset Alzheimer's disease and primary familial brain calcification co-morbidity.

PSEN1/SLC20A2 double mutation causes early-onset Alzheimer's disease and primary familial brain calcification co-morbidity.

Primary familial brain calcification (PFBC; formerly Fahr's disease) and early-onset Alzheimer's disease (EOAD) may share partially overlapping pathogenic principles. Although the heterozygous loss-of-function mutation c.1523 + 1G > T in the PFBC-linked gene SLC20A2 was detected in a patient with asymmetric tremor, early-onset dementia, and brain calcifications, CSF β-amyloid parameters and FBB-PET suggested cortical β-amyloid pathology. Genetic re-analysis of exome sequences revealed the probably pathogenic missense mutation c.235G > A/p.A79T in PSEN1. The SLC20A2 mutation segregated with mild calcifications in two children younger than 30 years. We thus describe the stochastically extremely unlikely co-morbidity of genetic PFBC and genetic EOAD. The clinical syndromes pointed to additive rather than synergistic effects of the two mutations. MRI data revealed the formation of PFBC calcifications decades before the probable onset of the disease. Our report furthermore exemplifies the value of neuropsychology and amyloid PET for differential diagnosis.

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来源期刊
Neurogenetics
Neurogenetics 医学-临床神经学
CiteScore
3.90
自引率
0.00%
发文量
24
审稿时长
6 months
期刊介绍: Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry. All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.
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