Wetzel-Smith Monica K, Hunkapiller Julie, Bhangale Tushar R, Srinivasan Karpagam, Maloney Janice A, Atwal Jasvinder K, Sa Susan M, Yaylaoglu Murat B, Foreman Oded, Ortmann Ward, Rathore Nisha, Hansen David V, Tessier-Lavigne Marc, Mayeux Richard, Pericak-Vance Margaret, Haines Jonathan, Farrer Lindsay A, Schellenberg Gerard D, Goate Alison, Behrens Timothy W, Cruchaga Carlos, Watts Ryan J, Graham Robert R
Department of Neuroscience, Genentech, South San Francisco, California, USA.
Department of Human Genetics, Genentech, South San Francisco, California, USA.
Nat Med. 2014 Dec;20(12):1452-7. doi: 10.1038/nm.3736. Epub 2014 Nov 24.
We have identified a rare coding mutation, T835M (rs137875858), in the UNC5C netrin receptor gene that segregated with disease in an autosomal dominant pattern in two families enriched for late-onset Alzheimer's disease and that was associated with disease across four large case-control cohorts (odds ratio = 2.15, Pmeta = 0.0095). T835M alters a conserved residue in the hinge region of UNC5C, and in vitro studies demonstrate that this mutation leads to increased cell death in human HEK293T cells and in rodent neurons. Furthermore, neurons expressing T835M UNC5C are more susceptible to cell death from multiple neurotoxic stimuli, including β-amyloid (Aβ), glutamate and staurosporine. On the basis of these data and the enriched hippocampal expression of UNC5C in the adult nervous system, we propose that one possible mechanism in which T835M UNC5C contributes to the risk of Alzheimer's disease is by increasing susceptibility to neuronal cell death, particularly in vulnerable regions of the Alzheimer's disease brain.
我们在UNC5C网蛋白受体基因中鉴定出一种罕见的编码突变T835M(rs137875858),该突变在两个富含晚发性阿尔茨海默病的家族中以常染色体显性模式与疾病共分离,并且在四个大型病例对照队列中与疾病相关(优势比 = 2.15,Pmeta = 0.0095)。T835M改变了UNC5C铰链区的一个保守残基,体外研究表明,这种突变导致人类HEK293T细胞和啮齿动物神经元中的细胞死亡增加。此外,表达T835M UNC5C的神经元对多种神经毒性刺激(包括β-淀粉样蛋白(Aβ)、谷氨酸和星形孢菌素)导致的细胞死亡更敏感。基于这些数据以及UNC5C在成体神经系统中丰富的海马表达,我们提出T835M UNC5C导致阿尔茨海默病风险增加的一种可能机制是增加对神经元细胞死亡的易感性,特别是在阿尔茨海默病大脑的易损区域。