Couratier P, Corcia P, Lautrette G, Nicol M, Marin B
Inserm UMR1094, CRCSLA-fédération Tours-Limoges, service de neurologie, CHU de Limoges, 2, avenue Martin-Luther-King, 87000 Limoges, France.
CRCSLA-fédération Tours-Limoges, CHU de Tours, 37044 Tours cedex 9, France.
Rev Neurol (Paris). 2017 May;173(5):273-279. doi: 10.1016/j.neurol.2017.04.001. Epub 2017 Apr 24.
ALS is now understood to be a complex multisystem neurodegenerative disease because areas other than the motor cortices of the brain undergo degeneration. Frontotemporal dementia (FTD) may be associated with motor neuron disease, and the transactive response DNA-binding protein 43 (TDP-43) is a major pathological substrate underlying both diseases. The recent discovery of a gene that can cause both FTD, ALS and FTD-ALS, C9ORF72, has modified the way for considering these two pathologies. These findings would allow the development of potential biomarkers and therapeutic targets for these devastating diseases. This review summarizes the key points leading up to our current understanding of the genetic, clinical and neuropathological overlap between FTD and ALS.
如今人们认识到,肌萎缩侧索硬化症(ALS)是一种复杂的多系统神经退行性疾病,因为大脑运动皮层以外的区域也会发生退化。额颞叶痴呆(FTD)可能与运动神经元病有关,而反式激活应答DNA结合蛋白43(TDP - 43)是这两种疾病的主要病理底物。最近发现的一种可导致FTD、ALS以及FTD - ALS的基因C9ORF72,改变了我们对这两种病症的认识方式。这些发现将有助于开发针对这些毁灭性疾病的潜在生物标志物和治疗靶点。本综述总结了一些关键点,这些关键点促成了我们目前对FTD和ALS之间遗传、临床及神经病理学重叠的理解。