Therapeutic and diagnostic challenges for frontotemporal dementia.
Therapeutic and diagnostic challenges for frontotemporal dementia.
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DOI：
10.3389/fnagi.2014.00204
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发表时间：
2014
影响因子：
4.8
通讯作者：
Lewis J
中科院分区：
文献类型：
--
作者：
D'Alton S;Lewis J
在寻找治疗调节剂的过程中，额颞叶痴呆(FTD)传统上被阿尔茨海默病(AD)等其他疾病所掩盖。FTD是一种临床和病理多样化的疾病，最近的一些发现引发了FTD，例如家族性和散发性疾病的新基因变异，以及在超过一半的病例中发现43 kDa的TAR DNA结合蛋白(TDP-43)作为包裹体的定义成分。结合对tau功能和功能障碍的不断扩展的知识--tau是一种在大多数剩余病例中病理聚集的蛋白质--对FTD的理解比以往任何时候都更多。这些进展可能为假说疗法的发展指明了潜在的方法，但FTD仍然高度复杂，tau和TDP-43在神经退行性变中的作用仍然完全不清楚。这里讨论了潜在的治疗策略面临的挑战，其中包括足够准确的疾病诊断和提供有效治疗的尖端技术。
In the search for therapeutic modifiers, frontotemporal dementia (FTD) has traditionally been overshadowed by other conditions such as Alzheimer’s disease (AD). A clinically and pathologically diverse condition, FTD has been galvanized by a number of recent discoveries such as novel genetic variants in familial and sporadic forms of disease and the identification of TAR DNA binding protein of 43 kDa (TDP-43) as the defining constituent of inclusions in more than half of cases. In combination with an ever-expanding knowledge of the function and dysfunction of tau—a protein which is pathologically aggregated in the majority of the remaining cases—there exists a greater understanding of FTD than ever before. These advances may indicate potential approaches for the development of hypothetical therapeutics, but FTD remains highly complex and the roles of tau and TDP-43 in neurodegeneration are still wholly unclear. Here the challenges facing potential therapeutic strategies are discussed, which include sufficiently accurate disease diagnosis and sophisticated technology to deliver effective therapies.
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影响因子：
16.2
作者：
Ash PE;Bieniek KF;Gendron TF;Caulfield T;Lin WL;Dejesus-Hernandez M;van Blitterswijk MM;Jansen-West K;Paul JW 3rd;Rademakers R;Boylan KB;Dickson DW;Petrucelli L
通讯作者：
Petrucelli L
影响因子：
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影响因子：
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DOI：
10.1073/pnas.94.1.298
发表时间：
1997-01-07
影响因子：
11.1
作者：
Alonso, AD;GrundkeIqbal, I;Iqbal, K
通讯作者：
Iqbal, K
影响因子：
12.7
作者：
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